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Trial · NCT05845749

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

NCT05845749, Phase 1, Phase 2, Active not recruiting, University of California, San Francisco

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI. Trials, record NCT05845749. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05845749

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
NCT number
NCT05845749
Status
Active not recruiting
Phases
Phase 1, Phase 2
Enrollment
6 participants, actual
Lead sponsor
University of California, San Francisco
Conditions
MPS IVA, MPS VI
Interventions
Vosoritide Injection [Voxzogo]
First posted
2023-05-06
Last updated
2026-03-10

Source ClinicalTrials.gov

Registered outcomes2

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
PrimarySafety and tolerability: Incidence of adverse events while treated with vosoritide48 weeks
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
SecondaryChange in height velocity while treated with vosoritide120 weeksExplore 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

Outcomes0

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

None in this release.

Results by group0

Each measurement the trial posted, group by group, in the words and numbers it posted.

None in this release.

Participant flow0

How many started, completed or left each group, period by period.

None in this release.

Adverse events0

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

None in this release.

Adverse event totals0

The totals the trial posted for each group.

None in this release.

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov
2026-11-12
ClinicalTrials.gov
Results, from AACT
2026-03-23
AACT
NIH grant links
2026-11-13
NIH RePORTER

Sources 3

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
  • NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ClinicalTrials.govnone stated
  • NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT05845749

GET https://conductscience.org/api/trials/v1/NCT05845749/registered