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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
DFT383 in Pediatric Participants With Nephropathic CystinosisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Diseases, cystinosis, hereditary disease, inborn errors of metabolism, kidney disorder, lysosomal storage disease, metabolic disease, nephropathic cystinosis, renal tubular transport disease, urinary system disorderRecruitingPhase 1 / Phase 22026-09-22NCT06910813
Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Diseases, disorder of glycogen metabolism, glycogen storage disease I, glycogen storage disease Ib, glycogen storage disease due to GLUT2 deficiency, glycogen storage disease due to glucose-6-phosphatase deficiency type IA, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, kidney disorder, metabolic disease, renal tubular transport disease, urinary system disorderRecruitingNot stated2026-06-01NCT07459582
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene TherapyBone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Fanconi anemia complementation group A, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, kidney disorder, metabolic disease, renal tubular transport disease, urinary system disorderActive, not recruitingNot stated2026-04-13NCT04437771
Gene Therapy for Fanconi Anemia, Complementation Group ABone Marrow Failure Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fanconi anemia complementation group A, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Urogenital Diseases, anemia, bone marrow disorder, hematologic disorder, hereditary disease, kidney disorder, renal tubular transport disease, urinary system disorderActive, not recruitingPhase 22025-12-22NCT04248439
A Study Comparing the Necessity of a Second Transurethral Resection in High-Risk Non-Muscle-Invasive Bladder Cancer Patients With Negative Results From Post-Initial Resection Urine Genome-Wide Low-Depth SequencingCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Non-Muscle Invasive Bladder Neoplasms, Urogenital Diseases, Urogenital Neoplasms, benign urinary system neoplasm, carcinoma, epithelial neoplasm, hereditary disease, kidney disorder, neoplasm, renal tubular transport disease, urinary bladder disorder, urinary bladder neoplasm, urinary system disorderRecruitingNot applicable2025-06-25NCT07036731
The Necessity of a Second Transurethral Resection in High-risk Non-muscle-invasive Bladder Cancer Patients With Negative Urine Biomarker After Initial Transurethral ResectionCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Non-Muscle Invasive Bladder Neoplasms, Urogenital Diseases, Urogenital Neoplasms, benign urinary system neoplasm, carcinoma, epithelial neoplasm, hereditary disease, kidney disorder, neoplasm, renal tubular transport disease, urinary bladder disorder, urinary bladder neoplasm, urinary system disorderRecruitingNot applicable2024-03-01NCT06287541
Observational Follow-up Study of Haplo-identical Transplants in Fanconi DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, hereditary disease, kidney disorder, renal tubular transport disease, urinary system disorderNot yet recruitingNot stated2023-06-15NCT05903365