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Mucopolysaccharidoses clinical trials

View clinical trials related to Mucopolysaccharidoses.

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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: NCT06333041 Not yet recruiting - Sanfilippo Syndrome Clinical Trials

Study of Cannabidiol in Sanfilippo Syndrome

Start date: May 2024
Phase: Phase 2/Phase 3
Study type: Interventional

The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in treating the neurobehavioral symptoms and functional outcomes of Sanfilippo syndrome. Each participant's caregiver will be asked to complete surveys related to the participant's behavior, mood, sleep, stooling, pain, and caregiver stress intermittently throughout the study. All participants will be enrolled into one of two cohorts based on enrollment order: 1. Sentinel Safety Cohort (first 5 participants) - all participants treated with Epidiolex (cannabidiol) 2. Controlled Cohort (next 30 participants) - participants randomized 1:1 (equal chance) to start treatment with Epidiolex (cannabidiol) or placebo for 16 weeks, followed by an 8-week washout period (no treatment). Participants then switch to the opposite treatment group for 16 weeks followed by all participants treated for 52 weeks with Epidiolex (cannabidiol).

NCT ID: NCT06181136 Recruiting - Clinical trials for Mucopolysaccharidosis Type IIIA

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Start date: December 7, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and exploratory clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). The core study period is 25 weeks (approximately 6 months) and is followed by a 72-week (approximately 18 month) open-label extension (OLE).

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: NCT06095388 Recruiting - Clinical trials for Mucopolysaccharidosis III-A

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

Start date: October 4, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

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.

NCT ID: NCT06075537 Enrolling by invitation - Clinical trials for Mucopolysaccharidosis II

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Start date: September 20, 2023
Phase: Phase 2/Phase 3
Study type: Interventional

This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous (IV) enzyme replacement therapy (ERT) for Hunter syndrome (MPS II). Participants who complete at least through the Week 49 visit in Study DNLI-E-0002 and do not discontinue study intervention early and participants who complete Study DNLI-E-0007 will be enrolled in this OLE. All participants will receive DNL310 for up to 5 years from the time of entry in this OLE. Participants, site staff, and the Sponsor will remain blinded to the original treatment assignment for participants entering this OLE from Study DNLI-E-0007.

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: NCT06022380 Completed - Clinical trials for Mucopolysaccharidosis Type II

Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II

COHERE
Start date: September 28, 2020
Phase:
Study type: Observational

The Mucopolysaccharidoses (MPS) are a family of genetic metabolic disorders, caused by specific enzyme deficiencies which result in accumulation of partially degraded glycosaminoglycans (GAGs) within various tissues. As GAGs are common in the body a number of different organ systems can be affected. Involvement of the upper and lower respiratory tract in MPS Type II results in significant airway compromise, with progressive airway obstruction being responsible for a significant proportion of the morbidity and mortality associated with this condition. Hearing loss is a universal finding in MPS, with a third of patients suffering with severe profound hearing loss. There is an unmet need for strong clinical evidence to guide treatment of head, neck and respiratory disease in MPS disorders. A Core Outcome Set (COS) describes the minimum outcome data that should be measured in a clinical study for a particular condition. The lack of an agreed COS for MPS II in general, and specifically head, neck and respiratory disease, makes comparison between studies difficult. There is also a lack of information detailing patient and parent perspectives on the MPS disorders. The ideal COS for head, neck and respiratory disease associated with MPS II would combine both patient/parent and clinician opinion and could be used in the design of all subsequent clinical studies. Following literature review the investigators have created a list of outcomes previously reported for qualitative and quantitative studies investigating head, neck and respiratory disease in MPS II. For the proposed research the investigators will seek opinions of patients, parents, clinicians and scientists to rate these outcomes via the Delphi method. Outcomes scored highest by patients, parents, clinicians and scientists will form a COS for head, neck and respiratory disease in MPS II. The development of a COS can help limit variability outcomes in studies investigating different interventions in MPS II.

NCT ID: NCT05825131 Not yet recruiting - Clinical trials for Sanfilippo Syndrome Type C

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

MPSIIIC
Start date: June 10, 2024
Phase:
Study type: Observational [Patient Registry]

This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.