Not applicableLooking for participantsNCT06921720What this trial is testingPhosphorus-31 Spectroscopy in Phosphate DiabetesWho this might be right forPhosphate DiabetesX-linked Hypophosphatemia Hospices Civils de Lyon 65
Not applicableEnrolling By InvitationNCT06938542What this trial is testingPalliative Care Needs of Children With Rare Diseases and Their FamiliesWho this might be right forTrisomy 13 SyndromeArthrogryposis Congenita Multiplex With Intestinal AtresiaAsparagine Synthetase Deficiency+16 more Children's National Research Institute 480
Not applicableStudy completedNCT06190574What this trial is testingDiagnostic Efficacy and Prognostic Value of 18F-FDG PET/CT (MR) in Pediatric Solid BlastomaWho this might be right for18F-FDGNeuroblastomaHepatoblastoma+1 more Union Hospital, Tongji Medical College, Huazhong University of Science and Technology 50
Large-scale testing (Phase 3)Study completedNCT00346918What this trial is testingSirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)Who this might be right forAutosomal Dominant Polycystic Kidney Disease (ADPKD) University of Zurich 100
Large-scale testing (Phase 3)Study completedNCT01885559What this trial is testingHALT Progression of Polycystic Kidney Disease Study BWho this might be right forKidney, Polycystic National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 486
Early research (Phase 1)Looking for participantsNCT04715191What this trial is testingInterleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid TumorsWho this might be right forLiver CancerRhabdomyosarcomaMalignant Rhabdoid Tumor+3 more Baylor College of Medicine 24
Not applicableStudy completedNCT00152126What this trial is testingChemotherapy With CD133+ Select Autologous Hematopoietic Stem Cells for Children With Solid Tumors and LymphomasWho this might be right forNeuroblastomaCentral Nervous System TumorsLymphomas+1 more St. Jude Children's Research Hospital 26
Testing effectiveness (Phase 2)Study completedNCT02038543What this trial is testingHydroxyproline Influence on Oxalate MetabolismWho this might be right forHyperoxaluria Mayo Clinic 22
Not applicableLooking for participantsNCT03193476What this trial is testingRegistry for Patients With X-Linked HypophosphatemiaWho this might be right forX-Linked Hypophosphatemia Kyowa Kirin Pharmaceutical Development Ltd 1,489
Testing effectiveness (Phase 2)Looking for participantsNCT05361720What this trial is testingGenetic Testing to Select Therapy for the Treatment of Advanced or Metastatic Kidney Cancer, OPTIC RCC StudyWho this might be right forAdvanced Clear Cell Renal Cell CarcinomaMetastatic Clear Cell Renal Cell CarcinomaStage III Renal Cell Cancer AJCC v8+1 more Vanderbilt-Ingram Cancer Center 54
Early research (Phase 1)Active Not RecruitingNCT04377932What this trial is testingInterleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid TumorsWho this might be right forLiver CancerRhabdomyosarcomaMalignant Rhabdoid Tumor+3 more Baylor College of Medicine 14
Testing effectiveness (Phase 2)Study completedNCT03819647What this trial is testingEvaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary HyperoxaluriaWho this might be right forPrimary Hyperoxaluria Biocodex 15
Not applicableLooking for participantsNCT07086989What this trial is testingCardiovascular Risk in Children With Chronic Conditions StudyWho this might be right forKidney TransplantFamilial HypercholesterolaemiaType 1 Diabetes Mellitus (T1DM)+22 more Semmelweis University 300
Post-approval studies (Phase 4)Study completedNCT01245374What this trial is testingNorditropin NordiFlex® Device Compared to the Device Previously Used by Patients or ParentsWho this might be right forGrowth Hormone DisorderGrowth Hormone Deficiency in ChildrenFoetal Growth Problem+6 more Novo Nordisk A/S 103
Testing effectiveness (Phase 2)Study completedNCT05190744What this trial is testingProbenecid (PB) to Treat Hereditary Nephrogenic Diabetes Insipidus (NDI), ADPKD Treated With Tolvaptan, and Severely Polyuric Patients With Previous Lithium AdministrationWho this might be right forAutosomal Dominant Polycystic Kidney DiseaseNephrogenic Diabetes InsipidusAcquired Nephrogenic Diabetes Insipidus+1 more Mayo Clinic 36
Testing effectiveness (Phase 2)Study completedNCT03847909What this trial is testingEvaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2Who this might be right forPrimary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 2 (PH2)Kidney Diseases+2 more Novo Nordisk A/S 35
Not applicableLooking for participantsNCT03988764What this trial is testingMonogenic Diabetes Misdiagnosed as Type 1Who this might be right forDiabetes Mellitus, Type 1Monogenic DiabetesNeonatal Diabetes+4 more McGill University Health Centre/Research Institute of the McGill University Health Centre 5,000
Large-scale testing (Phase 3)Study completedNCT02251275What this trial is testingLong Term Safety of Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney DiseaseWho this might be right forPolycystic Kidney, Autosomal Dominant Otsuka Pharmaceutical Development & Commercialization, Inc. 1,803
Not applicableStudy completedNCT03836144What this trial is testingEffect of Urine Alkalinazation on Urinary Inflammatory Markers in Patients With CystinuriaWho this might be right forCystinuriaRenal LithiasisGlomerulonephritis+1 more European Georges Pompidou Hospital 63
Testing effectiveness (Phase 2)Study completedNCT02163577What this trial is testingStudy of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)Who this might be right forX-linked Hypophosphatemia Kyowa Kirin, Inc. 52