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hunter-syndrome-mps-ii Clinical Trials

A listing of hunter-syndrome-mps-ii medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 16 clinical trials
S Susan Mathus

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS 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 tividenofusp alfa (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 …

2 - 16 years of age Both Phase 2/3
E Elizabeth Jalazo, Dr.

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

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.

- 100 years of age Both Phase 3

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.

1 - 18 years of age Both Phase 1/2

Autologous Ex-vivo Gene Modified HSCT in MPSII

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 …

3 - 12 years of age Male Phase 1/2
S Site Contact

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

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 …

- 6 years of age Male Phase 4

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

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

18 - 18 years of age Both Phase 1

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302 [NCT02412787]). Participants must have completed one of the previous studies. The main aim of this study is to collect more information about the safety of the treatments, idursulfase-IT and elaprase, in children and adults with Hunter syndrome …

3 - 19 years of age Male Phase 2/3

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

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.

- 100 years of age Male Phase 2/3
L Lisa Burke, RN

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

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).

- 55 years of age Both Phase 2
E Emma Canepa, MS, CCRP

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

18 - 50 years of age Female Phase 1

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