Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia

• Status: Recruiting
• Trial end date: 2027-12-02
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sanofi

Criteria

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.