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NCT06668805 · ClinicalTrials.gov registry record · Phase 2

A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

A Phase 2 study of Turner Syndrome and Noonan Syndrome, sponsored by BioMarin Pharmaceutical.

Recruiting
Registry status
Phase 2
Development phase
54
Enrollment target
20
Study locations

NCT06668805 is a Phase 2 study of Turner Syndrome and Noonan Syndrome that is actively recruiting participants, run by BioMarin Pharmaceutical. The registered enrollment target is 54 participants, below the 515-participant average among 14 other Turner Syndrome trials with a reported enrollment target (90% lower). The trial reports 20 study locations across 18 states.

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The verdict

NCT06668805, a Phase 2 study of Turner Syndrome and Noonan Syndrome, is actively recruiting participants, sponsored by BioMarin Pharmaceutical.

RECRUITING
Registry status
Phase 2
Development phase
54 participants
Enrollment target
20
Study locations

Study Summary

The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.

Interventions

  • DRUG Vosoritide Injection

Study Locations (20)

New York

  • Icahn School of Medicine at Mount Sinai - New York
  • Children's Hospital at Montefiore - The Bronx

Texas

  • Children's Medical Center Dallas - Dallas
  • University of Texas Health Science Center at Houston (UT Health) - Houston

California

  • Children's Hospital of Orange County Main Campus - Orange - Orange

Colorado

  • Children's Hospital Colorado - Aurora

Delaware

  • Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children) - Wilmington

District of Columbia

  • Children's National Medical Center - Washington D.C.

Florida

  • Nicklaus Children's Hospital - Miami

Idaho

  • St. Luke's Children's Endocrinology and Diabetes - Boise

Trial Details

FieldValue
Enrollment Target 54 participants
Start Date 2024-11-22
Est. Completion 2041-09
Phase Phase 2

Sponsor

BioMarin Pharmaceutical

80 total trials

What the Registry Record Tells You About NCT06668805

The ClinicalTrials.gov registry entry for NCT06668805 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 515-participant average among 14 other Turner Syndrome trials with a reported enrollment target (90% lower). The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.

The record links to 3 conditions, with Turner Syndrome appearing as the primary indexed condition, and to 1 intervention - of which Vosoritide Injection is the first listed.

NCT06668805 reports 20 study locations spanning 18 distinct geographic areas - top geographies include New York, Texas, California.

Frequently Asked Questions

What is clinical trial NCT06668805 about?

NCT06668805 is a clinical study titled "A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment". The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluat...

What is the current status of trial NCT06668805?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 54 participants. The study started on 2024-11-22. Estimated completion is 2041-09.

What conditions does trial NCT06668805 study?

This clinical trial studies the following conditions: Turner Syndrome, Noonan Syndrome, Short Stature Homeobox- Containing Gene SHOX Deficiency.

What interventions are being tested in trial NCT06668805?

The interventions under investigation include: Vosoritide Injection (DRUG).

Who is sponsoring clinical trial NCT06668805?

This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT06668805 being conducted?

This trial has 20 study locations across California, Colorado, Delaware, District of Columbia, Florida. 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

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