Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Achondroplasia: early-phase trial registry
4 of 10 indexed Achondroplasia trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #1,724 of 28,707 conditions by registered trial count.
10 US clinical trials · 4 currently recruiting · #1,724 of 28,707 conditions by registered trial count
Achondroplasia: 10 US clinical trials tracked, 4 recruiting.
Achondroplasia is the subject of 10 registered US clinical trials on ClinicalTrials.gov, 4 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 5 in Phase 1-2 (earlier-stage). The most active sponsor is BioMarin Pharmaceutical, running 5 of these trials.
Enrollment posture brief
Registry condition achondroplasia
OPEN 4 | RECR 40% | LATE 1 | EARLY 5 | RANK #1724 | SPON 5
Volume-matched condition peers
Nearest open-share peer: Acute Myeloid Leukemia (AML) (40% recruiting · 30 trials)
Achondroplasia lists 4 open slots (40% of 10 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Achondroplasia on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Achondroplasia is recruiting below its size-band peers.
4 of 10 indexed trials (40%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Achondroplasia research.
BioMarin Pharmaceutical accounts for 5 of 10 trials (50%). 5 distinct sponsors appear on at least one study for this condition label.
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Most Achondroplasia trials plan to enrol fewer than 100 people.
Median target enrolment is 92 among the 10 trials that report a genuine target (sentinel values excluded). 6 of those (60%) plan for fewer than 100 participants.
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Achondroplasia splits between interventional and observational designs.
6 interventional and 4 observational studies make up this index (60% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
10 total, page 1 of 1
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
Named interventions
Most-linked intervention names on Achondroplasia records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Achondroplasia trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Achondroplasia page
10 registered trials, 4 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 4 Achondroplasia trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- BioMarin Pharmaceutical sponsors the most Achondroplasia trials on record, review their full trial history before evaluating a specific study. See BioMarin Pharmaceutical's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Achondroplasia condition card totals 10 registered trials; most-linked intervention BMN 111; top US site state Wisconsin. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.