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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderActive, not recruitingPhase 32026-09-17NCT03911505
A Phase 2, Open-Label, Single-Arm Trial of FT819 in Participants With Lupus NephritisFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Skin and Connective Tissue Diseases, Urogenital Diseases, autoimmune disease, connective tissue disorder, glomerulonephritis, immune system disorder, kidney disorder, lupus nephritis, nephritis, systemic lupus erythematosus, urinary system disorderRecruitingPhase 22026-09-14NCT07570862
Discontinuation of Low-Dose Levothyroxine in Older Adults With Subclinical Hypothyroidismendocrine system disorder, hypothyroidism, thyroid gland disorderNot yet recruitingPhase 42026-08-26NCT07787585
ADAPT - A Patient Registry of the Real-world Use of Orenitram®lung disorder, pulmonary arterial hypertension, pulmonary hypertension, respiratory system disorderActive, not recruitingNot stated2026-04-02NCT03045029
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseEnrolling by invitationPhase 32026-02-06NCT05967351
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18Glycogen Storage Disease Type II Infantile OnsetRecruitingPhase 32026-01-22NCT04808505