Achondroplasia Clinical Trials

12 recruiting

Achondroplasia Trials at a Glance

11 actively recruiting trials for achondroplasia are listed on ClinicalTrialsFinder across 6 cities in 19 countries. The largest study group is Phase 2 with 5 trials, with the heaviest enrollment activity in Madison, Houston, and Paris. Lead sponsors running achondroplasia studies include Ascendis Pharma A/S, BioMarin Pharmaceutical, and Abbisko Therapeutics Co, Ltd.

Browse achondroplasia trials by phase

Treatments under study

About Achondroplasia Clinical Trials

Looking for clinical trials for Achondroplasia? There are currently 12 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Achondroplasia trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Achondroplasia clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 111 of 11 trials

Recruiting
Phase 2

A Study of TYRA-300 in Children With Achondroplasia: BEACH301

Achondroplasia
Tyra Biosciences, Inc92 enrolled15 locationsNCT06842355
Recruiting
Phase 2

A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.

Achondroplasia
Ascendis Pharma A/S24 enrolled5 locationsNCT06732895
Recruiting
Phase 2Phase 3

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Achondroplasia
BioMarin Pharmaceutical160 enrolled2 locationsNCT07441876
Recruiting
Phase 2

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

Achondroplasia
Ascendis Pharma A/S72 enrolled18 locationsNCT06079398
Recruiting
Phase 2

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Achondroplasia
QED Therapeutics, a BridgeBio company77 enrolled13 locationsNCT07169279
Recruiting

Achondroplasia Natural History Multicenter Clinical Study

Achondroplasia
Johns Hopkins University1,500 enrolled1 locationNCT02597881
Recruiting

Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia

AchondroplasiaHypochondroplasia
SYSNAV40 enrolled1 locationNCT07388966
Recruiting

VIrtual STudy in Achondroplasia for the US (VISTA)

Achondroplasia
BioMarin Pharmaceutical170 enrolled7 locationsNCT06168201
Recruiting

A Study in Children With Achondroplasia

Achondroplasia
Abbisko Therapeutics Co, Ltd260 enrolled8 locationsNCT07301463
Recruiting
Phase 3

Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia(AOBA Study)

Achondroplasia
Kyowa Kirin Co., Ltd.6 enrolled9 locationsNCT06926491
Recruiting

Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)

AchondroplasiaHypochondroplasia
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico200 enrolled1 locationNCT05328050