search

Active clinical trials for "Mucopolysaccharidosis I"

Results 1-10 of 59

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

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

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

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

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

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

Baby Detect : Genomic Newborn Screening

Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1134 more

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.

Recruiting4 enrollment criteria

Mucopolysaccharidosis I (MPS I) Registry

Mucopolysaccharidosis I (MPS I)

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities. The objectives of the Registry are: To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase) To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

Recruiting3 enrollment criteria
12...6

Need Help? Contact our team!


We'll reach out to this number within 24 hrs