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Mucopolysaccharidosis I clinical trials

View clinical trials related to Mucopolysaccharidosis I.

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NCT ID: NCT06406153 Completed - Clinical trials for Mucopolysaccharidosis Type 1

Efficacy and Safety of P046 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients

Start date: September 10, 2022
Phase: Phase 3
Study type: Interventional

The purpose of this phase III study is to assess the efficacy and safety of P046 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients. All patients receive Aldurazyme® for 12 weeks, followed by P046 for another 12 weeks. The primary outcome is the assessment of the maintenance of the mean uGAG levels at the end of each medication administration. The secondary outcomes are the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs).

NCT ID: NCT06149403 Recruiting - Clinical trials for MPS-IH (Hurler Syndrome)

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

HURCULES
Start date: December 11, 2023
Phase: Phase 3
Study type: Interventional

A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).

NCT ID: NCT06103487 Enrolling by invitation - Clinical trials for Mucopolysaccharidosis I

Long Term Follow-Up for RGX-111

Start date: July 24, 2023
Phase:
Study type: Observational

RGX-111-5101 is a long-term follow up study that evaluates the long term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.

NCT ID: NCT06036693 Recruiting - Clinical trials for Mucopolysaccharidosis II

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Start date: December 20, 2017
Phase:
Study type: Observational

The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collection of extensive phenotypic data from French MPS patients.

NCT ID: NCT05687474 Recruiting - Cystic Fibrosis Clinical Trials

Baby Detect : Genomic Newborn Screening

Start date: September 1, 2022
Phase:
Study type: Observational

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.

NCT ID: NCT05682144 Recruiting - Clinical trials for Mucopolysaccharidosis IS

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

Start date: April 12, 2023
Phase: Phase 1
Study type: Interventional

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

NCT ID: NCT05634512 Recruiting - Clinical trials for Hematopoietic Cell Transplantation

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Start date: November 17, 2022
Phase:
Study type: Observational

This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant. Sixteen patients will be enrolled over a 24 month period.

NCT ID: NCT05619900 Recruiting - Clinical trials for Mucopolysaccharidosis II

Registry of Patients Diagnosed With Lysosomal Storage Diseases

LSD Registry
Start date: May 31, 2022
Phase:
Study type: Observational [Patient Registry]

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.

NCT ID: NCT05134571 Completed - Clinical trials for Mucopolysaccharidosis I

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

Start date: October 28, 2021
Phase: Phase 4
Study type: Interventional

This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I. Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) from baseline to Week 26. The study will also evaluate the effect on uGAG level and liver volume (hepatomegaly) after 26 weeks, with Aldurazyme treatment in Chinese MPS I participants. Treatment duration will include: 2 weeks of screening, 26 weeks of treatment and 1 week of follow-up period. During the treatment period, weekly visits are designed to accommodate weekly administration of Aldurazyme (laronidase).

NCT ID: NCT05073783 Completed - Pompe Disease Clinical Trials

A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting

HomERT
Start date: October 14, 2021
Phase:
Study type: Observational

Primary objective: To obtain data pertaining to the safety and tolerability of alglucosidase alfa and laronidase treatments administered in a home-care infusion setting. Secondary objectives: - To evaluate personal satisfaction of both cohorts of patients treated in a home-care infusion setting. - To evaluate the infusion compliance in both cohorts of patients treated in a home-care infusion setting.