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Vosoritide for Selected Genetic Causes of Short Stature
NCT04219007 · View on ClinicalTrials.gov ↗
Study Summary
Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte. The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will benefit from treatment with vosoritide, a CNP analog, a selective NPR-B agonist which directly targets the growth plate. This study will enroll patients with short stature in selected genetic categories and will follow them for a 6 month observation period to obtain a baseline growth velocity, safety profile and quality of life assessment. Patients will then be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes.
Conditions Studied
Interventions
- DRUG Vosoritide
Study Locations (1)
District of Columbia
- Children's National Hospital — Washington D.C.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 56 participants |
| Start Date | 2020-08-04 |
| Est. Completion | 2035-06-01 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04219007
The ClinicalTrials.gov registry entry for NCT04219007 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 56 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Andrew Dauber, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Short Stature appearing as the primary indexed condition, and to 1 intervention — of which Vosoritide is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04219007 reports 1 study location spanning 1 distinct geographic area — top geographies include District of Columbia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04219007 about?
NCT04219007 is a clinical study titled "Vosoritide for Selected Genetic Causes of Short Stature". Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte. The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will b...
What is the current status of trial NCT04219007?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 56 participants. The study started on 2020-08-04. Estimated completion is 2035-06-01.
What conditions does trial NCT04219007 study?
This clinical trial studies the following conditions: Short Stature. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04219007?
The interventions under investigation include: Vosoritide (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04219007?
This trial is sponsored by Andrew Dauber, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04219007 being conducted?
This trial has 1 study location across District of Columbia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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