NCT06382155 A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
| NCT ID | NCT06382155 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | BioMarin Pharmaceutical |
| Condition | Idiopathic Short Stature |
| Study Type | INTERVENTIONAL |
| Enrollment | 100 participants |
| Start Date | 2024-10-21 |
| Primary Completion | 2026-06 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 100 participants in total. It began in 2024-10-21 with a primary completion date of 2026-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).
Eligibility Criteria
Key Inclusion Criteria: 1. Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts 2. If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs). Key Exclusions: 1. Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency. 2. Previous treatment with a growth promoting agent
Contact & Investigator
Medical Director MD
STUDY DIRECTOR
BioMarin Pharmaceutical
Frequently Asked Questions
Who can join the NCT06382155 clinical trial?
This trial is open to participants of all sexes, aged 3 Years or older, up to 11 Years, studying Idiopathic Short Stature. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06382155 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06382155 currently recruiting?
Yes, NCT06382155 is actively recruiting participants. Contact the research team at medinfo@bmrn.com for enrollment information.
Where is the NCT06382155 trial being conducted?
This trial is being conducted at California City, United States, California City, United States, California City, United States, Los Angeles, United States and 11 additional locations.
Who is sponsoring the NCT06382155 clinical trial?
NCT06382155 is sponsored by BioMarin Pharmaceutical. The principal investigator is Medical Director MD at BioMarin Pharmaceutical. The trial plans to enroll 100 participants.