NCT06067425

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.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2023

Shorter than P25 for phase_2

Geographic Reach
5 countries

9 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 20, 2023

Completed
14 days until next milestone

First Posted

Study publicly available on registry

October 4, 2023

Completed
6 days until next milestone

Study Start

First participant enrolled

October 10, 2023

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 12, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 12, 2025

Completed
Last Updated

February 25, 2025

Status Verified

February 1, 2025

Enrollment Period

1.3 years

First QC Date

September 20, 2023

Last Update Submit

February 21, 2025

Conditions

Osteochondrodysplasia

Outcome Measures

Primary Outcomes (1)

  • Number of participants with adverse events (AE), serious adverse events (SAE), and adverse events of special interest (AESI) during the treatment-emergent period

    Baseline to Week 52

Secondary Outcomes (31)

  • Change in annualized growth velocity (AGV) Zscore

    Baseline to Week 26 and Week 52

  • Change in AGV (cm/year)

    Baseline to Week 26 and Week 52

  • Change in height Z score

    Baseline to Week 26 and Week 52

  • Change in upper-to-lower body segment ratio

    Baseline to Week 26 and Week 52

  • Change in upper to lower extremity ratio

    Baseline to Week 26 and Week 52

  • +26 more secondary outcomes

Study Arms (3)

Cohort 1

EXPERIMENTAL
Drug: SAR442501

Cohort 2

EXPERIMENTAL
Drug: SAR442501

Cohort 3

EXPERIMENTAL
Drug: SAR442501

Interventions

SAR442501DRUG

Solution for injection; Subcutaneous injection

Cohort 1Cohort 2Cohort 3

Eligibility Criteria

Age0 Days - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

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

MeSH Terms

Conditions

Osteochondrodysplasias

Condition Hierarchy (Ancestors)

Bone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Clinical Sciences & Operations

    Sanofi

    STUDY DIRECTOR

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

February 25, 2025

Record last verified: 2025-02

Data Sharing

IPD Sharing
Will share

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Locations

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