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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

Study Summary

This trial is testing whether vosoritide, a drug that targets the growth plate, can help people with certain genetic causes of short stature.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
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 outcome measures
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.

Find a Location

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.

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can young adults participate in this research project?

"This particular trial is only for children aged 3 to 10. However, there are 17 other trials that patients under 18 can participate in and 7 trials available for patients 65 and older."

Answered by AI

How many individuals have signed up for this research project?

"As of 4/29/2021, this clinical trial is not recruiting patients. The study was initially posted on 8/4/2020. If you are looking for other similar studies, there are 13 trials for dwarfism and 5 trials for Vosoritide that are currently recruiting participants."

Answered by AI

Could you please explain what prior research has been conducted with Vosoritide?

"At the moment, there are 5 ongoing studies about Vosoritide. 1 of those is in Phase 3. Although most of the research for Vosoritide takes place in Osaka and Texas, there are 53 different locations running trials for this medication."

Answered by AI

What makes this trial unique compared to other similar ones?

"There have been a total of 5 clinical trials for Vosoritide since 2016. The first study was sponsored by BioMarin Pharmaceutical and included 30 patients. It completed Phase 2 approval in 2016. The most recent trial concluded in 2020."

Answered by AI

Are there any dangers associated with Vosoritide use?

"Vosoritide's safety is estimated to be a 2. In other words, while there is some evidence that it is safe, no data exists yet to show that it is effective."

Answered by AI

How do I qualify to participate in this research?

"This clinical trial is recruiting 35 patients, within the ages of 3 and 10 who have dwarfism. It is important that patients also meet the following criteria: B. Hypochondroplasia - Subjects with heterozygous variants in FGFR3 gene associated with hypochondroplasia are eligible. Subjects with variants in FGFR3 known to cause achondroplasia or thanatophoric dysplasia or SADDAN syndrome will be excluded., C. Patients with heterozygous defects in NPR2 are eligible. Patients with homozygous defects in NPR2 will be excluded., Age >3 years 0 days"

Answered by AI

Are people still being accepted into this research project?

"According to the latest information on clinicaltrials.gov, this particular trial is not seeking patients at this time. The trial was initially posted on 8/4/2020 and last updated on 4/29/2021; however, there are 18 other trials that are currently looking for participants."

Answered by AI

Who else is applying?

What state do they live in?
Illinois
How old are they?
< 18
What site did they apply to?
Children's National Hospital
What portion of applicants met pre-screening criteria?
Met criteria

How responsive is this trial?

Typically responds via
Email
Most responsive sites:
  1. Children's National Hospital: < 24 hours
Average response time
  • < 1 Day
Recent research and studies
clinicaltrials.govStudy
Vosoritide for Selected Genetic Causes of Short Stature
Andrew Dauber 2020. "Vosoritide for Selected Genetic Causes of Short Stature". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04219007.
~7 spots leftby Apr 2025
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