Phase 3 study for people with Achondroplasia

Recruiting Phase 3

Sources: ClinicalTrials.gov Checked: 11 October 2026 Not medical advice

This trial at a glance

Status Recruiting
Phase Phase 3
Sponsor BioMarin Pharmaceutical
Registry number NCT07441876
Started 20 April 2026 Today 16 October 2026 Estimated completion 1 June 2029

Where this trial is running

Trial sites and the status recorded for each
SiteCityState or countryStatus at this site
Murdoch Children's Research Institute Parkville VIC Recruiting

voxsanity.com.au · trial sites from public registries · registry record synced 11 October 2026 · not medical advice

Who can join

Age 2 Years to 17 Years
Sex Anyone, regardless of sex
Healthy volunteers Not accepted
Enrolment target 160

The official eligibility criteria

Registry text, reproduced exactly. These are the words the trial's sponsor published on ClinicalTrials.gov. They have not been edited, shortened or rewritten, and they are written for researchers rather than for patients.

Inclusion Criteria:

1. Participants must be aged ≥ 2 to \< 11 years (Phase 2) or ≥ 2 to \< 18 years (Phase 3), at the time of signing the informed consent

2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses

3. Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)

4. Are ambulatory and able to stand without assistance

Exclusion Criteria:

1. Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency)

2. Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated with evidence of normalized thyroid-stimulating hormone (TSH) levels, diabetes mellitus, unless considered well-controlled, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy, anemia defined as hemoglobin \< 10 g/dL, vitamin D deficiency, significant hip pathology.

3. Have history of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension.

4. Have had bone fractures of the long bones or spine within 6 months prior to screening.

5. Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the treatment of ACH or short stature at any time

6. Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start

Dates on the record

Started 20 April 2026
Estimated completion 1 June 2029
Registry last updated 24 September 2026
Checked by Voxsanity 11 October 2026

Results, publications and the official record

Sources and dates

  • ClinicalTrials.gov – the record this page reformats
  • ClinicalTrials.gov processing date: 24 September 2026.
  • This page last checked by Voxsanity: 11 October 2026.

Voxsanity reformats data from public registries into this layout. It does not edit, interpret or add to what the registry published.

Not medical advice.

Individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.

Letter for your doctor

A one-page summary of this trial to print or email to your GP. It carries the registry's own record of the trial and its eligibility criteria, and nothing Voxsanity has written about whether it suits you.