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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)Bone Marrow Failure Disorders, Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute lymphoblastic leukemia, acute myeloid leukemia, anemia, aplastic anemia, autoimmune disease, bone marrow disorder, chronic myeloid leukemia, disease, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, inborn errors of metabolism, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myeloid leukemia, neoplasm, non-Hodgkin lymphomaRecruitingNot stated2026-07-06NCT01351545
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-06-01NCT05337553
QUELIMMUNE (SCD-PED) PediAtric SurVeillance REgistryFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Inflammation, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, acute kidney injury, kidney disorder, kidney failure, urinary system disorderRecruitingNot stated2026-05-14NCT06517810
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular DystrophyCongenital, 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 invitationNot stated2026-05-04NCT06270719
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical PracticeCongenital, 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 invitationNot stated2025-09-10NCT06606340
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic MalignanciesB-cell neoplasm, CD4+/CD56+ hematodermic neoplasm, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Neoplasms by Histologic Type, Neoplasms by Site, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Skin and Connective Tissue Diseases, T-cell non-Hodgkin lymphoma, acute biphenotypic leukemia, acute lymphoblastic leukemia, acute myeloid leukemia, acute undifferentiated leukemia, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, malignant histiocytosis, mixed phenotype acute leukemia, myelodysplastic syndrome, neoplasm, skin disorder, skin neoplasmRecruitingPhase 12024-12-06NCT05476770