X-Linked Clinical Trials in Boston, Massachusetts

8 recruitingBoston, Massachusetts

Showing 18 of 8 trials

Recruiting

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

Erythropoietic Protoporphyria (EPP)X-Linked Porphyria (XLP)
Portal Therapeutics, Inc.50 enrolled19 locationsNCT07567131
Recruiting
Phase 1Phase 2

Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

X-Linked Chronic Granulomatous Disease
Ensoma15 enrolled9 locationsNCT06876363
Recruiting
Phase 1Phase 2

Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators

X-linked Myotubular Myopathy
Astellas Gene Therapies9 enrolled4 locationsNCT07052929
Recruiting

A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition

X-linked Myotubular Myopathy
Astellas Gene Therapies50 enrolled6 locationsNCT06581146
Recruiting
Phase 3

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

Erythropoietic Protoporphyria (EPP)X-Linked Protoporphyria (XLP)
Disc Medicine, Inc150 enrolled28 locationsNCT06910358
Recruiting
Phase 1Phase 2

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Gene TherapySevere Combined Immunodeficiency, X-Linked
David Williams12 enrolled4 locationsNCT03311503
Recruiting

The Myelin Disorders Biorepository Project

AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369
Recruiting

Rett Syndrome Registry

Genetic DiseaseNeurologic DisorderNervous System Diseases+7 more
International Rett Syndrome Foundation3,000 enrolled18 locationsNCT05432349