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A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

NCT06668805 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-05-11

No results posted yet for this study

Summary

The purpose of this study in children with 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.

Conditions

  • Noonan Syndrome

Interventions

DRUG

Vosoritide Injection

Modified recombinant human C-type natriuretic peptide Vosoritide

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD, PhD · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-22
Primary Completion
2027-03-31
Completion
2041-09-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • France
  • Germany
  • Italy
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06668805 on ClinicalTrials.gov