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NCT07126262 · ClinicalTrials.gov registry record · Phase 2
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
A Phase 2 study of Hypochondroplasia, sponsored by BioMarin Pharmaceutical.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 60
- Enrollment target
- 20
- Study locations
NCT07126262: Recruiting Phase 2 study of Hypochondroplasia, sponsored by BioMarin Pharmaceutical.
NCT07126262 is a Phase 2 study of Hypochondroplasia that is actively recruiting participants, run by BioMarin Pharmaceutical. The registered enrollment target is 60 participants, below the 180-participant average among 4 other Hypochondroplasia trials with a reported enrollment target (67% lower). The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07126262, a Phase 2 study of Hypochondroplasia, is actively recruiting participants, sponsored by BioMarin Pharmaceutical.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 60 participants
- Enrollment target
- 20
- Study locations
Study Summary
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Vosoritide
Study Locations (20)
California
- Cedars-Sinai Medical Center - Los Angeles
- Benioff Children's Hospital - Oakland - Oakland
- Benioff Children's Hospital - Oakland - Oakland
District of Columbia
- Children's National Medical Center - Washington D.C.
- Children's National Medical Center - Washington D.C.
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Missouri
- University of Missouri - Columbia
- University of Missouri - Columbia
Victoria
- Royal Children's Hospital Melbourne - Parkville
- Royal Children's Hospital Melbourne - Parkville
Arizona
- Phoenix Children's Hospital - Thomas Campus (Main) - Phoenix
Maryland
- The Johns Hopkins University School of Medicine - Baltimore
Minnesota
- University of Minneasota Masonic Children's Hospital - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 60 participants |
| Start Date | 2025-07-30 |
| Est. Completion | 2028-06-30 |
| Phase | Phase 2 |
What NCT07126262 shows while recruiting
NCT07126262 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 60 participants, a relatively small participant target, below the 180-participant average among 4 other Hypochondroplasia trials with a reported enrollment target (67% lower).
The record links to 1 condition, with Hypochondroplasia appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT07126262 names 20 study sites across 14 states, led by California, District of Columbia, Illinois.
Frequently Asked Questions
What is clinical trial NCT07126262 about?
NCT07126262 is a clinical study titled "A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months". The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
What is the current status of trial NCT07126262?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 60 participants. The study started on 2025-07-30. Estimated completion is 2028-06-30.
What conditions does trial NCT07126262 study?
This clinical trial studies the following conditions: Hypochondroplasia.
What interventions are being tested in trial NCT07126262?
The interventions under investigation include: Placebo (DRUG), Vosoritide (DRUG).
Who is sponsoring clinical trial NCT07126262?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07126262 being conducted?
This trial has 20 study locations across Arizona, California, District of Columbia, Illinois, Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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