Condition

Achondroplasia clinical trials and research

• 9Clinical trials
• 24Countries

Newest Achondroplasia clinical trials

All recruiting trials (9)

• BMN 333 versus daily vosoritide for growth in children with achondroplasia

  This trial tests whether weekly BMN 333 injections help children with achondroplasia grow as well as daily vosoritide.

  Recruiting at 25 sites Phase 2/3 BioMarin Pharmaceutical · Oakland, California and 27 other locations · NCT07441876

• Measuring limb movement in people with FGFR3-related skeletal dysplasia

  This project will try using Syde® wearable movement recordings to see which measurements reliably track limb movement in people aged 3 and older with achondroplasia or hypochondroplasia.

  Recruiting Sysnav · Liège and 3 other locations · NCT07388966

• Observing children with achondroplasia over time

  Collecting growth, clinical, and quality-of-life information from children with achondroplasia aged 2.5 to under 11 to see how the condition and treatments affect them over 6 months to 2 years.

  Recruiting at 3 sites Abbisko Therapeutics Co, Ltd · Beijing, Beijing Municipality and 7 other locations · NCT07301463

• Infigratinib treatment for children under 3 with achondroplasia

  This trial tests whether daily oral infigratinib helps children younger than 3 years who have achondroplasia.

  Recruiting at 12 sites Phase 2 QED Therapeutics, a BridgeBio company · Oakland, California and 11 other locations · NCT07169279

• Evaluating TYRA-300 for children with achondroplasia

  This study is testing a new medication called TYRA-300 to see if it is safe and helps children aged 3 to 10 with achondroplasia grow better.

  Recruiting at 25 sites Phase 2 Tyra Biosciences, Inc · Torrance, California and 24 other locations · NCT06842355

• Observational study of achondroplasia in the US

  This study looks at children in the U.S. with achondroplasia to see how they are doing with and without the treatment VOXZOGO™ over at least five years.

  Recruiting at 8 sites BioMarin Pharmaceutical · Oakland, California and 7 other locations · NCT06168201

• Evaluating a new treatment for infants with achondroplasia

  This study is testing a new treatment for infants with achondroplasia to see if it helps them grow better compared to a placebo.

  Recruiting at 18 sites Phase 2 Ascendis Pharma A/S · Saint Paul, Minnesota and 17 other locations · NCT06079398

• Registry for patients with achondroplasia and hypochondroplasia

  This study is collecting health information from people with achondroplasia and hypochondroplasia to help understand these conditions better and improve future treatments.

  Recruiting Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico · Milan · NCT05328050

• Multicenter achondroplasia natural history registry

  This project will create a U.S. registry to collect medical and treatment information from people with achondroplasia so researchers can see patterns over time.

  Recruiting Johns Hopkins University · Baltimore, Maryland · NCT02597881

Related topics

Quick answers

How many Achondroplasia clinical trials are recruiting right now?

9, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 24 countries. The most sites are in United States (6 trials), United Kingdom (5 trials), Australia (4 trials), Canada (4 trials), Italy (3 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 3, Phase 2/3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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