Not applicableStudy completedNCT03251989What this trial is testingRare CNS Tumors Outcomes &RiskWho this might be right forHigh Grade MeningiomaEpendymomaMedulloblastoma+2 more National Cancer Institute (NCI) 326
Not applicableLooking for participantsNCT04463316What this trial is testingGROWing Up With Rare GENEtic SyndromesWho this might be right forPrader-Willi SyndromePWS-like SyndromeSilver Russel Syndrome+30 more dr. Laura C. G. de Graaff-Herder 600
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Not applicableLooking for participantsNCT06065852What this trial is testingNational Registry of Rare Kidney DiseasesWho this might be right forAdenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis+81 more UK Kidney Association 35,000
Not applicableLooking for participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000
Testing effectiveness (Phase 2)Not Yet RecruitingNCT06804655What this trial is testingPharmacoscopy for Patients With Refractory Primary Brain TumorsWho this might be right forBrain (Nervous System) CancersGlioblastomaGlioma+5 more University of Zurich 40
Not applicableLooking for participantsNCT06573723What this trial is testingInstitutional Registry of Rare DiseasesWho this might be right forRare DiseasesAmyloidosisSarcoidosis+23 more Hospital Italiano de Buenos Aires 380
Not applicableLooking for participantsNCT05259605What this trial is testingObservational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Using cIMPACT-NOW and 2021 WHO ClassificationWho this might be right forGliomaGlioneuronal TumorChoroid Plexus Tumor+10 more European Organisation for Research and Treatment of Cancer - EORTC 1,650
Testing effectiveness (Phase 2)UnknownNCT00004659What this trial is testingPhase II Randomized Study of Stereotactic Radiosurgery Plus Fractionated Whole-Brain Radiotherapy Vs Fractionated Whole-Brain Radiotherapy Alone for Multiple Primary or Metastatic Brain TumorsWho this might be right forBrain Neoplasms National Institute of Neurological Disorders and Stroke (NINDS) 40
Large-scale testing (Phase 3)Study completedNCT00790400What this trial is testingEfficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)Who this might be right forTuberous Sclerosis Complex (TSC)Lymphangioleiomyomatosis (LAM) Novartis Pharmaceuticals 118
Early research (Phase 1)Looking for participantsNCT04541082What this trial is testingPhase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System NeoplasmsWho this might be right forCentral Nervous System NeoplasmsGlioblastomaGliosarcoma, Adult+20 more Jazz Pharmaceuticals 102
Not applicableStudy completedNCT05199402What this trial is testingClinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare DiseasesWho this might be right forTuberous SclerosisEpilepsy Imagine Institute 10