Not applicableActive Not RecruitingNCT05388370What this trial is testingPASS of Paediatric Patients Initiating SelumetinibWho this might be right forNeurofibromatosis Type 1 AstraZeneca 124
Very early researchStudy completedNCT00657202What this trial is testingRanibizumab for Neurofibromas Associated With Neurofibromatosis 1Who this might be right forNeurofibromatosis Type 1Cutaneous Neurofibromas Massachusetts General Hospital 11
Testing effectiveness (Phase 2)Active Not RecruitingNCT05309668What this trial is testingPharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PNWho this might be right forNeurofibromatosis Type 1 AstraZeneca 36
Testing effectiveness (Phase 2)Study completedNCT01673009What this trial is testingPhase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasWho this might be right forNeurofibromatosis Indiana University 36
Early research (Phase 1)Study completedNCT01031901What this trial is testingTopical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)Who this might be right forTuberous SclerosisNeurofibromatosesAngiofibroma+1 more The University of Texas Health Science Center, Houston 52
Not applicableStudy completedNCT01595139What this trial is testingMicroRNAs in Patients With Neurofibromatosis Type 1Who this might be right forGliomaNeurofibromatosis Type 1 Ann & Robert H Lurie Children's Hospital of Chicago 9
Not applicableLooking for participantsNCT07561957What this trial is testingA Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) GuidelinesWho this might be right forChronic Kidney DiseaseProteinuriaBlood Pressure Control+5 more St. James's Hospital, Ireland 80
Early research (Phase 1)Study completedNCT01903681What this trial is testingAssessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep DisturbancesWho this might be right forSleep Problems Neurim Pharmaceuticals Ltd. 16
Not applicableEnrolling By InvitationNCT03836300What this trial is testingParent and Infant Inter(X)Action Intervention (PIXI)Who this might be right forFragile X SyndromeAngelman SyndromePrader-Willi Syndrome+11 more RTI International 120
Testing effectiveness (Phase 2)Study completedNCT00470977What this trial is testingTreatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related MaculopathyWho this might be right forCoats' DiseaseIdiopathic Retinal TelangiectasiaRetinal Angiomatous Proliferation+7 more Manhattan Eye, Ear & Throat Hospital 18
Not applicableStudy completedNCT02479360What this trial is testingReliability of Functional Outcome Measures in Neurofibromatosis 1Who this might be right forNeurofibromatosis 1, NF1 Guy's and St Thomas' NHS Foundation Trust 49
Post-approval studies (Phase 4)Study completedNCT05864846What this trial is testingInvestigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis ComplexWho this might be right forTuberous Sclerosis Complex Associated Neuropsychiatric Disease Jazz Pharmaceuticals 79
Post-approval studies (Phase 4)Active Not RecruitingNCT05044819What this trial is testingAssessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral SolutionWho this might be right forLennox Gastaut SyndromeDravet SyndromeTuberous Sclerosis Complex Jazz Pharmaceuticals 154
Not applicableLooking for participantsNCT05955014What this trial is testingData Collection Protocol for Patients With Von Hippel Lindau DiseaseWho this might be right forVon Hippel Lindau Disease M.D. Anderson Cancer Center 125
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07131722What this trial is testingStudy to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type IIWho this might be right forNeurofibromatosis Type IINF2 PRG Science & Technology Co., Ltd. 25
Not applicableLooking for participantsNCT06764602What this trial is testingObservational, Retrospective, Multicenter, Nonprofit Study on the Prevalence of Renal Involvement in Pediatric Patients With Tuberous SclerosisWho this might be right forTuberous Sclerosis Complex (TSC) IRCCS Azienda Ospedaliero-Universitaria di Bologna 80
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
Not applicableStudy completedNCT00062166What this trial is testingNatural History and Management of Pancreatic Lesions in Von Hippel-Lindau DiseaseWho this might be right forVon Hippel-Lindau Disease National Cancer Institute (NCI) 340
Not applicableLooking for participantsNCT07088991What this trial is testingClinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsiaWho this might be right forNeurofibromatosis AstraZeneca 200
Not applicableLooking for participantsNCT01496625What this trial is testingNational Eye Institute Biorepository for Retinal DiseasesWho this might be right forAge-Related Macular DegenerationDiabetic RetinopathyVon Hippel-Lindau Syndrome+2 more National Eye Institute (NEI) 650