NIH Clinical Center: Search the Studies

Search the Studies

7 Studies found from all possible combinations of your search terms. Select a protocol that you wish to review.

Protocol Number	Title	Protocol Status	Min-Max Age	Institute	Keywords
20-HG-0156	A Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Assess The Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-Onset GM2 Gangliosidosis (Tay-Sachs Disease And Sandhoff Disease)	No longer recruiting/follow-up only	2-125 Years	NHGRI	Late-Onset
20-HG-0025	A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects with GNE Myopathy	Participants currently recruited/enrolled	18-125 Years	NHGRI	Myopathy
19-M-0079	Evaluation of Novel PET Radioligands as Inflammatory Biomarkers in Rheumatoid Arthritis and Myositis	Participants currently recruited/enrolled	18-99 Years	NIMH	Myopathy
18-N-0083	ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients	No longer recruiting/follow-up only	3-5 Years	NINDS	Myopathy
11-HG-0218	A Natural History Study of Patients with GNE Myopathy and GNE-Related Diseases	Participants currently recruited/enrolled	4-80 Years	NHGRI	Myopathy
11-EI-0245	Generation of Induced Pluripotent Stem (iPS) Cell Lines from Somatic Cells of Participants with Eye Diseases and from Somatic Cells of Matched Controls	Participants currently recruited/enrolled	1-125 Years	NEI	Late-Onset
00-N-0043	Clinical and Molecular Manifestations of Inherited Neurological Disorders	Participants currently recruited/enrolled	2-125 Years	NINDS	Myopathy