A Study in Children With Achondroplasia
ConditionAchondroplasia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age30-11
SponsorAbbisko Therapeutics Co, Ltd
About this trial
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.
Eligibility criteria
Qualifiers
Prior to screening, the guardians and children with ACH (if applicable) must be willing and able to provide signed informed consent.
Clinical diagnosis of ACH confirmed FGFR3 mutation by genetic testing.
Male or female aged ≥2.5 to <11 years old at screening.
Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening.
Disqualifiers
Bone age ≥14 years as assessed by the investigator based on hand and wrist X-ray taken within 6 months prior to Day 1.
Current evidence of growth plate closure (proximal tibia, distal femur), or AGV ≤ 1.5 cm/year over a period ≥6 months prior to screening.
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.
Celiac disease responsive to a gluten-free diet is allowed
Trial design
Treatments tested in this trial
- No Interventions
- complete a natural history observation of ACH for at least 6 months and up to 2 years
Treatment groups
260 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov