NCT06668805 A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
| NCT ID | NCT06668805 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | BioMarin Pharmaceutical |
| Condition | Noonan Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 30 participants |
| Start Date | 2024-11-22 |
| Primary Completion | 2027-03 |
Eligibility & Interventions
Eligibility Fast-Check
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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 30 participants in total. It began in 2024-11-22 with a primary completion date of 2027-03.
⚠ 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 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.
Eligibility Criteria
Inclusion Criteria: 1. Participants must be ≥ 3 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form 2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome. 3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex. 4. Tanner Stage 1, at time of signing the ICF. 5. Previous or current hGH treatment for short stature associated with their condition. 6. Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts Exclusion Criteria: 1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia. 2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. 3. Bone age advanced beyond chronological age by more than 2 years. 4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension, 5. Have an unstable condition likely to require surgical intervention during the study. 6. Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays. 7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period. 8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
Contact & Investigator
Medical Director, MD, PhD
STUDY DIRECTOR
BioMarin Pharmaceutical
Frequently Asked Questions
Who can join the NCT06668805 clinical trial?
This trial is open to participants of all sexes, aged 3 Years or older, up to 11 Years, studying Noonan Syndrome. 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 NCT06668805 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 NCT06668805 currently recruiting?
Yes, NCT06668805 is actively recruiting participants. Contact the research team at medinfo@bmrn.com for enrollment information.
Where is the NCT06668805 trial being conducted?
This trial is being conducted at Los Angeles, United States, Orange, United States, Aurora, United States, Wilmington, United States and 11 additional locations.
Who is sponsoring the NCT06668805 clinical trial?
NCT06668805 is sponsored by BioMarin Pharmaceutical. The principal investigator is Medical Director, MD, PhD at BioMarin Pharmaceutical. The trial plans to enroll 30 participants.