Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Experimental achondroplasia drug trial halted early

NCT ID NCT06067425

Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested an experimental drug called SAR442501 in children with achondroplasia, a common form of dwarfism. The goal was to see if the drug was safe and could improve growth. However, the trial was stopped early after enrolling only 16 children, so we have very little information about how well it worked.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
SAR442501 (an experimental drug given as a shot under the skin)
What this could lead to
If successful, this drug could help children with achondroplasia grow more, potentially reducing some health complications.
What could go wrong
The trial was terminated early with only 16 participants, so results are very limited. It is unclear if the drug is safe or effective, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

16 people

The number who actually took part.

Started

Oct 2023

Finished

Feb 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 days to 12 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Achondroplasia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

achondroplasia osteochondrodysplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Osteochondrodysplasia

Contacts and locations

Locations

  • Investigational Site Number : 0360001

    Parkville, Victoria, 3052, Australia

  • Investigational Site Number : 1560001

    Wuhan, 430030, China

  • Investigational Site Number : 1560002

    Shanghai, 200120, China

  • Investigational Site Number : 3800001

    Rome, Roma, 00168, Italy

  • Investigational Site Number : 3800002

    Milan, Lombardy, 20122, Italy

  • Investigational Site Number : 4100001

    Seoul, Seoul-teukbyeolsi, 03080, South Korea

  • Investigational Site Number : 4100002

    Seoul, Seoul-teukbyeolsi, 06351, South Korea

  • Investigational Site Number : 7240001

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

  • Investigational Site Number : 7240002

    Vitoria-Gasteiz, Basque Country, 01008, Spain

More trials for these conditions

Other studies related to the condition(s) this trial covers.