search

Active clinical trials for "Mucopolysaccharidosis II"

Results 1-10 of 68

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis...

Mucopolysaccharidosis (MPS)Hunter Syndrome

The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome. In this open label, single arm study, all participants will receive ELAPRASE treatment and a prophylactic ITR. Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week cycle of: Rituximab (intravenously [IV], weekly for 4 weeks); Methotrexate (oral, 3 times per week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle). Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18 regarding the need for administering another 5-week cycle of the ITR. Participants will be in the study for approximately 112 weeks (including 6 weeks for screening, up to 104 weeks for treatment, and 2 weeks for follow-up).

Recruiting17 enrollment criteria

A Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants...

Mucopolysaccharidosis II

This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of DNL310, an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.

Recruiting10 enrollment criteria

A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India...

Hunter Syndrome

The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome. Participants will receive Elaprase once per week over a 3-hour period which can be reduced to 1 hour as determined by the study doctor. Participants will need to visit the clinic weekly during the duration of the study.

Recruiting17 enrollment criteria

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients...

Mucopolysaccharidosis IIHunter Syndrome

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug

Recruiting15 enrollment criteria

Autologous Ex-vivo Gene Modified HSCT in MPSII

Mucopolysaccharidosis II

Patients with MPS II have a clinical disorder marked by progressive brain disease, neurological and somatic symptoms due to the accumulation of undigested glycosaminoglycans in all cells of the body. This study will be the first in human clinical trial to explore the safety, tolerability and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human IDS gene) in MPSII patients. Following treatment with the gene therapy patients will be followed up for a minimum of 2 years.

Recruiting23 enrollment criteria

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Mucopolysaccharidosis II

A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.

Recruiting26 enrollment criteria

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis...

Mucopolysaccharidosis Type IIMetabolic Diseases

This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.

Recruiting9 enrollment criteria

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.

Recruiting34 enrollment criteria

A Study of DNL310 in Pediatric Participants With Hunter Syndrome

Mucopolysaccharidosis II

This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL310, an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.

Recruiting17 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
12...7

Need Help? Contact our team!


We'll reach out to this number within 24 hrs