DRI16646_upreACH-2
Research type
Research Study
Full title
A Phase 2, open-label, multi-center, 2-stage sequential cohort, dose escalation study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of subcutaneous SAR442501 in pediatric participants with Achondroplasia
IRAS ID
1008724
Contact name
Patrick Maury
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Eudract number
2023-503677-37
Clinicaltrials.gov Identifier
Research summary
Achondroplasia is a genetic condition that people are born with. In most patients, it is caused by a mutation (change) in a gene called “FGFR3.” The mutation affects bone growth of the skull, spine, arms, and legs, resulting in short stature, difficulty walking, and often frequent ear infections and back pain. More serious effects may include enlarged tonsils, breathing problems while sleeping, called “sleep apnoea,” or enlarged ventricles in the brain, all of which may require surgery.
The main objective of the study is to assess the side effects of the study medicine, SAR442501. SAR442501 is being developed as a possible treatment for achondroplasia by blocking the activity of FGFR3. Treatment is provided as an injection, given under the skin.
This study has 2 stages, and it is open-label, so participants and researchers both know which treatment is given. Children are divided into groups based on their age and each group receives 1 of 2 doses of SAR442501 for at least 52 weeks in the primary treatment period, and at least 52 weeks in the extended treatment period.
Children with achondroplasia aged birth to less than 12 years, with a confirmed mutation in the FGFR3 gene, can participate is the study. Children with certain medical problems, or anything else that makes them unsuitable for the study, cannot participate.
Participants will be required to attend 4 study visits in the clinic over the first 3 months, and then approximately every 3 months until study completion. During the study, participants will have certain procedures and tests, including: physical exams, assessment of vital signs, blood tests, ECG tests, growth measurements, x-rays, etc.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
24/WM/0053
Date of REC Opinion
2 Jul 2024
REC opinion
Further Information Favourable Opinion