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Trackers› Clinical trials› Achondroplasia
Clinical trial register · United States · industry-sponsored

Achondroplasia Trials in the USA

Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors — the full US registry across all sponsors runs to over 22,000 recruiting studies alone.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice — all 9094 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 17 Sep 2026, 08:23 UTC
9094
Studies in scope
industry-sponsored, all four open statuses
4935
Recruiting now
open to new participants
2188
Lead sponsors
across recruiting studies
Top states
California 8 Texas 8 Maryland 7 Delaware 6 Wisconsin 6
Narrow the registerReset
Recruitment status
Phase
Lead sponsor
BioMarin Pharmaceutical5 QED Therapeutics, a BridgeBio company2 Tyra Biosciences, Inc1 Ascendis Pharma Growth Disorders A/S1 Ascendis Pharma A/S1
State
California8 Texas8 Maryland7 Delaware6 Wisconsin6
All 18 states
Target enrolment
to
10 of 9094 studies
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Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Recruiting
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
NCT07441876Achondroplasia
BioMarin Pharmaceutical
Phase 2/3
160
California, Delaware, Illinois, Maryland, Ohio, Pennsylvania, Texas
2026-04-20
Recruiting
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
NCT07169279Achondroplasia
QED Therapeutics, a BridgeBio company
Phase 2
77
California, Maryland, Wisconsin
2025-11-19
Recruiting
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
NCT06842355Achondroplasia
Tyra Biosciences, Inc
Phase 2
92
California, Colorado, Delaware, Maryland, Missouri, North Carolina, Tennessee, Texas
2025-03-04
Recruiting
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
NCT06079398Achondroplasia
Ascendis Pharma A/S
Phase 2
72
Minnesota, Texas, Wisconsin
2024-01-23
By invitation
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
NCT05929807Achondroplasia
Ascendis Pharma Growth Disorders A/S
Phase 2/3
140
Arkansas, Colorado, Delaware, Minnesota, Montana, New York, Texas, Wisconsin
2023-06-21
Recruiting
VIrtual STudy in Achondroplasia for the US (VISTA)
NCT06168201Achondroplasia
BioMarin Pharmaceutical
Observational
170
California, Maryland, North Carolina, Ohio, South Dakota, Texas, Wisconsin
2023-02-21
By invitation
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
NCT05145010Achondroplasia
QED Therapeutics, a BridgeBio company
Phase 2
300
California, Colorado, Delaware, Maryland, Missouri, Ohio, Tennessee, Wisconsin
2021-12-06
Active, closed
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT03989947Achondroplasia
BioMarin Pharmaceutical
Phase 2
73
California, Delaware, Georgia, Illinois, Ohio, Tennessee, Texas, Wisconsin
2019-06-12
Active, closed
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03424018Achondroplasia
BioMarin Pharmaceutical
Phase 3
119
California, Delaware, Georgia, Illinois, Maryland, Missouri, Ohio, Texas
2017-12-12
Active, closed
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT02724228Achondroplasia
BioMarin Pharmaceutical
Phase 2
30
California, Illinois, Maryland, Tennessee, Texas
2016-01-26
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Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 17 September 2026. https://priyalifescience.com/clinical-trials-usa/condition/achondroplasia

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Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.