Expanded Access to Mepsevii

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

NCT01387854 ·Status: COMPLETED

Natural History Study of Patients With MPS IIIA

NCT02746341 ·Status: COMPLETED

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

NCT00214773 ·Status: COMPLETED

A Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

NCT02097251 ·Status: NO_LONGER_AVAILABLE

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

NCT00048711 ·Status: COMPLETED ·Phase: PHASE2

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

NCT01856218 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT03227042 ·Status: ACTIVE_NOT_RECRUITING

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT05492799 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE4

MPS (RaDiCo Cohort) (RaDiCo-MPS)

NCT06036693 ·Status: RECRUITING

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT02493998 ·Status: COMPLETED

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

NCT02716246 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

BMN 110 US Expanded Access Program

NCT01858103 ·Status: APPROVED_FOR_MARKETING

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

NCT04201405 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I

NCT03071341 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

NCT02371226 ·Status: COMPLETED ·Phase: PHASE1

RGX-111 Gene Therapy in Patients With MPS I

NCT03580083 ·Status: SUSPENDED ·Phase: PHASE1/PHASE2

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

NCT01509768 ·Status: COMPLETED

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

NCT00048620 ·Status: COMPLETED ·Phase: PHASE1

Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

NCT02597114 ·Status: COMPLETED ·Phase: PHASE1

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

NCT02618512 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis

NCT04112602 ·Status: COMPLETED

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

NCT06078553 ·Status: RECRUITING