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

NCT06067425 | Terminated Phase 2 Results DMC FDA Drug

• 3 other identifiers: DRI16646U1111-1280-53742023-503677-37
• Study Type: interventional
• Target: 16
• Locations: 5 countries
• Sites: 9

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.

Conditions

Osteochondrodysplasia

Outcome Measures

Primary Outcomes (1)

• Stage 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)

  An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. An SAE was defined as any untoward medical occurrence (whether considered to be related to study drug or not) that at any dose: resulted in death; or was life-threatening; or required inpatient hospitalization or prolongation of existing hospitalization; or resulted in persistent or significant disability/incapacity; or was a congenital anomaly/birth defect; or was an important medical event. An AESI was an AE (serious or nonserious) of scientific and medical concern specific to the Sponsor's product or program, for which ongoing monitoring and immediate notification by the Investigator to the Sponsor was required. The TEAEs were defined as events that were newly reported or reported to worsen in severity after the start of study drug up to 5 days post last dose of the study drug.

  From the first dose of study drug administration (Day 1) up to early termination of the study, approximately 58 weeks (Stage 1: SAR442501 Dose A) and 46 weeks (Stage 1: SAR442501 Dose B)

Secondary Outcomes (30)

• Stage 1: Change From Baseline to Weeks 26 and 52 in Growth Velocity Z-Score

  Baseline (Day 1) and Weeks 26 and 52

• Stage 1: Change From Baseline to Weeks 26 and 52 in Annualized Growth Velocity

  Baseline (Day 1) and Weeks 26 and 52

• Stage 1: Change From Baseline to Weeks 26 and 52 in Height Z-Score

  Baseline (Day 1) and Weeks 26 and 52

• Stage 1: Change From Baseline to Weeks 26 and 52 in Upper to Lower Body Segment Ratio

  Baseline (Day 1) and Weeks 26 and 52

• Stage 1: Change From Baseline to Weeks 26 and 52 in Upper to Lower Extremity Ratio

  Baseline (Day 1) and Weeks 26 and 52

Study Arms (3)

Cohort 1

EXPERIMENTAL

Drug: SAR442501

Cohort 2

EXPERIMENTAL

Drug: SAR442501

Cohort 3

EXPERIMENTAL

Drug: SAR442501

Interventions

SAR442501 DRUG

Solution for injection; Subcutaneous injection

Cohort 1; Cohort 2; Cohort 3

Eligibility Criteria

• Age: 0 Days - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (9)

Investigational Site Number : 0360001

Parkville, Victoria, 3052, Australia

Location

Investigational Site Number : 1560002

Shanghai, 200120, China

Location

Investigational Site Number : 1560001

Wuhan, 430030, China

Location

Investigational Site Number : 3800002

Milan, Lombardy, 20122, Italy

Location

Investigational Site Number : 3800001

Rome, Roma, 00168, Italy

Location

Investigational Site Number : 4100001

Seoul, Seoul-teukbyeolsi, 03080, South Korea

Location

Investigational Site Number : 4100002

Seoul, Seoul-teukbyeolsi, 06351, South Korea

Location

Investigational Site Number : 7240002

Vitoria-Gasteiz, Basque Country, 01008, Spain

Location

Investigational Site Number : 7240001

Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain

Location

Related Links

• DRI16646 Plain Language Results Summary

MeSH Terms

Conditions

Osteochondrodysplasias

Condition Hierarchy (Ancestors)

Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

The study was early terminated per Sponsor decision, for reasons not related to safety concerns.

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi aventis recherche & développement

Contact-US@sanofi.com

800-633-1610

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 20, 2023
• First Posted: October 4, 2023
• Study Start: October 10, 2023
• Primary Completion: February 12, 2025
• Study Completion: February 12, 2025
• Last Updated: June 25, 2026
• Results First Posted: June 25, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

KR (2); AU (1); CN (2); IT (2); ES (2)