[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"BioMarin Pharmaceutical\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":299},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,11,0,[8,40,63,99,133,156,181,204,229,254,279],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":22,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100529009","virtual-study-in-achondroplasia-for-the-us-vista-100529009",false,"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","ALL","0 Months","18 Years",{"count":20,"type":21},170,"ESTIMATED","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.",[26],"Achondroplasia","RECRUITING","2026-06-29",{"date":30,"type":31},"2026-06-30","ACTUAL",{"date":33,"type":31},"2023-02-21",{"date":35,"type":21},"2028-02",{"name":37,"class":38},"BioMarin Pharmaceutical","INDUSTRY",8,{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":48,"enrollmentInfo":49,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":51,"conditions":52,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":54,"lastUpdatePostDateStruct":55,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":62},"100539910","a-study-to-assess-growth-in-children-with-idiopathic-short-stature-100539910","NCT06309979","A Study to Assess Growth in Children With Idiopathic Short Stature","A Multicenter, Observational Study to Characterize Growth in Children With Idiopathic Short Stature","Inclusion Criteria:\n\n1. Participants must be \\> 2 years old, and ≤ 14 years old (female) or ≤ 16 years old (males) at the time of signing the informed consent.\n2. A height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth chart (https:\u002F\u002Fwww.cdc.gov\u002Fgrowthcharts\u002Fzscore.htm).\n3. Participants who have either never received hGH, or who are currently receiving hGH treatment.\n4. Historic stimulation test result with serum or plasma GH level greater than 10 μg\u002FL.\n5. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.\n\nExclusion Criteria:\n\n1. Diagnosis of systemic disease or condition that may cause short stature, eg renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. Children with such diagnoses can be considered for inclusion if their condition is well controlled, at the discretion of the Medical Monitor.\n2. Known presence of one or more pituitary hormone deficiencies\n3. Bone age advanced over chronological age by more than 3 years.\n4. For hGH naïve participants, historic stimulation test result with serum or plasma GH level greater than 10 μg\u002FL or serum IGF-1 in the normal range for age (between -1.00 SDs and +2.00 SDs).\n5. For participants currently on hGH treatment, historic results before GH treatment of stimulation test with serum or plasma GH level greater than 10 μg\u002FL or serum IGF-1 test between -1.00 SDs and +2.00 SDs.\n6. Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .","2 Years","16 Years",{"count":50,"type":21},300,"Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.",[53],"Idiopathic Short Stature","2026-06-18",{"date":56,"type":31},"2026-06-23",{"date":58,"type":31},"2024-08-08",{"date":60,"type":21},"2040-12-31",{"name":37,"class":38},38,{"id":64,"slug":65,"hasResults":11,"nctId":66,"briefTitle":67,"officialTitle":68,"acronym":4,"eligibilityCriteria":69,"healthyVolunteers":11,"sex":16,"minAge":18,"maxAge":70,"enrollmentInfo":71,"targetDuration":4,"studyType":73,"phases":74,"briefSummary":76,"conditions":77,"keywords":79,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":72},"100629670","phase-4-immune-modulation-during-palynziq-treatment-in-adults-impala-100629670","NCT07477691","Immune Modulation During Palynziq® Treatment in Adults (IMPALA)","A Phase 4 Study of Immune Modulation During Palynziq® Treatment in Adults With Phenylketonuria (PKU)","Inclusion Criteria:\n\n* Adults between 18 and 65 years old\n* Have a confirmed diagnosis of phenylketonuria (PKU)\n* Are in generally good health based on medical evaluation\n* Are willing and medically eligible to receive Palynziq and methotrexate (MTX) Cohort A: Have never taken Palynziq before and are willing to start it during the study Cohort B: Have blood \\> 600 μmol\u002FL after taking Palynziq for at least 24 weeks, are on a daily dose of at least 20mg and unable to increase the dose further\n* Agree to use required contraception if they or their partner could become pregnant\n* Are willing to carry two epinephrine devices at all times during Palynziq treatment\n\nExclusion Criteria:\n\n* Pregnant, breastfeeding, planning to become pregnant, planning to father a child, or not using effective birth control if applicable\n* Have a known severe allergy or hypersensitivity reaction to methotrexate (MTX), Palynziq, or other PEG-containing medications\n* Have a serious active infection or a history of severe or recurrent infections\n* Have significant medical conditions that may affect safety or participation (such as serious heart, lung, liver, kidney, immune, neurological, psychiatric, or cancer-related conditions)\n* Have a history of substance or alcohol abuse within the past 12 months\n* Have had an organ transplant or are taking chronic immunosuppressive medications\n* Are currently taking medications that are not allowed in the study, including other PKU treatments besides Palynziq\n* Are using, or plan to use, injectable PEG-containing medications other than Palynziq during the study\n* Have major surgery planned during the study participation period\n* Are currently participating in another clinical study involving Palynziq\n* In the opinion of the study doctor, are not a suitable candidate for the study or may have difficulty complying with study requirements","65 Years",{"count":72,"type":21},12,"INTERVENTIONAL",[75],"PHASE4","Study 165-401 is a Phase 4, open-label study designed to examine the concomitant use of methotrexate (MTX) to suppress immune responses to Palynziq and improve tolerability and efficacy in adults with PKU.",[78],"Phenylketonuria",[80,78,81,82,83,84,85,86,87,88,89,90],"PKU","Pegvaliase","Palynziq","Methotrexate","Immune Response","Safety","Immune Modulation","BMN 165-401","Hyperphenylalaninemia","Enzyme Substitution Therapy","Pharmacologic Immunosuppression","2026-06-16",{"date":93,"type":31},"2026-06-17",{"date":95,"type":31},"2026-05-29",{"date":97,"type":21},"2027-09-30",{"name":37,"class":38},{"id":100,"slug":101,"hasResults":11,"nctId":102,"briefTitle":103,"officialTitle":104,"acronym":105,"eligibilityCriteria":106,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":107,"enrollmentInfo":108,"targetDuration":4,"studyType":73,"phases":110,"briefSummary":113,"conditions":114,"keywords":115,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":125,"lastUpdatePostDateStruct":126,"startDateStruct":127,"completionDateStruct":129,"leadSponsor":131,"locationsCount":132},"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","17 Years",{"count":109,"type":21},160,[111,112],"PHASE2","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.",[26],[26,116,117,118,119,120,121,122,123,124],"ACH","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":95,"type":31},{"date":128,"type":21},"2026-04",{"date":130,"type":21},"2029-09",{"name":37,"class":38},10,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":4,"eligibilityCriteria":139,"healthyVolunteers":11,"sex":16,"minAge":140,"maxAge":141,"enrollmentInfo":142,"targetDuration":4,"studyType":73,"phases":144,"briefSummary":145,"conditions":146,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":148,"startDateStruct":150,"completionDateStruct":152,"leadSponsor":154,"locationsCount":155},"100545457","phase-2-a-phase-2-study-of-vosoritide-in-children-with-idiopathic-short-stature-100545457","NCT06382155","A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature","A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature","Key Inclusion Criteria:\n\n1. Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts\n2. If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg\u002FL or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs).\n\nKey Exclusions:\n\n1. Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency.\n2. Previous treatment with a growth promoting agent","3 Years","11 Years",{"count":143,"type":21},100,[111],"The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).",[53],"2026-05-20",{"date":149,"type":31},"2026-05-22",{"date":151,"type":31},"2024-10-21",{"date":153,"type":21},"2036-12",{"name":37,"class":38},47,{"id":157,"slug":158,"hasResults":11,"nctId":159,"briefTitle":160,"officialTitle":161,"acronym":4,"eligibilityCriteria":162,"healthyVolunteers":11,"sex":16,"minAge":140,"maxAge":141,"enrollmentInfo":163,"targetDuration":4,"studyType":73,"phases":165,"briefSummary":166,"conditions":167,"keywords":169,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":172,"lastUpdatePostDateStruct":173,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":180},"100567485","phase-2-a-study-of-vosoritide-in-children-with-noonan-syndrome-with-inadequate-growth-during-or-after-human-growth-hormone-treatment-100567485","NCT06668805","A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment","A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment","Inclusion Criteria:\n\n1. Participants must be ≥ 3 years old, and \\\u003C 11 years old (females) or \\\u003C 12 years old (males), at the time of signing the informed consent form\n2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.\n3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex.\n4. Tanner Stage 1, at time of signing the ICF.\n5. Previous or current hGH treatment for short stature associated with their condition.\n6. Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts\n\nExclusion Criteria:\n\n1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.\n2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.\n3. Bone age advanced beyond chronological age by more than 2 years.\n4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,\n5. Have an unstable condition likely to require surgical intervention during the study.\n6. Evidence of decreased growth velocity (AGV \\\u003C 1.5 cm\u002Fyear) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays.\n7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.\n8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.",{"count":164,"type":21},30,[111],"The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.",[168],"Noonan Syndrome",[170,120,118,171],"Short Stature","Developmental Endocrine System Diseases Natriuretic Peptide, C-Type","2026-05-07",{"date":174,"type":31},"2026-05-11",{"date":176,"type":31},"2024-11-22",{"date":178,"type":21},"2041-09",{"name":37,"class":38},36,{"id":182,"slug":183,"hasResults":11,"nctId":184,"briefTitle":185,"officialTitle":186,"acronym":4,"eligibilityCriteria":187,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":188,"enrollmentInfo":189,"targetDuration":4,"studyType":73,"phases":191,"briefSummary":192,"conditions":193,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":195,"lastUpdatePostDateStruct":196,"startDateStruct":198,"completionDateStruct":200,"leadSponsor":202,"locationsCount":203},"100602649","phase-2-a-study-of-vosoritide-versus-placebo-in-children-with-hypochondroplasia-aged-0-to--36-months-100602649","NCT07126262","A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to \u003C 36 Months","A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to \u003C 36 Months","Key Inclusion Criteria:\n\n1. Participants must be 0 to \\\u003C 36 months of age at randomization.\n2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).\n3. Participants aged 0 to \\\u003C 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \\\u003C 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.\n4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.\n\nKey Exclusion Criteria:\n\n1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).\n2. Have an unstable medical condition likely to require surgical intervention during the study period.\n3. Taking any of the prohibited medications.\n4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\\> 3 months) at any time.\n5. Require any investigational agent prior to completion of study period.\n6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.\n7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.\n8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.\n9. Have known hypersensitivity to vosoritide or its excipients.\n10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.\n11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.","36 Months",{"count":190,"type":21},60,[111],"The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \\\u003C 36 months over a 52-week period.",[194],"Hypochondroplasia","2026-03-26",{"date":197,"type":31},"2026-03-31",{"date":199,"type":31},"2025-07-30",{"date":201,"type":21},"2028-06-30",{"name":37,"class":38},26,{"id":205,"slug":206,"hasResults":11,"nctId":207,"briefTitle":208,"officialTitle":209,"acronym":210,"eligibilityCriteria":211,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":212,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":214,"conditions":215,"keywords":217,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":222,"startDateStruct":224,"completionDateStruct":226,"leadSponsor":228,"locationsCount":203},"100501778","a-long-term-post-marketing-safety-study-of-palynziq-in-patients-with-pku-palace-100501778","NCT05813678","A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)","A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria","PALace","Inclusion Criteria:\n\n* Documented diagnosis of PKU per local standard of care\n* Currently receiving or planned to receive pegvaliase treatment within 30 days after the date of enrollment, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation.\n* Subject (or legally authorized representative) is willing and able to provide written informed consent after the nature of the study has been explained and prior to any data collection.\n\nExclusion Criteria:\n\n* Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and\u002For comply with any aspect of the study.\n* Currently participating in an interventional study of any investigational product, device, or procedure\n* Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)\n* German subjects \\\u003C16 years if age",{"count":213,"type":21},450,"This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union (EU) only) in subjects receiving pegvaliase for the treatment of PKU. Subjects for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment (incident-users) or have previously started treatment with pegvaliase at the date of enrollment (prevalent-users) are eligible for participation in this study.",[216],"Phenylketonuria (PKU)",[218,219,81,82,80,78,220],"Observational","Safety Study","Phase 4","2026-03-23",{"date":223,"type":31},"2026-03-25",{"date":225,"type":31},"2022-06-06",{"date":227,"type":21},"2033-11-01",{"name":37,"class":38},{"id":230,"slug":231,"hasResults":11,"nctId":232,"briefTitle":233,"officialTitle":234,"acronym":4,"eligibilityCriteria":235,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":236,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":238,"conditions":239,"keywords":240,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":246,"lastUpdatePostDateStruct":247,"startDateStruct":249,"completionDateStruct":251,"leadSponsor":253,"locationsCount":5},"100539545","a-long-term-post-marketing-study-of-immune-response-in-patients-receiving-palynziq-treatment-for-pku-palisade-100539545","NCT06305234","A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)","A Multi-Center, Prospective, Longitudinal, Study Evaluating Immunologic, Inflammatory, and Laboratory Parameters Associated With Long-Term Palynziq® (Pegvaliase) Treatment in Subjects With Phenylketonuria (PKU) in the United States","Inclusion Criteria:\n\n* Subjects enrolled at US sites participating in the 165-501 study.\n\nExclusion Criteria:\n\n* Legal incapacity or limited legal capacity without legal guardian representation.\n* Subject is unable or unwilling to provide informed consent for the additional interventional burden of the study (blood sampling).",{"count":237,"type":21},200,"This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503.",[216],[80,78,82,241,242,243,244,245],"pegvaliase","observational","safety study","immunogenicity assessment","inflammatory assessment","2026-03-20",{"date":248,"type":31},"2026-03-24",{"date":250,"type":31},"2024-01-20",{"date":252,"type":21},"2033-11-18",{"name":37,"class":38},{"id":255,"slug":256,"hasResults":11,"nctId":257,"briefTitle":258,"officialTitle":258,"acronym":259,"eligibilityCriteria":260,"healthyVolunteers":11,"sex":261,"minAge":4,"maxAge":4,"enrollmentInfo":262,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":264,"conditions":265,"keywords":267,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":246,"lastUpdatePostDateStruct":272,"startDateStruct":273,"completionDateStruct":275,"leadSponsor":277,"locationsCount":278},"100483785","a-global-multicenter-study-to-assess-maternal-fetal-and-infant-outcomes-of-exposure-to-palynziq-pegvaliase-during-pregnancy-and-breastfeeding-100483785","NCT05579548","A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding","PALomino","Inclusion Criteria:\n\n* Subject (or a legally authorized representative) consent obtained prior to enrollment. Consent will be obtained in compliance with any country-specific regulations or requirements\n* Confirmation of ongoing pregnancy. Subjects with unknown outcomes will be classified as prospective pregnancies; subjects who have undergone prenatal testing (eg, targeted ultrasound, amniocentesis) regardless of findings will be classified as retrospective pregnancies\n* Diagnosed with PKU per local standard of care\n* Documentation that the subject was treated with pegvaliase at any point starting from 2 weeks prior to the date of LMP\n* Agrees to permit the Investigator (ie, CRP, CCA, PI) to contact the subject's HCPs (eg, PCP, PKU-treating physician, OB, nurse, midwife) and the infant's HCP (eg, pediatrician, neonatologist) for medical information\n\nExclusion Criteria:\n\n• Currently participating in a BioMarin sponsored interventional study of any investigational product, device, or procedure","FEMALE",{"count":263,"type":21},50,"This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding.",[266],"Phenylketonuria, Maternal",[220,218,268,269,270,271],"Maternal","Pregnancy Outcomes","Exposure","Breastfeeding Outcomes",{"date":248,"type":31},{"date":274,"type":31},"2022-11-22",{"date":276,"type":21},"2032-10-04",{"name":37,"class":38},4,{"id":280,"slug":281,"hasResults":11,"nctId":282,"briefTitle":283,"officialTitle":283,"acronym":4,"eligibilityCriteria":284,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":285,"enrollmentInfo":286,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":288,"conditions":289,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":290,"lastUpdatePostDateStruct":291,"startDateStruct":293,"completionDateStruct":295,"leadSponsor":297,"locationsCount":298},"100532449","a-multicenter-multinational-observational-study-of-children-with-hypochondroplasia-100532449","NCT06212947","A Multicenter Multinational Observational Study of Children With Hypochondroplasia","Inclusion Criteria:\n\n* Participants must be ≤ 15 years old at the time of signing the informed consent\n* Participants must have genetic confirmation of Hypochondroplasia diagnosis\n\nExclusion Criteria:\n\n* Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature\n* Received an investigational product or medical device within 6 months before the Screening visit","15 Years",{"count":287,"type":21},400,"This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.",[194],"2025-04-01",{"date":292,"type":31},"2025-04-03",{"date":294,"type":31},"2023-11-27",{"date":296,"type":21},"2043-12-31",{"name":37,"class":38},43,""]