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
This trial does not accept healthy volunteersQualifiers
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 population
Children with Achondroplasia
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
No Interventions
Other interventionNo Interventions
complete a natural history observation of ACH for at least 6 months and up to 2 years
Other interventionno interventions
Treatment groups
260 Participants
are divided into 1 treatment groupGroup A: Children with Achondroplasia2 interventions
Trial outcomes
Primary outcomes
1
Annualized growth velocity (AGV)
Annualized growth velocity (AGV)
Time frame
Through the study completion, an average of three months, up to 2 yearsSecondary outcomes
1
standing height
calculated to the nearest 0.1 cm
Time frame
an average of three months, up to 2 years2
sitting height
calculated to the nearest 0.1 cm
Time frame
an average of three months, up to 2 years3
sitting height to standing height ratio
This parameter is calculated as the ratio of sitting height to total standing height. It is used to assess the abnormality in body proportions, specifically the relative trunk-to-lower limb length. In patients with achondroplasia, this ratio is typically increased. Within the clinical trial, this measure is used to evaluate the treatment drug's effect on body proportions.
Time frame
an average of three months, up to 2 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov