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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study Evaluating APG777 in Atopic DermatitisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypersensitivity, Immediate, Skin Diseases, Eczematous, Skin and Connective Tissue Diseases, atopic eczema, dermatitis, hereditary disease, hereditary skin disorder, immune system disorder, skin disorderActive, not recruitingPhase 22026-10-06NCT06395948
A Long-term Safety and Efficacy Study Evaluating APG777 in Atopic DermatitisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypersensitivity, Immediate, Skin Diseases, Eczematous, Skin and Connective Tissue Diseases, atopic eczema, dermatitis, hereditary disease, hereditary skin disorder, immune system disorder, skin disorderRecruitingPhase 22026-09-23NCT07003425
A Gaucher Disease Gene Therapy Trial With FLT201Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gaucher Disease, Type 1, Gaucher disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosisRecruitingPhase 32026-09-03NCT07223944
Phase 2 Study to Assess the Safety, PK, and PD of Sonefpeglutide (HM15912) in SBS-IF SubjectsDigestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Postoperative Complications, digestive system disorder, intestinal disorder, malabsorption syndrome, short bowel syndromeRecruitingPhase 22026-08-12NCT04775706
Pregnancy Exposure Registry for Vumerity (Diroximel Fumarate)CNS demyelinating autoimmune disease, autoimmune disease, autoimmune disorder of the nervous system, demyelinating disease, immune system disorder, multiple sclerosis, nervous system disorderRecruitingNot stated2026-08-11NCT05658497
A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA)autonomic nervous system disorder, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, multiple system atrophy, nervous system disorder, neurodegenerative disease, primary dysautonomia, synucleinopathyActive, not recruitingPhase 32026-07-14NCT06706622
A Study of the Effect and Safety of Sparsentan in the Treatment of Patients With IgA NephropathyFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, IgA glomerulonephritis, Male Urogenital Diseases, Urogenital Diseases, autoimmune disease, glomerulonephritis, immune system disorder, kidney disorder, nephritis, urinary system disorderActive, not recruitingPhase 32026-04-20NCT03762850
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babiescentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-03-09NCT05789758
Lysosomal Acid Lipase (LAL) Deficiency RegistryAcid Cholesteryl Ester Hydrolase Deficiency, Type 2, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Wolman disease, cholesteryl ester storage disease, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic diseaseRecruitingNot stated2026-03-06NCT01633489
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry PatientsBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingNot stated2025-11-14NCT06663358
USPIO Enhanced MR Imaging in CNS Tumours (UMIC)Ear Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Otorhinolaryngologic Neoplasms, acoustic neuroma, astrocytoma (excluding glioblastoma), cranial nerve neoplasm, cranial nerve neuropathy, epithelial neoplasm, glioma, neoplasm, nerve sheath neoplasm, nervous system disorder, neuroendocrine neoplasm, neuroepithelial neoplasm, neuroma, otorhinolaryngologic disease, peripheral nervous system neoplasm, primitive neuroectodermal tumor, retrocochlear disease, schwannoma, vestibulocochlear nerve disorderRecruitingNot applicable2024-08-27NCT06572475