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

NCT ID: NCT06067425

Last Updated: 2025-02-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-10-10
• Study Completion Date: 2025-02-12

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.

Conditions

Osteochondrodysplasia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1

Group Type: EXPERIMENTAL

SAR442501

Intervention Type: DRUG

Solution for injection; Subcutaneous injection

Cohort 2

Group Type: EXPERIMENTAL

SAR442501

Intervention Type: DRUG

Solution for injection; Subcutaneous injection

Cohort 3

Group Type: EXPERIMENTAL

SAR442501

Intervention Type: DRUG

Solution for injection; Subcutaneous injection

Interventions

SAR442501

Solution for injection; Subcutaneous injection

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 0 Days
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Investigational Site Number : 0360001

Parkville, Victoria, Australia

Investigational Site Number : 1560002

Shanghai, , China

Investigational Site Number : 1560001

Wuhan, , China

Investigational Site Number : 3800002

Milan, Lombardy, Italy

Investigational Site Number : 3800001

Rome, Roma, Italy

Investigational Site Number : 4100001

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 4100002

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 7240002

Vitoria-Gasteiz, Basque Country, Spain

Investigational Site Number : 7240001

Esplugues de Llobregat, Catalunya [Cataluña], Spain

Countries

Australia; China; Italy; South Korea; Spain

Other Identifiers

U1111-1280-5374

Identifier Type: REGISTRY

Identifier Source: secondary_id

2023-503677-37

Identifier Type: REGISTRY

Identifier Source: secondary_id

DRI16646

Identifier Type: -

Identifier Source: org_study_id