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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute LeukemiaDisease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RecurrenceRecruitingPhase 12026-10-09NCT06376162
A Multicenter Study to Evaluate Next-Generation Sequencing (NGS) Testing and Monitoring of B-Cell Recovery to Guide Management Following Chimeric Antigen Receptor T-cell (CART) Induced Remission in Children and Young Adults With B Lineage Acute Lymph...Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmRecruitingNot applicable2026-10-09NCT05621291
A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 MutationHemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute biphenotypic leukemia, acute leukemia of ambiguous lineage, acute myeloid leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, mixed phenotype acute leukemia, myeloid leukemia, neoplasmRecruitingPhase 1 / Phase 22026-10-09NCT04065399
Safety of MOON101 for the Treatment of Peanut AllergyHypersensitivity, Immediate, Nut and Peanut Hypersensitivity, Peanut Hypersensitivity, food allergy, immune system disorderActive, not recruitingPhase 12026-10-05NCT07580898
Post-Approval Study of PALMAZ MULLINS XD™ in Treating Pulmonary Artery StenosisArterial Occlusive Diseases, Stenosis, Pulmonary Artery, cardiovascular disorder, vascular disorderRecruitingNot stated2026-10-02NCT07122518
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIRecruitingPhase 22026-10-02NCT07047144
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille SyndromeAbnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorderActive, not recruitingPhase 32026-10-02NCT05035030
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 32026-09-29NCT06914609
Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell TransplantB-cell neoplasm, Burkitt Leukemia, Burkitt lymphoma, CD4+/CD56+ hematodermic neoplasm, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Virus Infections, Epstein-Barr virus infection, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Mast Cell Activation Disorders, Mastocytosis, Systemic, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Skin and Connective Tissue Diseases, Tumor Virus Infections, acute biphenotypic leukemia, acute leukemia, acute leukemia of ambiguous lineage, acute myeloid leukemia, acute undifferentiated leukemia, bone marrow disorder, hematologic disorder, hematopoietic and lymphoid system neoplasm, hereditary disease, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, malignant histiocytosis, mast cell leukemia, mastocytosis, mixed phenotype acute leukemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts-1, myeloid leukemia, myeloproliferative neoplasm, neoplasm, skin disorder, skin neoplasm, viral infectious diseaseActive, not recruitingPhase 22026-09-25NCT03779854
Efficacy and Safety of 186 mcg of OPN-375 Nasal Spray Twice a Day (BID) in Adolescents With Chronic Rhinosinusitis Without Nasal PolypsChronic Rhinosinusitis Without Nasal PolypsRecruitingPhase 32026-09-18NCT06850805
Quantra QPlus in Pediatric Cardiovascular ProceduresBlood Loss, Surgical, Hemorrhage, Intraoperative Complications, Pathologic Processes, Pathological Conditions, Signs and SymptomsNot yet recruitingNot stated2026-09-17NCT07597278
Reference Range Study for the Quantra System With the QPlus Cartridge in Pediatric PatientsEmbolism and Thrombosis, cardiovascular disorder, thrombotic disease, vascular disorderNot yet recruitingNot stated2026-09-17NCT07447713
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, blood platelet disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired)RecruitingPhase 32026-09-14NCT07115004
CNP-103 in Adolescent and Adult Subjects Ages 12-35 With Recently Diagnosed (Within 6 Months) Stage 3 Type 1 Diabetes (T1D)Nutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingPhase 1 / Phase 22026-09-11NCT06783309
BOND: Direct Breastfeeding to Enhance Maternal and Infant Health in Congenital Heart DiseaseCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorderRecruitingNot applicable2026-09-10NCT07582848
The HOPE Biobank Resource (BMT CTN 2402 HOPE)Bone Marrow Failure, Bone Marrow Failure Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited hemoglobinopathyRecruitingNot stated2026-08-27NCT07227155
A Phase 3 Trial to Compare IV BCV Versus IV CDV for Treatment of Adenovirus Infection After Allo-HCTDNA Virus Infections, Infections, adenoviridae infectious disease, viral infectious diseaseRecruitingPhase 32026-08-10NCT07387367
Illuminate: A Clinical Study Evaluating CAR T Immune Cell Therapy (BCB-276) for Patients With Diffuse Intrinsic Pontine Glioma (DIPG).Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, brain stem glioma, brainstem cancer, central nervous system cancer, central nervous system disorder, central nervous system neoplasm, childhood malignant neoplasm, diffuse intrinsic pontine glioma, epithelial neoplasm, glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-08-04NCT07680439
NRSTS2021, A Risk Adapted Study Evaluating Maintenance Pazopanib, Limited Margin, Dose-Escalated Radiation Therapy and Selinexor in Non-Rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Metastatic Dermatofibrosarcoma Protuberans, Neoplasms by Histologic Type, Neoplasms, Adipose Tissue, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, Urogenital Abnormalities, Urogenital Diseases, Well Differentiated Liposarcoma, angiosarcoma, clear cell sarcoma, connective tissue neoplasm, dermatofibrosarcoma protuberans, disorder of sexual differentiation, endocrine system disorder, epithelioid hemangioendothelioma, extraskeletal myxoid chondrosarcoma, fibrosarcoma, glomus tumor, gonadal disorder, granular cell cancer, granular cell tumor, hemangioendothelioma, hemangioma, histiocytoma, Angiomatoid fibrous, inflammatory leiomyosarcoma, intimal sarcoma, leiomyosarcoma, liposarcoma, low grade fibromyxoid sarcoma, malignant myoepithelioma, malignant perineurioma, mixed neoplasm, myoepithelial tumor, myomatous neoplasm, myxofibrosarcoma, myxoid liposarcoma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurofibroma, neurofibrosarcoma, neuromuscular disease, osteosarcoma, peripheral nervous system disorder, peripheral nervous system neoplasm, sarcoma, synovial sarcoma, undifferentiated (embryonal) sarcoma, vascular cancerRecruitingPhase 1 / Phase 22026-08-03NCT06239272
Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid LeukemiaHemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute biphenotypic leukemia, acute leukemia of ambiguous lineage, acute myeloid leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, myeloid leukemia, neoplasmRecruitingPhase 12026-08-03NCT06177067
The REPLACE Registry for Cholbam® (Cholic Acid)Abnormalities, Multiple, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Peroxisomal Disorders, Urogenital Diseases, Zellweger spectrum disorders, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of bile acid synthesis, inborn errors of metabolism, kidney disorder, liver disorder, metabolic disease, nervous system disorder, urinary system disorderActive, not recruitingNot stated2026-07-31NCT03115086
Cerliponase Alfa Observational Study in the USCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative diseaseActive, not recruitingNot stated2026-06-30NCT04476862
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingPhase 1 / Phase 22026-06-04NCT07252050
HER2-specific Chimeric Antigen Receptor (CAR) T Cells for Children With EpendymomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, ependymoma, epithelial neoplasm, glioma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 12026-06-02NCT04903080
Asthma Navigator Intervention to Improve Health Equity in ChildrenAsthma in ChildrenActive, not recruitingNot applicable2026-05-22NCT06513832