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Mucopolysaccharidosis Type IIIA clinical trials

View clinical trials related to Mucopolysaccharidosis Type IIIA.

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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: NCT04201405 Active, not recruiting - Clinical trials for Mucopolysaccharidosis Type IIIA

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA

Start date: January 7, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

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.

NCT ID: NCT03612869 Active, not recruiting - Clinical trials for Mucopolysaccharidosis Type IIIA

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

AAVance
Start date: December 17, 2018
Phase: Phase 2/Phase 3
Study type: Interventional

MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

NCT ID: NCT02037880 Completed - Clinical trials for Mucopolysaccharidosis Type IIIB

Natural History Studies of Mucopolysaccharidosis III

Start date: February 2014
Phase: N/A
Study type: Observational

The purpose of this study is to assess rates of decline in motor and cognitive functional measures, and to assess potential biomarkers, in order to identify potential outcome measure appropriate for use in therapeutic clinical trials.