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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 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.

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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

FieldValue
Enrollment Target 60 participants
Start Date 2025-07-30
Est. Completion 2028-06-30
Phase Phase 2

Sponsor

BioMarin Pharmaceutical

80 total trials

What the Registry Record Tells You About NCT07126262

The ClinicalTrials.gov registry entry for NCT07126262 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 60 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 180-participant average among 4 other Hypochondroplasia trials with a reported enrollment target (67% lower). The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.

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 reports 20 study locations spanning 14 distinct geographic areas - top geographies include 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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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.