vestronidase alfa: Clinical Trials

6 trials of vestronidase alfa, newest first
ClinicalTrials Daily  ·  Areas  ·  Conditions  ·  Drugs  ·  Sponsors  ·  

3 completed, 1 available, 1 no longer available, 1 recruiting. 2 Phase 3, 1 Phase 1, 1 Phase 2.

By phase and status

PhaseTrialsStatus
Phase 32Completed 2
Phase 11Recruiting 1
Phase 21Completed 1

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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Aldurazyme (laronidase) - MPS I, MPS II		Phase 1RecruitingUniversity of California, San Francisco102021-07-012026-03-17
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
UX003 - Sly Syndrome, MPS VII		Phase 3CompletedUltragenyx Pharmaceutical Inc122015-11-102020-07-30
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
UX003 - Sly Syndrome, MPS VII		Phase 2CompletedUltragenyx Pharmaceutical Inc82015-07-212019-10-30
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
UX003, Placebo - MPS 7, Sly Syndrome		Phase 3CompletedUltragenyx Pharmaceutical Inc122014-12-012020-07-30
Expanded Access to Mepsevii
Mepsevii - MPS VII, Mucopolysaccharidosis VII		AvailableUltragenyx Pharmaceutical Inc2026-07-30
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
UX003 - Mucopolysaccharidosis Type 7		No longer availableJoyce Fox2017-02-06

Conditions studied

Mucopolysaccharidosis VII (6)  ·  Lysosomal Storage Diseases (4)  ·  Metabolic Diseases (4)  ·  Rare Diseases (4)  ·  Mucopolysaccharidoses (3)  ·  Gaucher Disease (1)  ·  Mucopolysaccharidosis IV (1)  ·  Mucopolysaccharidosis VI (1)  ·  Sudden Infant Death (1)  ·  Wolman Disease (1)

Sponsors

Ultragenyx Pharmaceutical Inc (4)  ·  Joyce Fox (1)  ·  University of California, San Francisco (1)

Countries

United States (5)  ·  Portugal (2)  ·  Brazil (1)  ·  Mexico (1)  ·  Spain (1)

Top investigators

Chester Whitley, MD (1)  ·  Mislen Bauer, MD (1)  ·  Paul Harmatz, MD (1)  ·  Raymond Wang, MD (1)  ·  Tippi MacKenzie, MD (1)

Results publications

PMID 37415957, PMID 30467742, PMID 32063397, PMID 33874971, PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 29478819, PMID 30337566, PMID 32102934, PMID 35331634, PMID 37955580, PMID 39891377, PMID 9918480

NIH funding

2015 (2)