[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"achondroplasia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:achondroplasia":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,14,0,[8,65,87,112,123,137,171,195,215,239,261,285,305,327],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":54,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":64},"100053847","phase-2-interventional-study-of-infigratinib-in-children--3-years-old-with-achondroplasia-ach-100053847",false,"NCT07169279","Interventional Study of Infigratinib in Children \u003C 3 Years Old With Achondroplasia (ACH)","Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia","Inclusion Criteria:\n\n* Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation.\n* Age 0 to 32 months (2 years and 8 months) at screening.\n* Signed informed consent, which must be obtained from each participant's parent(s) or legal guardian.\n* Parent(s)\u002FGuardian(s) willing and able to attend all study visits and comply with all study requirements.\n* Parent(s)\u002FGuardian(s) willing and able to comply with the routine care of the study participants according to local guidance for the management of infants and young children with ACH.\n* Able to swallow age-appropriate oral medication.\n* In participants \\\u003C1 year old, be compliant with recommended vitamin D supplementation of 5 10 μg\u002Fday or higher (or as recommended by country specific guidelines).\n\nExclusion Criteria:\n\n* Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth.\n* Gestational age at birth \\\u003C37 weeks and\u002For birth weight \\\u003C2500 grams.\n* Gastroesophageal reflux disease requiring prolonged treatment (\\>1 week) with prohibited medications.\n* Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery.\n* History of fracture of a long bone or spine within 6 months prior to screening.\n* Any other significant concurrent disease or condition that, in the view of the investigator and\u002For sponsor, would confound assessment of efficacy or safety of infigratinib and\u002For would require treatment with a prohibited medication (per protocol), and\u002For would place the participant at high risk for poor treatment compliance or for failure to complete the study.\n* Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature, including (but not limited to) r-hGH, IGF-1, CNP analog, FGF ligand trap, or treatment targeting FGFR inhibition at any time.\n* Regular long-term (\\>3 weeks; more than twice\u002Fyear) treatment with supraphysiologic doses of glucocorticoid therapy (ie, \\>15 mg\u002Fm2\u002Fday of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (for over 3 weeks within 6 months of the screening visit. NOTE: Low-dose topical, inhaled, or intranasal corticosteroids are acceptable.\n* Significant abnormality in screening laboratory results,\n* Allergy or hypersensitivity to any components of the study drug.","ALL","0 Years","32 Months",{"count":20,"type":21},77,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \\\u003C 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \\\u003C 3 years old with ACH at the selected dose.",[27],"Achondroplasia",[29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51],"skeletal dysplasia","endochondral ossification","achondroplasia","ACH","Shortened proximal limbs","fibroblast growth factor receptor 3","FGFR3","disproportionate short stature","quality of life","dwarfism","bone diseases","musculoskeletal diseases","osteochondrodysplasia","functional abilities","annualized growth velocity","annualized height velocity","growth","growth disorder","congenital","AHV","AGV","infant and toddler","endochondral bone formation","RECRUITING","2026-07-09",{"date":55,"type":56},"2026-07-13","ACTUAL",{"date":58,"type":56},"2025-11-19",{"date":60,"type":21},"2032-03",{"name":62,"class":63},"QED Therapeutics, a BridgeBio company","INDUSTRY",12,{"id":66,"slug":67,"hasResults":11,"nctId":68,"briefTitle":69,"officialTitle":70,"acronym":4,"eligibilityCriteria":71,"healthyVolunteers":11,"sex":16,"minAge":72,"maxAge":73,"enrollmentInfo":74,"targetDuration":4,"studyType":22,"phases":76,"briefSummary":77,"conditions":78,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":79,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":86},"100053332","phase-2-a-study-of-tyra-300-in-children-with-achondroplasia-beach301-100053332","NCT06842355","A Study of TYRA-300 in Children With Achondroplasia: BEACH301","A Multicenter, Phase 2, Dose-Escalation\u002FDose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301","Inclusion Criteria:\n\n* Aged 3 to 10 years old (inclusive) at the time of consent.\n* Informed consent provided by parent(s) or legal guardian(s). As study participants are less than 18 years old, participants are willing and able to provide written assent (where applicable and required).\n* Molecular diagnosis of achondroplasia (FGFR3 G380R).\n* Radiographically confirmed open growth plates at Screening, as determined by bone age X-ray.\n* Able to stand and ambulate independently.\n* Able to take oral medication.\n* Sentinel Safety Cohort only: aged 5 to 10 years old (inclusive).\n* Cohort 1 only: aged 3 to 10 years old (inclusive) and are naive to prior growth accelerating therapy.\n* Cohort 2 only: aged 3 to 10 years old (inclusive) and have received prior growth accelerating therapy.\n\nExclusion Criteria:\n\n* Presence or history of any concurrent disease or condition that would interfere with study participation, safety evaluations, or any uncontrolled or untreated condition that could impact pediatric growth.\n* Diagnosis of endocrine condition that alters calcium\u002Fphosphate homeostasis.\n* Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolled in the study.\n* Taking medications that are strong inhibitors or inducers of cytochrome P450 (Cyp) 3A4.\n* History or current evidence of corneal or retinal disorder\u002Fkeratopathy.\n* Presence of guided growth hardware\u002F8 plates. Planned or anticipated orthopedic surgeries.","3 Years","10 Years",{"count":75,"type":21},92,[24],"The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with open growth plates.",[27],{"date":55,"type":56},{"date":81,"type":56},"2025-03-04",{"date":83,"type":21},"2030-06",{"name":85,"class":63},"Tyra Biosciences, Inc",19,{"id":88,"slug":89,"hasResults":11,"nctId":90,"briefTitle":91,"officialTitle":91,"acronym":92,"eligibilityCriteria":93,"healthyVolunteers":11,"sex":16,"minAge":94,"maxAge":95,"enrollmentInfo":96,"targetDuration":98,"studyType":99,"phases":4,"briefSummary":100,"conditions":101,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":102,"lastUpdatePostDateStruct":103,"startDateStruct":105,"completionDateStruct":107,"leadSponsor":109,"locationsCount":111},"100529009","virtual-study-in-achondroplasia-for-the-us-vista-100529009","NCT06168201","VIrtual STudy in Achondroplasia for the US (VISTA)","VISTA","Inclusion Criteria:\n\n* Physician diagnosis of achondroplasia\n* Age at time of enrollment:\n\nPrimary Pediatric Cohort: ≤13 years old Secondary Adolescent and Adult Cohort: ≥14 years old\n\n* Receiving medical care in the United States\n* Complete PicnicHealth's onboarding process, including signing informed consent and authorization for medical record retrieval\n\nExclusion Criteria:\n\n* Lack of any medical records","0 Months","18 Years",{"count":97,"type":21},170,"5 Years","OBSERVATIONAL","This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor.\n\nData will be collected in two formats for the primary pediatric study population:\n\n1. Participant-mediated access to electronic health records(including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. .\n2. Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data.\n\nData will be collected in the following format for the adult cohort:\n\n\\- Participant-mediated access to electronic health records (including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care.\n\nThe primary study population will include approximately 150 pediatric individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa) however they will only be counted once, based on their treated status at the point of enrollment.\n\nThe secondary study population will include 20 adolescent and adult participants.",[27],"2026-06-29",{"date":104,"type":56},"2026-06-30",{"date":106,"type":56},"2023-02-21",{"date":108,"type":21},"2028-02",{"name":110,"class":63},"BioMarin Pharmaceutical",8,{"id":113,"slug":4,"hasResults":11,"nctId":68,"briefTitle":69,"officialTitle":70,"acronym":4,"eligibilityCriteria":71,"healthyVolunteers":11,"sex":16,"minAge":72,"maxAge":73,"enrollmentInfo":114,"targetDuration":4,"studyType":22,"phases":115,"briefSummary":77,"conditions":116,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":118,"startDateStruct":119,"completionDateStruct":120,"leadSponsor":121,"locationsCount":122},"100580826",{"count":75,"type":21},[24],[27],"2026-06-27",{"date":104,"type":56},{"date":81,"type":56},{"date":83,"type":21},{"name":85,"class":63},18,{"id":124,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":125,"targetDuration":4,"studyType":22,"phases":126,"briefSummary":25,"conditions":127,"keywords":128,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":131,"startDateStruct":133,"completionDateStruct":134,"leadSponsor":135,"locationsCount":136},"100605956",{"count":20,"type":21},[24],[27],[29,30,31,32,33,34,35,129,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50],"encodhondral bone formation","2026-06-16",{"date":132,"type":56},"2026-06-17",{"date":58,"type":56},{"date":60,"type":21},{"name":62,"class":63},13,{"id":138,"slug":139,"hasResults":11,"nctId":140,"briefTitle":141,"officialTitle":142,"acronym":143,"eligibilityCriteria":144,"healthyVolunteers":11,"sex":16,"minAge":145,"maxAge":146,"enrollmentInfo":147,"targetDuration":4,"studyType":22,"phases":149,"briefSummary":151,"conditions":152,"keywords":153,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":162,"lastUpdatePostDateStruct":163,"startDateStruct":165,"completionDateStruct":167,"leadSponsor":169,"locationsCount":170},"100626918","phase-2-study-to-evaluate-the-efficacy-and-safety-of-bmn-333-versus-vosoritide-in-children-with-achondroplasia-100626918","NCT07441876","Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia","A Multicenter, Randomized, Operationally Seamless Phase 2\u002F3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia","ASPEN","Inclusion Criteria:\n\n1. Participants must be aged ≥ 2 to \\\u003C 11 years (Phase 2) or ≥ 2 to \\\u003C 18 years (Phase 3), at the time of signing the informed consent\n2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses\n3. Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)\n4. Are ambulatory and able to stand without assistance\n\nExclusion Criteria:\n\n1. Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency)\n2. Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated with evidence of normalized thyroid-stimulating hormone (TSH) levels, diabetes mellitus, unless considered well-controlled, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy, anemia defined as hemoglobin \\\u003C 10 g\u002FdL, vitamin D deficiency, significant hip pathology.\n3. Have history of any renal insufficiency or cardiac\u002F cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension.\n4. Have had bone fractures of the long bones or spine within 6 months prior to screening.\n5. Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the treatment of ACH or short stature at any time\n6. Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start","2 Years","17 Years",{"count":148,"type":21},160,[24,150],"PHASE3","This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2\u002F3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH). The study consists of a Phase 2 part and a Phase 3 part.",[27],[27,32,154,155,156,157,158,159,160,161],"Bone Diseases, Developmental Dwarfism","Bone Diseases","Genetic Diseases, Inborn","Musculoskeletal Diseases","Natriuretic Peptide, C-type","Osteochondrodysplasias","Physiological Effects of Drugs","Skeletal Dysplasias","2026-05-27",{"date":164,"type":56},"2026-05-29",{"date":166,"type":21},"2026-04",{"date":168,"type":21},"2029-09",{"name":110,"class":63},10,{"id":172,"slug":173,"hasResults":11,"nctId":174,"briefTitle":175,"officialTitle":176,"acronym":177,"eligibilityCriteria":178,"healthyVolunteers":11,"sex":16,"minAge":179,"maxAge":146,"enrollmentInfo":180,"targetDuration":4,"studyType":22,"phases":182,"briefSummary":183,"conditions":184,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":185,"lastUpdatePostDateStruct":186,"startDateStruct":188,"completionDateStruct":190,"leadSponsor":192,"locationsCount":194},"100572409","phase-2-a-clinical-trial-to-evaluate-efficacy-and-safety-of-navepegritide-in-adolescents-12---18-years-of-age-with-achondroplasia-100572409","NCT06732895","A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.","A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-18 Years of Age) With Achondroplasia.","teACH","Inclusion Criteria:\n\n* Written, signed informed consent and\u002For assent of the participant, participant parent(s) or legal guardian(s) of the participant, and as required by the institutional review board\u002Fhuman research ethics committee\u002Findependent ethics committee (IRB\u002FHREC\u002FIEC). For participants who are below the age of consent, a written assent will be obtained in accordance with applicable requirements as required by IRB\u002FHREC\u002FIEC. Upon reaching the legal age of consent, depending on applicable requirements, these participants will be asked to give their own written consent.\n* Male or female, between 12 (inclusive) and 18 years of age at the time of randomization\n* Clinical diagnosis of ACH with documented genetic confirmation available. Documentation of historic test results are acceptable for proof of diagnosis.\n* Parent(s)\u002Flegal guardian(s) willing and able to administer weekly SC injections of IMP and to follow the protocol.\n* At least one historical standing height measurement available from medical records. The measurement must have been collected between 6 months to 15 months prior to the time of screening.\n\nExclusion Criteria:\n\n* Participation (signed informed consent) in any interventional clinical trial within 3 months prior to Screening unless no doses of IMP was given.\n* Decreased growth velocity (AGV less than 1.5 cm\u002Fyear based on measurement over a period of at least 6 months) or radiological evidence of growth plate closure.\n* Known or suspected hypersensitivity to the IMP or related products (trehalose, tris\\[hydroxymethyl\\]aminomethane, succinate, and mPEG).\n* Have a growth disorder or medical condition other than ACH that results in short stature, or abnormal growth such as SADDAN, hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudo-ACH, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, or diabetes mellitus.\n* Severe mutation in the FGFR3 gene, e.g. two variants on the same allele or severe ACH with developmental delay and acanthosis nigricans, are not eligible for trial participation.\n* Have received any dose of prescription medications and\u002For IMP (placebo treatment only is allowed, if documented) or surgical intervention intended to affect stature, growth, or body proportionality at any time.\n* Requires, or anticipated to require, chronic (more than 4 weeks) or repeated treatment (more than twice\u002Fyear and less than 3 weeks\u002Fyear) with systemic corticosteroids during participation in the trial. Chronic use of high dose inhaled corticosteroids is not allowed.\n* Known history of presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.\n* Known history of any bone-related surgery affecting growth potential of long bones, such as:\n\n  * Orthopedic reconstructive surgery for bone lengthening (e.g., procedures for leg bowing such as 8-plate are not exclusionary).\n  * Ventriculoperitoneal (VP) shunt and laminectomy with full recovery are allowed with minimum of 6 months of bone healing.\n  * Bone fracture within 6 months prior to screening (within 2 months for fracture of digits and buckle fractures).\n* Clinically significant findings at Screening, such as:\n\n  * Expected to require surgical intervention during participation in the trial that may significantly affect trial parameters (confounding of safety events) or would prevent the participant from performing trial procedures. Common surgeries, such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement, are permitted.\n  * Severe untreated sleep apnea or newly initiated sleep apnea treatment (e.g., Continuous Positive Airway Pressure \\[CPAP\\] in the previous 2 months prior to Screening.\n  * MS disease, such as Salter-Harris fractures or clinical and\u002For radiographic evidence of severe hip pathology\n  * Otherwise, are considered by the Investigator to be unfit to receive trial treatment or undergo trial related procedures.\n* Have a clinically significant finding or arrhythmia as determined by the investigator in consultation with the medical monitor that indicates abnormal cardiac function or conduction that includes, but is not exclusive to:\n\n  * Repaired or unrepaired coarctation.\n  * Moderate or greater complexity congenital heart disease including tetralogy of Fallot, Atrioventricular septal defects, truncus arteriosus, total anomalous pulmonary venous return, double outlet right ventricle, or single ventricle heart disease.\n* QT corrected using Fridericia's correction (QTcF) ≥ 450 msec at Screening.\n* Known history or presence of condition that impacts hemodynamic stability (such as autonomic dysfunction and orthostatic intolerance).\n* Known history or presence of the following:\n\n  * Chronic anemia (iron deficiency anemia that is resolved or adequately treated in the Investigator's opinion is allowed).\n  * Chronic renal insufficiency defined as estimated glomerular filtration rate (eGFR) according to the revised bedside Schwartz equation less than 60 mL\u002Fmin\u002F1.73 m2 for more than 3 months.\n  * Chronic or recurrent illness that can affect hydration or volume status, including conditions associated with decreased nutritional intake or increased volume loss.\n* Known history or presence of malignant disease.\n* Participant with serum 25-hydroxy-vitamin D (25OHD) levels of less than 30 nmol\u002FL (less than 12 ng\u002FmL) at Screening Visit will be excluded. Participants with 25OHD levels between 30-50 nmol\u002FL (12-20 ng\u002FmL) can be randomized provided treatment with Vitamin D supplementation is initiated according to local standards.\n* Any disease or condition that, in the opinion of the Investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, complications or manifestations, or medications that might impact safety or be considered confounding.\n* Sexually active male and female participants and female partners of male participants of childbearing potential not using a highly effective form of contraceptive for the entire trial period and for 90 days after last dose of trial treatment.\n* Female participants who are pregnant, lactating or breastfeeding.","12 Years",{"count":181,"type":21},24,[24],"The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg\u002Fkg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.",[27],"2026-04-20",{"date":187,"type":56},"2026-04-21",{"date":189,"type":56},"2024-12-10",{"date":191,"type":21},"2027-09",{"name":193,"class":63},"Ascendis Pharma A\u002FS",5,{"id":196,"slug":197,"hasResults":11,"nctId":198,"briefTitle":199,"officialTitle":200,"acronym":4,"eligibilityCriteria":201,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":145,"enrollmentInfo":202,"targetDuration":4,"studyType":22,"phases":204,"briefSummary":205,"conditions":206,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":207,"lastUpdatePostDateStruct":208,"startDateStruct":210,"completionDateStruct":212,"leadSponsor":214,"locationsCount":122},"100522190","phase-2-a-clinical-trial-to-evaluate-efficacy-and-safety-of-transcon-cnp-compared-with-placebo-in-infants-0-to-2-years-of-age-with-achondroplasia-100522190","NCT06079398","A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to \u003C2 Years of Age) With Achondroplasia","A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to \u003C2 Years of Age) With Achondroplasia Followed by an Open Label Extension (OLE) Period","Inclusion Criteria:\n\n* Written, signed informed consent by the parent(s)\u002Fcaregiver(s) of the participant, and as required by the institutional review board\u002Fhuman research ethics committee\u002Findependent ethics committee (IRB\u002FHREC\u002FIEC).\n* Male or female younger than 2 years of age at the time of randomization; or for open label sentinel participants, at the time of first administration of IMP.\n* Clinical diagnosis of achondroplasia (ACH) with genetic confirmation of heterozygous genotype present during screening.\n* Parent(s)\u002Fcaregiver(s) willing to follow the protocol and instructions provided, including being able to administer weekly subcutaneous injections of trial treatment.\n* Compliance to daily Vitamin D supplementation for infants aged 14 days to 1 year. All participants older than 1 year of age with serum 25-hydroxyvitamin D (25OHD) measured below lower limit of reference range at screening should start daily Vitamin D supplementation prior to randomization.\n* Considered eligible based on the medical history, physical examination, and the results of vital signs, ECG, imaging, and clinical laboratory tests performed during the screening period.\n\nExclusion Criteria:\n\n* Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris\\[hydroxymethyl\\]aminomethane, succinate, and polyethylene glycol \\[PEG\\]).\n* Genetic confirmation of ACH homozygous genotype.\n* Premature birth with gestational age \\\u003C 32 weeks.\n* Premature birth with gestational age 32 to 37 weeks, unless time from birth is \\> 6 months at the time of screening and the child is in good nutritional status, defined as gain in body weight expected for age and diagnosis of ACH, as determined by the Investigator and confirmed with the Medical Monitor.\n* Anticipated, as assessed by Investigator and confirmed with Medical Monitor, to undergo surgical intervention during trial participation, including cervicomedullary decompression. Evaluation of immediate risk of requiring cervicomedullary decompression surgery will rely on the following assessments:\n\n  * Physical examination (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)\n  * Evidence of uncontrolled sleep apnea as confirmed by local standard of care assessment (e.g. polysomnography or simple sleep test) performed within 6 months prior to screening.\n  * MRI performed at screening indicating presence of severe cervicomedullary compression (CMC) or spinal cord damage. Presence of abnormal MRI T2 signal intensity at and immediately above and below the cervicomedullary junction should be considered high risk for requiring surgery and the participant is not eligible for trial participation.\n\nCommon surgeries, such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement are permitted during trial participation.\n\n* Have a growth disorder or medical condition, other than ACH, resulting in short stature or abnormal growth as determined by the Investigator and confirmed with the Medical Monitor.\n* Have received any dose of prescription medications and\u002For investigational medicinal product or device intended to affect stature, growth, or body proportionality (including human growth hormone or vosoritide) at any time.\n* Requires or anticipated to require chronic (\\> 4 weeks) or repeated treatment (more than twice\u002Fyear) with oral corticosteroids, or high-dose inhaled corticosteroids during trial participation.\n* History or presence of injury or disease of the growth plate(s), other than ACH, affecting growth potential of long bones, including Salter-Harris fracture and recent bone-related surgery, as determined by Investigator and confirmed with the Medical Monitor.\n* Have a clinically significant finding indicating abnormal cardiac function, including but not limited to:\n\n  * Repaired or unrepaired coarctation.\n  * Moderate or greater complexity congenital heart disease including tetralogy of Fallot, atrioventricular septal defects, truncus arteriosus, total anomalous pulmonary venous return, double outlet right ventricle, or single ventricle heart disease.\n  * QTcF ≥ 450 msec on screening 12-lead ECG.\n* History or presence of a condition impacting hemodynamic stability (such as autonomic dysfunction and orthostatic intolerance).\n* History or presence of the following:\n\n  * Chronic anemia.\n  * Chronic renal insufficiency.\n  * Chronic or recurrent illness that can affect hydration or volume status, including conditions associated with decreased nutritional intake or increased volume loss.\n* History or presence of malignant disease.\n* Any disease or condition that, in the opinion of the Investigator, may make the participant unlikely to fully complete the trial, not adhering to trial procedures, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, comorbid conditions, or medications that might impact safety or be considered confounding.",{"count":203,"type":21},72,[24],"This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP\u002Fkg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \\\u003C 2 years at the time of randomization.",[27],"2026-04-07",{"date":209,"type":56},"2026-04-08",{"date":211,"type":56},"2024-01-23",{"date":213,"type":21},"2028-12",{"name":193,"class":63},{"id":216,"slug":217,"hasResults":11,"nctId":218,"briefTitle":219,"officialTitle":220,"acronym":4,"eligibilityCriteria":221,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":222,"targetDuration":98,"studyType":99,"phases":4,"briefSummary":224,"conditions":225,"keywords":226,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":228,"lastUpdatePostDateStruct":229,"startDateStruct":231,"completionDateStruct":233,"leadSponsor":235,"locationsCount":238},"100254969","achondroplasia-natural-history-multicenter-clinical-study-100254969","NCT02597881","Achondroplasia Natural History Multicenter Clinical Study","Registry of Patients With Bone Disorders","Inclusion Criteria:\n\n* Molecular or clinical diagnosis of achondroplasia (as confirmed by physical exam and\u002For radiograph review by the PI, one of the co-PIs or other qualified clinical geneticists)\n* Subjects must have been seen for a clinical genetics visit at Johns Hopkins, Alfred I. DuPont Hospital for Children, University of Wisconsin-Madison or University of Texas\n* Subjects may be active clinical patients at the above sites or no longer treated at a given site but with sufficient retrospective clinical data for extraction as determined by the PI or co-PIs\n\nExclusion Criteria:\n\n* Skeletal dysplasia diagnosis other than heterozygous\n* Achondroplasia\n* There is no medical complication or condition which excludes a patient with achondroplasia",{"count":223,"type":21},1500,"The purpose of this study is to create an electronic registry to house phenotypic information from patients with achondroplasia. The initial focus of this registry will be to include U.S. patients with achondroplasia. Once populated, the collective data can be queried to pursue clinical research questions pertaining to health outcomes and treatment options for patients with this conditions. The registry is longitudinal in nature with the functionality to retrospectively enter patients' clinical data from the prenatal period up through the most recent encounter, with all intervening data entered in a chronologic fashion.",[27],[227],"natural history","2026-03-27",{"date":230,"type":56},"2026-04-01",{"date":232,"type":56},"2016-04",{"date":234,"type":21},"2030-12-31",{"name":236,"class":237},"Johns Hopkins University","OTHER",1,{"id":240,"slug":241,"hasResults":11,"nctId":242,"briefTitle":243,"officialTitle":244,"acronym":245,"eligibilityCriteria":246,"healthyVolunteers":11,"sex":16,"minAge":72,"maxAge":247,"enrollmentInfo":248,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":250,"conditions":251,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":253,"lastUpdatePostDateStruct":254,"startDateStruct":256,"completionDateStruct":258,"leadSponsor":259,"locationsCount":238},"100622849","prospective-longitudinal-monocentric-study-to-measure-limb-movement-in-patients-with-fgfr3-related-skeletal-dysplasia-100622849","NCT07388966","Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia","Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia Using Syde®","SKY-MOT3","Inclusion Criteria:\n\n1. Aged 3 years old or older at the time of consent.\n2. Written informed consent obtained:\n\n   1. From the parent(s) or legal guardian(s) for participants under 18 years old.\n   2. From participants 18 years old and older.\n3. Affiliated to, or beneficiary of a social security category\n4. Able to walk unassisted for at least 10 meters.\n5. Genetically confirmed diagnosis with one pathogenic variant (ACMG class IV \\& V) of achondroplasia or hypochondroplasia.\n6. Participant (and caregivers for participants under 18 years old) willing and able to comply with all study procedures including: questionnaires, Syde® related procedures\n\nExclusion Criteria:\n\n1. Subjects who have short stature condition other than ACH\u002FHCH.\n2. Presence of cognitive disorders that limit their understanding of the data collection process (training of device use and 4-week recording periods every 6 months, device return at the end of the study), the implication of the study and consent.\n3. Presence or history of any concurrent disease or condition that could interfere with study participation, impact pediatric growth, affect motor or balance or gait function (such as neurological, endocrine, infectious, allergic, osteoarthritis, or inflammatory), assessed by the investigator.\n4. Females who are pregnant, or planning to become pregnant during the study duration.\n5. Body Mass Index = 35 kg\u002Fm2.\n6. Recent upper and\u002For lower limbs injury (trauma\u002Ffracture or surgery) in the 6 months preceding inclusion.\n7. Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolled in the study.\n8. Presence of guided growth hardware (such as 8-plates) or planned orthopedic surgeries during the study.\n9. Vulnerable patient (guardianship, curatorship or safeguarding of justice), unable to provide informed consent or who is unable to express their consent.","65 Years",{"count":249,"type":21},40,"The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.",[27,252],"Hypochondroplasia","2026-02-18",{"date":255,"type":56},"2026-02-20",{"date":257,"type":56},"2026-02-06",{"date":108,"type":21},{"name":260,"class":63},"SYSNAV",{"id":262,"slug":263,"hasResults":11,"nctId":264,"briefTitle":265,"officialTitle":266,"acronym":4,"eligibilityCriteria":267,"healthyVolunteers":11,"sex":16,"minAge":72,"maxAge":179,"enrollmentInfo":268,"targetDuration":4,"studyType":22,"phases":270,"briefSummary":272,"conditions":273,"keywords":4,"overallStatus":274,"whyStopped":4,"lastUpdateSubmitDate":275,"lastUpdatePostDateStruct":276,"startDateStruct":278,"completionDateStruct":280,"leadSponsor":282,"locationsCount":284},"100615843","phase-1-a-study-of-absk061-to-assess-safety-tolerability-pharmacokinetics-and-efficacy-in-children-with-achondroplasia-100615843","NCT07297875","A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia","A Phase Ⅰ\u002FⅡ, Open-Label Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia","Inclusion Criteria:\n\n1. Prior to screening, the guardians and children with ACH (if applicable) must voluntarily provide signed informed consent.\n2. Patients with a clear clinical diagnosis of ACH confirmed by genetic testing for an FGFR3 mutation.\n3. Male or female, age at screening:\n\n   Dose Escalation Part A: 6 to \\\u003C 12 years (inclusive 6 years) Dose Escalation Part B: 3 to \\\u003C 6 years (inclusive 3 years) RDE Confirmation Part: 3 to \\\u003C 12 years (inclusive 3 years).\n4. Have completed at least 6 months (i.e., the \"Day 181\" visit) of growth assessment and observation of natural history of ACH in the observational study (ABSK061-001) before study entry.\n5. Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening\n\nExclusion Criteria:\n\n1. Known allergy or hypersensitivity to any component of the study drug.\n2. Bone age ≥ 14 years as assessed by the investigator based on hand and wrist X-ray.\n3. Have a form of skeletal dysplasia other than ACH or known medical conditions that result in short stature or abnormal growth, including but not limited to severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, chronic renal insufficiency, active celiac disease a, Vitamin D deficiency b, untreated hypothyroidism c, poorly controlled diabetes (HbA1c ≥8.0%) or diabetic complications\n4. History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.\n5. AGV ≤ 1.5 cm\u002Fyear over at least 6 months (i.e., must have completed the 'Day 181' visit) in the observational study (ABSK061-001), or current evidence of growth plate closure (proximal tibia, distal femur).\n6. Current epiphyseal injury (Salter-Harris fracture) or severe hip pain.\n7. For ACH-related complications: current severe sleep apnea, symptomatic and\u002For requiring intervention for hydrocephalus, or spinal cord compression at the cranio-cervical junction, or prior ventriculoperitoneal shunt surgery.\n8. Have received any dose of medications affecting stature or body proportionality, such as human growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids within 12 months prior to screening.\n9. Prior treatment with any CNP analogues or FGFR inhibitors. Prior use of any investigational drugs or investigational medical devices that affect height or body proportion.\n10. History of any prior bone-related surgery that affects long bone growth, such as orthopaedic reconstructive surgery, limb lengthening, or osteotomy (patients who have previously undergone foramen magnum decompression or intervertebral disc\u002Flaminectomy are allowed if they have fully recovered after surgery and bone healing has occurred for at least 6 months. Patients who have previously undergone eight-plate epiphysiodesis are allowed if the plate has been removed and healed for at least 4 weeks).",{"count":269,"type":21},110,[271,24],"PHASE1","This is a multicenter, non-randomized, open-label, phase I\u002FII study in children with ACH. This study will start with a dose escalation of ABSK061 in children with ACH to evaluate the safety, tolerability, PK, and efficacy. The RDE confirmation part will evaluate the safety and efficacy of ABSK061 at the recommended doses for expansion (RDEs) in children with ACH. All patients enrolled in the dose escalation part and RDE confirmation part can enter the extended treatment period to further evaluate the long-term safety, tolerability, and long-term efficacy of ABSK061 in children with ACH.",[27],"NOT_YET_RECRUITING","2025-12-17",{"date":277,"type":56},"2025-12-22",{"date":279,"type":21},"2025-12-10",{"date":281,"type":21},"2031-03-15",{"name":283,"class":63},"Abbisko Therapeutics Co, Ltd",7,{"id":286,"slug":287,"hasResults":11,"nctId":288,"briefTitle":289,"officialTitle":290,"acronym":4,"eligibilityCriteria":291,"healthyVolunteers":11,"sex":16,"minAge":292,"maxAge":293,"enrollmentInfo":294,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":296,"conditions":297,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":279,"lastUpdatePostDateStruct":298,"startDateStruct":300,"completionDateStruct":302,"leadSponsor":304,"locationsCount":111},"100616119","a-study-in-children-with-achondroplasia-100616119","NCT07301463","A Study in Children With Achondroplasia","A Multicenter, Longitudinal, Observational Study in Children With Achondroplasia","Inclusion Criteria:\n\n1. Prior to screening, the guardians and children with ACH (if applicable) must be willing and able to provide signed informed consent.\n2. Clinical diagnosis of ACH confirmed FGFR3 mutation by genetic testing.\n3. Male or female aged ≥2.5 to \\\u003C11 years old at screening.\n4. Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening.\n5. Ambulatory and able to stand without assistance.\n\nExclusion Criteria:\n\n1. Bone age ≥14 years as assessed by the investigator based on hand and wrist X-ray taken within 6 months prior to Day 1.\n2. Current evidence of growth plate closure (proximal tibia, distal femur), or AGV ≤ 1.5 cm\u002Fyear over a period ≥6 months prior to screening.\n3. Have a form of skeletal dysplasia other than ACH or known medical conditions that result in short stature or abnormal growth, including but not limited to severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, chronic renal insufficiency, active celiac disease a, Vitamin D deficiency b, untreated hypothyroidism c, poorly controlled diabetes (HbA1c ≥8.0%) or diabetic complications d.\n\n   1. Celiac disease responsive to a gluten-free diet is allowed\n   2. Vitamin D deficiency or insufficiency with a 25-hydroxyvitamin D \\[25- (OH) D\\] level ≥ 30 nmol\u002FL after supplementation is allowed. Vitamin D deficiency is defined as 25-(OH) D level \\\u003C30 nmol\u002FL. Vitamin D insufficiency is defined as 25-(OH) D level 30\\~50 nmol\u002FL. Patients with Vitamin D deficiency or insufficiency must be on Vitamin D regimen prior to screening\n   3. Patients with hypothyroidism meeting the following criteria are allowed to enroll: must be clinically euthyroid for one month prior to screening and, in the opinion of the investigator, have achieved any catch-up growth expected from thyroxine replacement\n   4. Patients with diabetes must have been on stable medication regimen for 3 months prior to screening\n4. History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.\n5. Impaired cardiac function or clinically significant cardiovascular disease, including any one of the following: New York Heart Association class II or higher heart disease, congenital heart disease (patients with repaired uncomplicated patent ductus arteriosus or atrial\u002Fventricular septal defect with repair are allowed), clinically significant arrhythmias requiring therapy, aortic regurgitation, congestive heart failure, or any other uncontrolled heart disease.\n6. For ACH-related complications: Current severe sleep apnea, symptomatic and\u002For requiring intervention for hydrocephalus, or spinal cord compression at the cranio-cervical junction, and has previously undergone ventriculoperitoneal shunt surgery.\n7. Bone fracture within 6 months prior to screening (within 2 months for finger and toe fractures).\n8. Have received any dose of medications affecting stature or body proportionality, such as human growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids within 3 months prior to screening, or long-term treatment (\\>3 months) with the above drugs at any time.\n9. Prior treatment with any CNP analogues or FGFR inhibitors. Prior use of any investigational drugs or investigational medical devices that affect stature or body proportionality.\n10. Any comorbidities, disease or condition that, in the opinion of the investigator, may make the patient unlikely to fully complete the study-related procedures, may affect protocol compliance.","30 Months","11 Years",{"count":295,"type":21},260,"The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural history observation of ACH for at least 6 months and up to 2 years.",[27],{"date":299,"type":56},"2025-12-24",{"date":301,"type":56},"2025-06-20",{"date":303,"type":21},"2039-04-30",{"name":283,"class":63},{"id":306,"slug":307,"hasResults":11,"nctId":308,"briefTitle":309,"officialTitle":310,"acronym":4,"eligibilityCriteria":311,"healthyVolunteers":11,"sex":16,"minAge":72,"maxAge":95,"enrollmentInfo":312,"targetDuration":4,"studyType":22,"phases":314,"briefSummary":315,"conditions":316,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":317,"lastUpdatePostDateStruct":318,"startDateStruct":320,"completionDateStruct":322,"leadSponsor":324,"locationsCount":326},"100587292","phase-3-evaluate-the-efficacy-and-safety-of-kk8398-in-patients-with-achondroplasiaaoba-study-100587292","NCT06926491","Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）","A Multicenter, Open-label, Single-arm, Phase 3 Study of KK8398 (Infigratinib) in Patients With Achondroplasia (AOBA Study)","Inclusion Criteria:\n\n1. Patients who are between 2.5 and 17.5 years old of age at the time of provisional registration. Patients who meet certain criteria in terms of Tanner stage and bone age.\n2. Patients who have been diagnosed with achondroplasia through genetic testing.\n\nExclusion Criteria:\n\n1. Patients who meet the criteria for a certain height determined by age and gender in the \"Growth Chart for Patients with Achondroplasia\" at the time of the pre-registration examination\n2. Patients who have received treatment with r-hGH or a CNP analogue within a certain period prior to the pre-enrollment examination.\n3. Patients who have had previous osteotomy or who are scheduled to undergo osteotomy or epiphyseal growth inhibition during the study period. Epiphyseal growth inhibition in which the plate was removed more than 26 weeks prior to the pre-registration examination will not be excluded if the patient has completely healed without sequelae, based on the judgment of the investigator or sub-investigator.",{"count":313,"type":21},6,[150],"This trial will evaluate the efficacy of KK8398 on annualized height velocity after 52 weeks of repeated administration of KK8398 to patients with achondroplasia",[27],"2025-04-28",{"date":319,"type":56},"2025-04-29",{"date":321,"type":56},"2024-12-01",{"date":323,"type":21},"2031-05-31",{"name":325,"class":63},"Kyowa Kirin Co., Ltd.",9,{"id":328,"slug":329,"hasResults":11,"nctId":330,"briefTitle":331,"officialTitle":332,"acronym":333,"eligibilityCriteria":334,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":335,"targetDuration":73,"studyType":99,"phases":4,"briefSummary":337,"conditions":338,"keywords":4,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":339,"lastUpdatePostDateStruct":340,"startDateStruct":342,"completionDateStruct":344,"leadSponsor":346,"locationsCount":238},"100464465","registry-for-patients-with-achondroplasia--hypochondroplasia-ompr-achhy-100464465","NCT05328050","Registry for Patients With Achondroplasia \u002F Hypochondroplasia (OMPR-Ach\u002FHy)","Local Registry for Data Collection of Patients With Achondroplasia \u002F Hypochondroplasia for Epidemiological, Care and Research Studies","OMPR-Ach\u002FHy","Inclusion Criteria:\n\n* Confirmed diagnosis of achondroplasia\u002Fhypochondroplasia\n* Patients (and\u002For Parents legal guardian when required) able to provide informed consent\n\nExclusion Criteria:\n\n* Absence of diagnosis of achondroplasia\u002Fhypochondroplasia\n* Patients (and\u002For Parents legal guardian when required) not able to provide informed consent",{"count":336,"type":21},200,"This registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.",[27,252],"2022-04-21",{"date":341,"type":56},"2022-04-28",{"date":343,"type":56},"2021-09-01",{"date":345,"type":21},"2036-12-31",{"name":347,"class":237},"Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico"]