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Achondroplasia clinical trials
Every US clinical trial registered for Achondroplasia — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
10 US clinical trials · 4 currently recruiting
The research picture
Achondroplasia has 10 registered US clinical trials, 4 of them open to new participants right now — about 40% of the total.
- 4
- recruiting participants now
- 40%
- of trials open to enrollment
- 1
- in Phase 3–4 (later-stage)
- 5
- top sponsor: BioMarin Pharmaceutical
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
VIrtual STudy in Achondroplasia for the US (VISTA)
BioMarin Pharmaceutical
NCT06168201
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Tyra Biosciences
NCT06842355
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
QED Therapeutics, a BridgeBio company
NCT07169279
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
Ascendis Pharma A/S
NCT06079398
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
QED Therapeutics, a BridgeBio company
NCT04035811
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
BioMarin Pharmaceutical
NCT03424018
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
BioMarin Pharmaceutical
NCT03989947
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
BioMarin Pharmaceutical
NCT02724228
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
BioMarin Pharmaceutical
NCT01603095
C-Type Natriuretic Peptide and Achondroplasia
Nemours Children's Clinic
NCT01541306
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 2 | 5 |
| Phase 3 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Achondroplasia Trial Landscape
ClinicalTrials.gov lists 10 US studies indexed under Achondroplasia, and 4 of those are currently open to recruitment — roughly 40% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Achondroplasia shows 1 late-stage studies (Phase 3 and Phase 4 combined) alongside 5 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Achondroplasia is led by BioMarin Pharmaceutical with 5 indexed trials, alongside 4 other organizations in the top contributor list. The list on this page surfaces up to 10 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Achondroplasia?
PlainTrial tracks 10 US clinical trials for Achondroplasia, of which 4 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Achondroplasia?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.