Actively Recruiting
A Study in Children With Achondroplasia
Led by Abbisko Therapeutics Co, Ltd · Updated on 2025-12-24
260
Participants Needed
8
Research Sites
723 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study in Children With Achondroplasia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Guardians and children with ACH must be willing and able to provide signed informed consent prior to screening
- Clinical diagnosis of ACH 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 14 years or older based on hand and wrist X-ray within 6 months prior to Day 1
- Evidence of growth plate closure in proximal tibia or distal femur, or annualized growth velocity 1.5 cm/year or less over at least 6 months prior to screening
- Skeletal dysplasia other than ACH or medical conditions causing short stature or abnormal growth, including severe achondroplasia with developmental delay and acanthosis nigricans, Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, chronic renal insufficiency, active celiac disease (except if responsive to gluten-free diet), vitamin D deficiency or insufficiency not corrected by supplementation, untreated hypothyroidism, poorly controlled diabetes, or diabetic complications
- History or presence of injury or disease affecting growth plates other than ACH
- Impaired cardiac function or significant cardiovascular disease, including NYHA class II or higher, certain congenital heart diseases, significant arrhythmias, aortic regurgitation, congestive heart failure, or other uncontrolled heart disease
- Current severe sleep apnea, symptomatic or requiring intervention for hydrocephalus, spinal cord compression at cranio-cervical junction, or previous 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 like human growth hormone, IGF-1, or anabolic steroids within 3 months prior to screening or long-term use exceeding 3 months
- Prior treatment with CNP analogues, FGFR inhibitors, or investigational drugs or devices affecting stature or body proportionality
- Any comorbidities or conditions likely to prevent full study participation or protocol compliance as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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