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Neuronal Ceroid Lipofuscinoses Clinical Trials

A listing of Neuronal Ceroid Lipofuscinoses medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 12 clinical trials
S Sydney Crane, RN

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical …

1 - 22 years of age All Phase 1
M Mette H Thrane, Master of Public Health

Azacitidine in Combination With Low Dose Intensity Venetoclax in Patients With AML Incl. Explorative AML Profiling

Multi-center phase II study of standard azacytidine treatment (AZA; D1-D7, 75mg/m2 qd) in combination with a short duration of "low-dose" venetoclax treatment (LD-VEN; D1-D14 before CR and D1-D7 after CR, 400mg qd) per 28 days cycle for elderly/unfit (arm 1) and relapsed/refractory (arm 2) patients with acute myeloid leukemia. AZA …

18 years of age All Phase 2

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