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Status:

TERMINATED

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

Lead Sponsor:

Sanofi

Conditions:

Osteochondrodysplasia

Eligibility:

All Genders

Up to 12 years

Phase:

PHASE2

Brief 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.

Detailed 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 Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

October 10 2023

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

February 12 2025

Estimated Enrollment :

16 Patients enrolled

Trial Details

Trial ID

NCT06067425

Start Date

October 10 2023

End Date

February 12 2025

Last Update

February 25 2025

Active Locations (9)

Enter a location and click search to find clinical trials sorted by distance.

Page 1 of 3 (9 locations)

1

Investigational Site Number : 0360001

Parkville, Victoria, Australia, 3052

2

Investigational Site Number : 1560002

Shanghai, China, 200120

3

Investigational Site Number : 1560001

Wuhan, China, 430030

4

Investigational Site Number : 3800002

Milan, Lombardy, Italy, 20122