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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

No Placebo-Only Group

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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.