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

Investigating the Safety and Effects of an Investigational Medication for Children with a Genetic Bone Condition

Not Recruiting
12 years or below
All
Phase 2
16 participants needed

Study 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.

Study Details

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 Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Osteochondrodysplasia
  • Age: 12 years or below
  • Gender: All

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.

Updated on 04 Apr 2025. Study ID: NCT06067425

This study investigates the safety and efficacy of an investigational medication in children with Achondroplasia, a genetic condition that affects bone growth and causes short stature. Achondroplasia is caused by a mutation in the FGFR3 gene, which affects the development of cartilage into bone, particularly in the long bones of the arms and legs.

Participants in the study will receive the investigational medication and undergo various study procedures to assess its effects. The study will include regular monitoring and assessments to evaluate the safety and tolerability of the treatment.

  • Who can participate: Children from birth up to 12 years of age with Achondroplasia and a confirmed mutation in the FGFR3 gene can participate. Guardians must provide informed consent.
  • Study details: Participants will take part in the study by receiving the investigational medication. They will be monitored for safety and effects throughout the study.
  • Study Timelines: The study will last 275 weeks.