Actively Recruiting
A Multicenter, Longitudinal, Observational Study in Children With Achondroplasia
Led by Abbisko Therapeutics Co, Ltd · Updated on 2025-12-24
260
Participants Needed
8
Research Sites
539 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting an observational study to gather detailed information about children with achondroplasia (ACH), a form of dwarfism. The study aims to collect data on body measurements, clinical features, related medical conditions, quality of life, and treatments over a period ranging from 6 months to 2 years. The study also evaluates the safety, tolerability, and appropriate dosing of an oral medication called ABSK061, as well as its effects on growth and disease burden in children with ACH. Participants aged 2.5 to under 11 years old, who have a confirmed diagnosis of ACH through genetic testing, will be observed without any interventions. The study will monitor changes in growth parameters such as annualized growth velocity, standing height, sitting height, and their ratios. Additionally, researchers will assess how well children accept the ABSK061 minitablets taken orally. The study includes safety evaluations and pharmacokinetic and pharmacodynamic assessments of the medication. During the study, children's growth and health will be regularly measured every three months for up to two years. Assessments include physical measurements, clinical evaluations, and monitoring for ACH complications. Researchers will observe how the disease progresses naturally and how the oral medication ABSK061 influences growth and disease-related factors. The study ensures ongoing safety monitoring and allows participants to be ambulatory and capable of standing without assistance throughout the observation period.
CONDITIONS
Brief Title
A Study in Children With Achondroplasia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Guardians and children must be willing and able to provide signed informed consent before screening.
- Clinical diagnosis of achondroplasia confirmed by FGFR3 mutation genetic testing.
- Male or female aged 2.5 months to under 11 years at screening.
- Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening.
- Ambulatory and able to stand without assistance.
You will not qualify if you...
- Bone age of 14 years or older based on hand and wrist X-ray within 6 months prior to Day 1.
- Evidence of growth plate closure or annualized growth velocity of 1.5 cm/year or less over 6 months before screening.
- Skeletal dysplasia other than achondroplasia or medical conditions causing short stature or abnormal growth including severe achondroplasia with developmental delay, Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, chronic renal insufficiency, active celiac disease, vitamin D deficiency, untreated hypothyroidism, poorly controlled diabetes, or diabetic complications.
- History or presence of injury or disease affecting growth potential of long bones other than achondroplasia.
- Impaired cardiac function or clinically significant cardiovascular disease such as heart failure or arrhythmias requiring therapy.
- Severe sleep apnea, symptomatic hydrocephalus requiring intervention, spinal cord compression at cranio-cervical junction, or prior ventriculoperitoneal shunt surgery.
- Bone fracture within 6 months prior to screening (2 months for finger and toe fractures).
- Use of medications affecting stature or body proportionality within 3 months before screening or long-term treatment with such drugs.
- Prior treatment with CNP analogues, FGFR inhibitors, or investigational drugs/devices affecting stature.
- Any comorbidities or conditions that may impair study compliance or completion as judged by the investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 6 months and up to 2 years
Participants are observed over time to complete a natural history observation of achondroplasia, including assessments of growth and other health parameters.
Visits approximately every 3 months for assessments
Trial Site Locations
Total: 8 locations
1
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Actively Recruiting
2
Guangzhou Women and Childrens Medical Center
Guangzhou, Guangzhou, China
Not Yet Recruiting
3
Henan Children's Hospital, Zhengzhou Children's Hospital
Zhengzhou, Henan, China
Not Yet Recruiting
4
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, China
Actively Recruiting
5
Xin Hua Hospital Affiliatod to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
Actively Recruiting
6
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, China
Not Yet Recruiting
7
West China Second University Hospital, Sichuan University
Chengdu, Sichuan, China
Active, Not Recruiting
8
Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Not Yet Recruiting
Research Team
J
Jing Zhang
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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