Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05845749
The ClinicalTrials.gov registry entry for NCT05845749 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 67-participant average among 5 other MPS IVA trials with a reported enrollment target (91% lower). The listed sponsor is University of California, San Francisco, which has 1,713 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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.
Learn More About Clinical Trials
Similar trials for MPS IVA
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
RECRUITING · Phase 1
-
Non-invasive Functional Assessment and Pathogenesis of Morquio A
RECRUITING
-
Cardiac Structure and Function in MPS
RECRUITING
-
Gait Analysis in MPS IVA
COMPLETED
-
Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
COMPLETED
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.