[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100615843":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":25,"locations":31,"responsibleParty":117,"collaborators":19,"id":119,"slug":120,"hasResults":121,"nctId":122,"briefTitle":123,"officialTitle":124,"acronym":19,"eligibilityCriteria":125,"healthyVolunteers":121,"sex":126,"minAge":127,"maxAge":128,"enrollmentInfo":129,"targetDuration":19,"studyType":132,"phases":133,"briefSummary":136,"conditions":137,"keywords":19,"overallStatus":139,"whyStopped":19,"lastUpdateSubmitDate":140,"lastUpdatePostDateStruct":141,"startDateStruct":144,"completionDateStruct":146,"leadSponsor":148,"locationsCount":149},{"fullName":5,"class":6},"Abbisko Therapeutics Co, Ltd","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"ABSK061","EXPERIMENTAL","Dose escalation part will be divided into Part A and Part B according to patient age, with Part A enrolling children with ACH aged 6 to \\\u003C 12 years (inclusive of 6 years) and Part B enrolling children with ACH aged 3 to \\\u003C 6 years (inclusive of 3 years). In this study, two sentinel patients will be set for both parts of each dose level, thereby the interval for the first dose between the first 2 patients and the third patient is not less than 28 days.\n\nThis study will employ the Bayesian optimal interval (BOIN) design for dose escalation part to guide dose escalation. The target toxicity rate for the MTD is φ = 0.25 and the maximum sample size for the dose escalation part will be 80 (50 in Part A and 30 in Part B). The DLT observation period is defined as the first 28 days after administration of ABSK061.\n\nAfter the RDE is confirmed in the dose Escalation part, the dose Expansion phase will be conducted.",[13],"Drug: ABSK061",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","ABSK061 is supplied as minitablets filled in capsules. Four strengths of capsules will be provided: 0.2 mg, 2 mg, 3 mg, and 5 mg. Each patient can only be administered with a single strength.\n\nAll patients will be administered orally once daily under the fasted state, i.e., fast from 2 hours before dosing to 1 hour after dosing. Dose will be calculated based on the patient's weight, and detailed rules for dose calculation are provided.\n\nTake out the ABSK061 capsule containing minitablets, take off the capsule cap and decant the content (minitablets), swallow the minitablets completely with water without chewing (the recommended water volume is 50-180 mL). If there is difficulty swallowing all the minitablets at once, they may be taken in several portions as needed, but the entire dose should be taken within 10 minutes. If needed, yogurt or apple sauce can be used as vehicles to facilitate swallowing of the minitablets.",[9],null,[21],{"name":22,"affiliation":23,"role":24},"Shuqi Zeng","12B, Building 1, No 515, Huanke Road, Pudong New Area, Shanghai 201210, China","STUDY_DIRECTOR",[26],{"name":27,"role":28,"phone":29,"phoneExt":19,"email":30},"Jing Zhang","CONTACT","+86-15002126439","jing.zhang@abbisko.com",[32,50,59,75,91,95,108],{"facility":33,"status":19,"city":34,"state":35,"zip":19,"country":36,"countryCode":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":44},"Beijing Children's Hospital, Capital Medical University","Beijing","Beijing Municipality","China","CN",{"type":39,"coordinates":40},"Point",[41,42],116.39723,39.9075,{"lat":42,"lon":41},[45,48],{"name":46,"role":28,"phone":19,"phoneExt":19,"email":47},"Di Wu","woody0928@sina.com",{"name":46,"role":49,"phone":19,"phoneExt":19,"email":19},"PRINCIPAL_INVESTIGATOR",{"facility":51,"status":19,"city":52,"state":53,"zip":19,"country":36,"countryCode":37,"cosmosGeoPoint":54,"geoPoint":58,"contacts":19},"Henan Children's Hospital, Zhengzhou Children's Hospital","Zhengzhou","Henan",{"type":39,"coordinates":55},[56,57],113.64861,34.75778,{"lat":57,"lon":56},{"facility":60,"status":19,"city":61,"state":62,"zip":19,"country":36,"countryCode":37,"cosmosGeoPoint":63,"geoPoint":67,"contacts":68},"Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology","Wuhan","Hubei",{"type":39,"coordinates":64},[65,66],114.26667,30.58333,{"lat":66,"lon":65},[69,72,73],{"name":70,"role":28,"phone":19,"phoneExt":19,"email":71},"Yanqin Ying","yingtea0909@126.com",{"name":70,"role":49,"phone":19,"phoneExt":19,"email":19},{"name":74,"role":49,"phone":19,"phoneExt":19,"email":19},"Xiuhua Ren",{"facility":76,"status":19,"city":77,"state":78,"zip":19,"country":36,"countryCode":37,"cosmosGeoPoint":79,"geoPoint":83,"contacts":84},"Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine","Shanghai","Shanghai Municipality",{"type":39,"coordinates":80},[81,82],121.45806,31.22222,{"lat":82,"lon":81},[85,88,90],{"name":86,"role":28,"phone":19,"phoneExt":19,"email":87},"Yongguo Yu","yuyongguo@xinhuamed.com.cn",{"name":86,"role":28,"phone":19,"phoneExt":19,"email":89},"yuyongguo@shsmu.edu.cn",{"name":86,"role":49,"phone":19,"phoneExt":19,"email":19},{"facility":92,"status":19,"city":93,"state":94,"zip":19,"country":36,"countryCode":37,"cosmosGeoPoint":19,"geoPoint":19,"contacts":19},"Chengdu Women's and Children's Central Hospital","Chengde","Sichuan",{"facility":96,"status":19,"city":97,"state":94,"zip":19,"country":36,"countryCode":37,"cosmosGeoPoint":98,"geoPoint":102,"contacts":103},"West China Second University Hospital, Sichuan University","Chengdu",{"type":39,"coordinates":99},[100,101],104.06667,30.66667,{"lat":101,"lon":100},[104,107],{"name":105,"role":28,"phone":19,"phoneExt":19,"email":106},"Jin Wu","wangdo620@163.com",{"name":105,"role":49,"phone":19,"phoneExt":19,"email":19},{"facility":109,"status":19,"city":110,"state":111,"zip":19,"country":36,"countryCode":37,"cosmosGeoPoint":112,"geoPoint":116,"contacts":19},"Children's Hospital Zhejiang University School of Medicine","Hangzhou","Zhejiang",{"type":39,"coordinates":113},[114,115],120.16142,30.29365,{"lat":115,"lon":114},{"type":118,"investigatorFullName":19,"investigatorTitle":19,"investigatorAffiliation":19,"oldNameTitle":19,"oldOrganization":19},"SPONSOR","100615843","phase-1-a-study-of-absk061-to-assess-safety-tolerability-pharmacokinetics-and-efficacy-in-children-with-achondroplasia-100615843",false,"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).","ALL","3 Years","12 Years",{"count":130,"type":131},110,"ESTIMATED","INTERVENTIONAL",[134,135],"PHASE1","PHASE2","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.",[138],"Achondroplasia","NOT_YET_RECRUITING","2025-12-17",{"date":142,"type":143},"2025-12-22","ACTUAL",{"date":145,"type":131},"2025-12-10",{"date":147,"type":131},"2031-03-15",{"name":5,"class":6},7]