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NCT05845749 · ClinicalTrials.gov registry record · Phase 1
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
A Phase 1 study of MPS IVA and MPS VI, sponsored by University of California, San Francisco.
- Active
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 1
- Study location
NCT05845749: Active Phase 1 study of MPS IVA and MPS VI, sponsored by University of California, San Francisco.
NCT05845749 is a Phase 1 study of MPS IVA and MPS VI that is active but no longer recruiting, run by University of California, San Francisco. The registered enrollment target is 6 participants, below the 67-participant average among 5 other MPS IVA trials with a reported enrollment target (91% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05845749, a Phase 1 study of MPS IVA and MPS VI, is active but no longer recruiting, sponsored by University of California, San Francisco.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI. Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and determine annualized growth velocity. The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS. Exploratory endpoints include changes in linear and segmental growth as well as biomarkers of growth and bone metabolism.
Primary Outcome
Incidence of treatment-emergent adverse events as assessed by the evaluation of vital signs, pulse oximetry, pulmonary function, ECG (cardiac arrhythmia), ECHO (doppler of aortic velocity for stenosis, aortic valve area, and qualitative assessment of aortic valve thickness), spinal X-rays (worsening scoliosis, lordosis or kyphosis), standing lower extremity X-rays (worsening of genu valgum), decrease in six-minute walk distance and linear and segmental growth for determination of excessive or di
Interventions
- DRUG Vosoritide Injection [Voxzogo]
Study Locations (1)
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2023-09-25 |
| Est. Completion | 2029-12-31 |
| Phase | Phase 1 |
What the registry record for NCT05845749 still lists
NCT05845749 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 67-participant average among 5 other MPS IVA trials with a reported enrollment target (91% lower).
The record links to 2 conditions, with MPS IVA appearing as the primary indexed condition, and to 1 intervention - of which Vosoritide Injection [Voxzogo] is the first listed.
NCT05845749 reports a single indexed study location in California.
Frequently Asked Questions
What is clinical trial NCT05845749 about?
NCT05845749 is a clinical study titled "Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI". This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI. Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety ...
What is the current status of trial NCT05845749?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2023-09-25. Estimated completion is 2029-12-31.
What conditions does trial NCT05845749 study?
This clinical trial studies the following conditions: MPS IVA, MPS VI.
What interventions are being tested in trial NCT05845749?
The interventions under investigation include: Vosoritide Injection [Voxzogo] (DRUG).
Who is sponsoring clinical trial NCT05845749?
This trial is sponsored by University of California, San Francisco, which has 1,713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05845749 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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