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13 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
001681-CHInvestigations of Individuals with MEHMO Syndrome or eIF2-Pathway Related ConditionsParticipants currently recruited/enrolled0-100 YearsNICHDX-Linked
001505-NA Phase 1/2a, Randomized, Double-Blind, Placebo-Controlled, First In-Patient Study of AJ201 to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Adults With Spinal and Bulbar Muscular Atrophy (SBMA)Participants currently recruited/enrolled18-120 YearsNINDSX-Linked
000404-CA Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects with VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) SyndromeParticipants currently recruited/enrolled18-75 YearsNCI X-Linked
000186-IPart B- Phase I/II, Non-randomized, Multicenter, Open-label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients with X-Linked Chronic Granulomatous DiseaseEnrolling by Invitation3-12 YearsNIAIDX-Linked
18-N-0083ASPIRO: 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) PatientsNo longer recruiting/follow-up only3-5 YearsNINDSX-Linked
15-I-0008A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients with X-linked Chronic Granulomatous Disease (CGD)Completed Study; data analyses ongoing3-65 YearsNIAIDX-Linked
15-EI-0038A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked RetinoschisisNo longer recruiting/follow-up only18-125 YearsNEI X-Linked
11-I-0007Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age with X-Linked Severe Combined ImmunodeficiencyParticipants currently recruited/enrolled2-40 YearsNIAIDX-Linked
05-I-0213Screening and Baseline Assessment of Patients with Abnormalities of Immune FunctionParticipants currently recruited/enrolled0-125 YearsNIAIDX-Linked
03-EI-0033X-Linked Juvenile Retinoschisis - Clinical and Molecular StudiesParticipants currently recruited/enrolled0-125 YearsNEI X-Linked
02-CH-0311Investigations into Inborn Errors of Cholesterol Synthesis and Related DisordersParticipants currently recruited/enrolled0-125 YearsNICHDX-Linked
00-HG-0209Molecular and Clinical Studies of Primary Immunodeficiency DiseasesNo longer recruiting/follow-up only3-125 YearsNHGRIX-Linked
94-I-0073Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and GranulocytesParticipants currently recruited/enrolled18-70 YearsNIAIDX-Linked