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

Recruiting Phase 2 Interventional Study
Noonan Syndrome
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
3 – 11
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
30 (estimated)
Sponsor
BioMarin Pharmaceutical · Industry
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About This Trial

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.

Trial Locations
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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 …
Contacts

Trial Specialist

1-800-983-4587

medinfo@bmrn.com

CONTACT

Study Manager

1-800-983-4587

Medinfo@bmrn.com

CONTACT