Overview
This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.
Description
Up to approximately 275 weeks: 3 weeks Screening + 52 weeks primary treatment period + up to approximately 216 weeks extended treatment period+ 4 weeks follow-up.
Eligibility
Inclusion Criteria:
- Participants must have ACH with a confirmed mutation in the FGFR3 gene
- Participants and/or parent(s) or legal representative(s) must be willing and able to perform all the study procedures to the best of their physical ability.
- Parent(s) or legal representative(s) capable of giving signed informed consent and participants capable of giving assent when applicable.
Exclusion Criteria:
- Have hypochondroplasia (or the N540K mutation) or short stature condition other than ACH (eg, trisomy 21, pseudochondroplasia)
- Participants have received any dose of medications or investigational product, including human growth hormone, IGF-1, intended to affect participants' stature or body proportions between the completion of OBS16647 and enrollment (Week 0/Day 1/Visit 2).
- Have a history of growth plate closure.
- Long bone fracture within 3 months of enrollment (Week 0/Day 1/Visit 2)
- Current evidence of corneal or retinal disorder/keratopathy.
- Participants have had a previous surgical intervention involving the foramen magnum (Stage 2 only).
- Hyperphosphatemia.
The above information is not intended to contain all considerations relevant to a potential
participation in a clinical trial.