Trial · Interventional
Active, not recruitingPhase 1/2Registry dataSafety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
Overview
- Status
- Active, not recruiting
- Phase
- Phase 1/2
- Enrollment
- 6 participants (actual)
- Sponsor
- University of California, San Francisco · Other
- Design
- Allocation not applicable · Open label (no masking)
- Countries
1 country
United States
- Start
- 25 Sept 2023
- Primary completion
- 31 Dec 2028 (estimated)
- Completion
- 31 Dec 2029 (estimated)
- First posted
- 6 May 2023
- Last update posted
- 10 Mar 2026
- Conditions
- MPS IVA · MPS VI
Timeline
1 trial · 1 ongoing
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
- Ph 1/2
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
NCT0584574925 Sept 2023 to est. 31 Dec 2029
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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
- Vosoritide Injection [Voxzogo]Drug
Arms1
Other
Vosoritide
This is a single arm open label study of daily SQ dose of vosoritide
Primary outcomes1
Outcomes the sponsor listed as primary.
Safety and tolerability: Incidence of adverse events while treated with vosoritide
Time frame · 48 weeks
Registry description
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 disproportionate growth. All safety assessments will be performed at a minimum at the beginning of the intervention (Visit 1) and the end of the intervention (Visit 3) in patients with MPS IVA and VI
Secondary outcomes(1)
Change in height velocity while treated with vosoritide
Time frame · 120 weeks
Registry description
Explore the change from baseline (0-24 weeks pre-intervention) in age-sex annualized height velocity after 96 weeks of daily subcutaneous vosoritide therapy in patients with MPS IVA and VI
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
No results are posted for this trial.
A trial that is still running, or that stopped early, may post results later.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 5 Years up to 10 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
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