Clinical Research Directory

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

This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). In Part 1, the core study period is 25 weeks (approximately 6 months); followed by an open-label extension (OLE), which extends through Week 97 (approximately 18 months); and a long-term extension (LTE), which extends through Week 241 (Year 5). In Part 2, the core study period is 25 weeks in duration followed by an OLE, which extends through Week 49, and an LTE, which extends through Week 241 (Year 5). Participants with MPS IIIA will be enrolled in three planned cohorts, and additional participants with MPS IIIA may be enrolled in three optional cohorts. The study will enroll up to approximately 30 participants in total: 26 participants in Part 1 (Cohorts A1, A2, A3, B1, and B2 - not enrolling) and 4 participants in Part 2 (Cohort C1 - actively enrolling).

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with 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.