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Active clinical trials for "Mucopolysaccharidoses"

Results 21-30 of 168

Gene Therapy in Patients With Mucopolysaccharidosis Disease

Mucopolysaccharidosis Type VI

This study investigated the safety and efficacy of gene therapy approaches for Mucopolysaccharidosis type VI disease caused by the deficiency of arylsulfatase B (ARSB) enzyme. The aim of the study is to evaluate the safety and efficacy of the treatment.

Active18 enrollment criteria

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis Type IIIBMPS III B

The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).

Active15 enrollment criteria

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Mucopolysaccharidosis II

Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.

Active7 enrollment criteria

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis...

Mucopolysaccharidosis Type IIIA

Patients with MPS IIIA have a clinical disorder marked by severe and progressive brain disease and neurological 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 SGSH gene) in MPSIIIA patients. Following treatment with the gene therapy patients will be followed up for a minimum of 3 years.

Active21 enrollment criteria

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and...

Mucopolysaccharidosis IV AMucopolysaccharidosis VI7 more

Mucopolysaccharidoses (MPS) are multisystemic diseases with significant clinical overlap between their types, with cardiac problems being among the most commonly observed manifestations and are also among the main causes of mortality in these patients. For some of the cardiovascular manifestations, such as aortic root dilation and valve diseases, there is no effective treatment currently available. Losartan, on the other hand, has been shown to be an effective drug for dilation of the aortic root, at least in animal models. This study aims to evaluate the safety and efficacy of losartan in patients with MPS VI and other mucopolysaccharidoses.

Active10 enrollment criteria

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.

Active9 enrollment criteria

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With...

Mucopolysaccharidosis IH

This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant

Active18 enrollment criteria

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

MPS IIIASanfilippo Syndrome2 more

The main objective of this study is to evaluate the efficacy and safety of ABO-102 for the treatment of MPS IIIA.

Active26 enrollment criteria

A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

Mucopolysaccharidosis III-A

The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.

Enrolling by invitation4 enrollment criteria

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Mucopolysaccharidosis IMucopolysaccharidosis II6 more

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.

Recruiting3 enrollment criteria
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