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NPR-B Agonist

Vosoritide for Dwarfism

Phase 2
Waitlist Available
Led By Andrew Dauber, MS MMSc
Research Sponsored by Andrew Dauber
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights

Summary

This trial is testing vosoritide, a medication that helps children grow taller by targeting their bone growth areas. It is aimed at children with genetic conditions that cause short stature. The medication works by mimicking a natural substance in the body to stimulate bone growth.

Eligible Conditions
  • Dwarfism

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from Baseline in annualized growth velocity
Change from Baseline in standardized height SDS
Incidence of Symptomatic Hypotension events [Safety and Tolerability]
+1 more
Secondary study objectives
Changes from Baseline in bone age/chronological age
Changes in the difference between arm span and height from baseline
Changes in the seated height ratio as a measure of body proportions
Other study objectives
Changes from Baseline in bone mineral density
Changes from baseline in quality of life after daily SC injections of vosoritide: Quality of Life of Short Stature Youth (QoLISSY) questionnaire score is expressed as a percentage
Characterize maximum concentration (Cmax) of vosoritide in plasma
+6 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Genetic Short StatureExperimental Treatment1 Intervention
Vosoritide, also known as BMN 111 or modified recombinant human C-type natriuretic peptide (CNP), is a 39-amino-acid peptide analog that includes the 37 C-terminal amino acids of the human CNP53 sequence plus the addition of 2 amino acids (Pro-Gly) on the N-terminus. This structural modification conveys resistance to neutral endopeptidase (NEP) degradation, resulting in prolonged half-life (t1/2) in comparison to endogenous CNP. This increase in t1/2 allows once daily subcutaneous (SC) administration. Vosoritide will be administered as a single 15 μg/kg subcutaneous injection given daily for 12 months.

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Who is running the clinical trial?

Andrew DauberLead Sponsor
Andrew Dauber, MS MMSc5.01 ReviewsPrincipal Investigator - Children's National Research Institute
Children's National Research Institute
5Patient Review
We had a great experience with this doctor. He was very thorough and took the time to answer all of our questions. We felt very comfortable with him and would highly recommend him to others.
~7 spots leftby Sep 2025
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