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RECRUITING 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

NCT06668805 · View on ClinicalTrials.gov ↗

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

31 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06668805

The ClinicalTrials.gov registry entry for NCT06668805 describes a study currently listed as recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is BioMarin Pharmaceutical, which has 31 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT06668805 reports 20 study locations spanning 18 distinct geographic areas — top geographies include New York, Texas, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT06668805?

The interventions under investigation include: Vosoritide Injection (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT06668805?

This trial is sponsored by BioMarin Pharmaceutical, which has 31 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial