Health TrialsbyHealthPanel

Clinical trials

7

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

First-in-human Study of UX016 in GNEM

The goal of this study is to assess the safety and dose of UX016 and its impact on muscle strength in adults with GNE Myopathy (GNEM).

Participants needed: 24
Trial details
Phase: Phase 1, Phase 2Age: 18-55Biological sex: AllType: InterventionalSponsor: Ultragenyx Pharmaceutical IncUpdated: Oct 13, 2026Locations: 2
Eligibility criteria

A confirmed diagnosis of GNEM (also known as distal myopathy with rimmed vacuole... [+5]

Ingestion of N-acetyl-D-mannosamine (ManNAc), SA, or related metabolites, includ... [+10]

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Status: Not yet recruiting

MPS IIIA Disease Monitoring Program

The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.

Participants needed: 55
Trial details
Biological sex: AllType: ObservationalSponsor: Ultragenyx Pharmaceutical IncUpdated: Oct 2, 2026
Eligibility criteria

Confirmed diagnosis of MPS IIIA [+3]

Presence or history of any condition that, in the view of the Investigator, woul...

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Status: Recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

Participants needed: 36
Trial details
Phase: Phase 2, Phase 3Biological sex: AllType: InterventionalSponsor: Ultragenyx Pharmaceutical IncUpdated: Sep 22, 2026Locations: 5
Eligibility criteria

No detectable or significantly reduced SGSH enzyme activity by leukocyte assay,... [+4]

Inability to participate in the clinical evaluation as determined by PI [+29]

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Status: Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.

Participants needed: 140
Trial details
Age: 2+Biological sex: AllType: ObservationalSponsor: Ultragenyx Pharmaceutical IncUpdated: Aug 26, 2026Locations: 19
Eligibility criteria

DTX401 (full or partial dose) administered in a parent clinical study (Group 1)... [+2]

Presence of any condition that would interfere with study participation, interpr...

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Status: Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

Participants needed: 50
Trial details
Biological sex: AllType: ObservationalSponsor: Ultragenyx Pharmaceutical IncUpdated: Aug 27, 2026Locations: 14
Eligibility criteria

Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic o... [+2]

Concurrent participation in other pharmaceutical company-sponsored interventiona...

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Status: Available

Expanded Access to Mepsevii

Individual patient expanded access requests may be considered for patients who have no other treatment options

Trial details
Biological sex: AllType: Expanded AccessSponsor: Ultragenyx Pharmaceutical IncUpdated: Jul 30, 2026
Eligibility criteria

Not listed

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Status: Available

Expanded Access to Triheptanoin

Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.

Trial details
Biological sex: AllType: Expanded AccessSponsor: Ultragenyx Pharmaceutical IncUpdated: Jul 31, 2026
Eligibility criteria

Not listed

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