Clinical trials for Mucopolysaccharidosis
16 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- ENROLLING BY INVITATIONPHASE2, PHASE3NCT06075537An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007Denali Therapeutics Inc. · PI: Medical Monitor
- ENROLLING BY INVITATIONNCT06103487Long Term Follow-Up for RGX-111REGENXBIO Inc.
- RECRUITINGPhase 1NCT05682144ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS IImmusoft of CA, Inc. · PI: Immusoft Clinical Development
- ENROLLING BY INVITATIONPhase 3NCT05594992An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) SubjectsJCR Pharmaceuticals Co., Ltd.
- ACTIVE NOT RECRUITINGPhase 1NCT05422482A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS ⅡGC Biopharma Corp
- RECRUITINGPHASE2, PHASE3NCT05371613A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)Denali Therapeutics Inc. · PI: Jose Alcantara Rodriguez, PharmD
- RECRUITINGN/ANCT05619900Registry of Patients Diagnosed With Lysosomal Storage DiseasesUniversity of California, San Francisco · PI: Tippi C MacKenzie, MD
- ACTIVE NOT RECRUITINGPhase 3NCT04573023A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)JCR Pharmaceuticals Co., Ltd.
- RECRUITINGPHASE2, PHASE3NCT05208281A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type IIAO GENERIUM · PI: Oksana A. Markova, MD, MSc
- RECRUITINGNCT05284006Non-invasive Functional Assessment and Pathogenesis of Morquio ANemours Children's Clinic · PI: Shunji Tomatsu, MD PhD
- ENROLLING BY INVITATIONPhase 3NCT04360265Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIAUltragenyx Pharmaceutical Inc · PI: Medical Director
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT04251026A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter SyndromeDenali Therapeutics Inc. · PI: Sam Lu, MD
- ACTIVE NOT RECRUITINGPhase 2NCT04348136An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type IIJCR Pharmaceuticals Co., Ltd.
- ACTIVE NOT RECRUITINGPhase 2NCT03708965An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis IIJCR Pharmaceuticals Co., Ltd.
- RECRUITINGNCT03604835Mucopolysaccharidosis VII Disease Monitoring ProgramUltragenyx Pharmaceutical Inc · PI: Medical Director
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT03153319Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VILundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · PI: Lynda Polgreen, MD