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Active clinical trials for "Mucopolysaccharidoses"

Results 11-20 of 168

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Mucopolysaccharidosis IH/SMucopolysaccharidosis IS

A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.

Recruiting15 enrollment criteria

A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With...

Mucopolysaccharidosis IHurler's Syndrome2 more

The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic response of their breastfed infants.

Recruiting6 enrollment criteria

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like...

AdrenoleukodystrophyBatten Disease14 more

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

Recruiting28 enrollment criteria

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Mucopolysaccharidosis DisordersHurler Syndrome27 more

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

Recruiting35 enrollment criteria

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Participants...

Mucopolysaccharidosis II

This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with DNL310, an investigational central nervous system (CNS)-penetrant intravenous (IV) enzyme replacement therapy (ERT) for Hunter syndrome (MPS II). Participants who complete at least through the Week 49 visit in Study DNLI-E-0002 and do not discontinue study intervention early and participants who complete Study DNLI-E-0007 will be enrolled in this OLE. All participants will receive DNL310 for up to 5 years from the time of entry in this OLE. Participants, site staff, and the Sponsor will remain blinded to the original treatment assignment for participants entering this OLE from Study DNLI-E-0007.

Enrolling by invitation5 enrollment criteria

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis...

Mucopolysaccharidosis II

An extension of Global Phase III open-label, multicenter designed to evaluate the Long-term safety and efficacy of study drug for the treatment of the MPS II.

Enrolling by invitation16 enrollment criteria

RGX-111 Gene Therapy in Patients With MPS I

Mucopolysaccharidosis Type I (MPS I)Hurler Syndrome1 more

RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.

Active9 enrollment criteria

An Extension Study of JR-171-101 Study in Patients With MPS I

Mucopolysaccharidosis I

Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 for the treatment of MPS I

Active7 enrollment criteria

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.

Active14 enrollment criteria

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Mucopolysaccharidosis IMucopolysaccharidosis II1 more

Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.

Active23 enrollment criteria
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