Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
260 participants
OBSERVATIONAL
2025-06-20
2039-04-30
Brief Summary
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Detailed Description
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Secondary Objectives To characterize the pharmacokinetics (PK) of ABSK061 and potential disproportional metabolites (if applicable) To evaluate changes from baseline in anthropometric parameters after administration of oral ABSK061 To evaluate the acceptability of ABSK061 minitablets for peroral administration in children with ACH
Exploratory Objectives To evaluate changes in ACH complications and disease burden after oral administration of ABSK061 To evaluate the pharmacodynamic (PD) profile in children with ACH after oral administration of ABSK061
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Children with Achondroplasia
Male or female aged ≥2.5 to \<11 years old at screening
No Interventions
No Interventions
complete a natural history observation of ACH for at least 6 months and up to 2 years
no interventions
Interventions
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No Interventions
No Interventions
complete a natural history observation of ACH for at least 6 months and up to 2 years
no interventions
Eligibility Criteria
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Inclusion Criteria
2. Clinical diagnosis of ACH confirmed FGFR3 mutation by genetic testing.
3. Male or female aged ≥2.5 to \<11 years old at screening.
4. Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening.
5. Ambulatory and able to stand without assistance.
Exclusion Criteria
2. Current evidence of growth plate closure (proximal tibia, distal femur), or AGV ≤ 1.5 cm/year over a period ≥6 months prior to screening.
3. 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.
1. Celiac disease responsive to a gluten-free diet is allowed
2. Vitamin D deficiency or insufficiency with a 25-hydroxyvitamin D \[25- (OH) D\] level ≥ 30 nmol/L after supplementation is allowed. Vitamin D deficiency is defined as 25-(OH) D level \<30 nmol/L. Vitamin D insufficiency is defined as 25-(OH) D level 30\~50 nmol/L. Patients with Vitamin D deficiency or insufficiency must be on Vitamin D regimen prior to screening
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
4. Patients with diabetes must have been on stable medication regimen for 3 months prior to screening
4. History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.
5. 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/ventricular septal defect with repair are allowed), clinically significant arrhythmias requiring therapy, aortic regurgitation, congestive heart failure, or any other uncontrolled heart disease.
6. For ACH-related complications: Current severe sleep apnea, symptomatic and/or requiring intervention for hydrocephalus, or spinal cord compression at the cranio-cervical junction, and has previously undergone ventriculoperitoneal shunt surgery.
7. Bone fracture within 6 months prior to screening (within 2 months for finger and toe fractures).
8. 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.
9. 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.
10. 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
ALL
No
Sponsors
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Abbisko Therapeutics Co, Ltd
INDUSTRY
Responsible Party
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Principal Investigators
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Yuan Lu
Role: STUDY_DIRECTOR
12B, Building 1, No 515, Huanke Road, Pudong New Area, Shanghai 201210, China
Locations
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Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Guangzhou Women and Childrens Medical Center
Guangzhou, Guangzhou, China
Henan Children's Hospital, Zhengzhou Children's Hospital
Zhengzhou, Henan, China
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, China
Xin Hua Hospital Affiliatod to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, China
West China Second University Hospital, Sichuan University
Chengdu, Sichuan, China
Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Countries
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Central Contacts
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Facility Contacts
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Di Wu
Role: primary
Li Liu
Role: primary
Haiyan Wei
Role: primary
Yanqin Ying
Role: primary
Yongguo Yu
Role: primary
Xinran Cheng
Role: primary
Wei Wu
Role: primary
Other Identifiers
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ABSK061-001
Identifier Type: -
Identifier Source: org_study_id