Necker-Enfants Malades Hospital | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis	Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Mast Cell Activation Disorders, Mastocytosis, Systemic, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Soft Tissue Neoplasms, acute myeloid leukemia, bone marrow disorder, chronic myelomonocytic leukemia, connective tissue disorder, connective tissue neoplasm, cutaneous mastocytosis, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, leukemia, maculopapular cutaneous mastocytosis, mast cell leukemia, mastocytosis, myelodysplastic/myeloproliferative disease, myeloid leukemia, neoplasm, skin disorder, skin neoplasm, skin pigmentation disorder, type III hypersensitivity disease	Recruiting	Phase 2	2026-09-15		NCT04996875
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome	Abnormalities, 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 disorder	Active, not recruiting	Phase 1 / Phase 2	2026-08-20		NCT05127226
Developmental and Epileptic Encephalopathy of Genetic Etiology: Natural History Through Reuse of Clinical Data	developmental and epileptic encephalopathy	Recruiting	Not stated	2025-06-29		NCT06380192