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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

Know your stage or your test results (such as EGFR, HER2 or BRCA)? Match trials to your own profile - nothing you type is saved.

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30 recruiting trials

for Rare Genetic Disease

Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) NCT07447648n/a13 sites📍 Paris📍 Caserta📍 Catania Fondazione SISA (Societa Italiana per lo Studio della Arter… Using a Speech-Generating Device to Support Communication in Rare Genetic Conditions NCT07039084NA1 site📍 Melbourne Murdoch Childrens Research Institute · Tests: Speech-generating device, Control Exome and Genome Sequencing of DNA Samples, as Required by Specific Research Projects and the Diagnostic Needs of the Gaslini Institute, to Be Performed Within the Framework of the "Joint Genomic Lab" Established Between IGG and IIT NCT07877688n/a1 site📍 Genova Istituto Giannina Gaslini Multi-Omics Genetic Investigation Strategies to Enhance the Diagnostic Workflow for Rare Diseases NCT07884227n/a1 site📍 Genova Istituto Giannina Gaslini Observational Study for Patients With Hemoglobinopathies and Rare Inherited Anemia and Covid 19 NCT04746066n/a2 sites📍 Genova📍 Genova Società Italiana Talassemie ed Emoglobinopatie Characterization and Contribution of Genome-wide DNA Methylation (DNA Methylation Episignatures) in Rare Diseases With Prenatal Onset NCT06475651n/a1 site📍 Paris Assistance Publique - Hôpitaux de Paris · Tests: Methylation analysis Rare Disease Registry Database for Movement Disorders in Southern Anhui Province NCT07838883n/a1 site📍 Wuhu First Affiliated Hospital of Wannan Medical College Precision Diagnosis and Therapy for Rare Diseases by Interpreting Non-coding Genomes NCT06775561n/a1 site📍 Bologna IRCCS Azienda Ospedaliero-Universitaria di Bologna · Tests: PARADIGM study aims to streamline the process from genomic characterization of RGD patients with ED/NMD to identificati… Genomic Uniformed-Screening Against Rare Disease In All Newborns NCT05990179NA1 site📍 New York Columbia University · Tests: Genome sequencing-based newborn screening Genetic Aortic Diseases and Cardiovascular Complications. An Observational Survey of Risk Factors. NCT07391293n/a1 site📍 Milan IRCCS Policlinico S. Donato · Tests: Journey to diagnosis, Quality of life, functional health and well-being, Awareness and self-esteem, acceptance of the c… ScATtEred Rare Disease Biobanks: a Model of Sample/Data Collection With susTainablE and Shared Criteria NCT06782230n/a1 site📍 San Donato Milanese IRCCS Policlinico S. Donato Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques NCT07336394n/a1 site📍 Beijing Chinese Academy of Medical Sciences, Fuwai Hospital Genome Medical Sequencing for Gene Discovery NCT01087320n/a1 site📍 Bethesda National Human Genome Research Institute (NHGRI) GROWing Up With Rare GENEtic Syndromes NCT04463316n/a1 site📍 Rotterdam dr. Laura C. G. de Graaff-Herder · Tests: Retrospective file studies Understanding Beta Cell Disorders Through the Study of Rare Genotypes (ENDURE) NCT06478121n/a1 site📍 Exeter University of Exeter · Tests: Data and Blood collection, MRI ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders NCT04398628n/a76 sites📍 Phoenix📍 Little Rock📍 Los Angeles American Thrombosis and Hemostasis Network Clinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid Tumors NCT05868629n/a8 sites📍 Torrance📍 Denver📍 Washington D.C. Novartis Pharmaceuticals · Tests: Non-investigational BBD Longitudinal Study of Osteogenesis Imperfecta NCT02432625n/a12 sites📍 Phoenix📍 Los Angeles📍 Wilmington Baylor College of Medicine National Exhaustive Cohort of Hereditary Stomatocytoses and Other Channelopathies Affecting the Red Blood Cell NCT04778657n/a1 site📍 Le Kremlin-Bicêtre Assistance Publique - Hôpitaux de Paris Pregnancy in Women With Rare Multisystemic Vascular Diseases: COGRare5 Study NCT04194619n/a37 sites📍 Angers📍 Bordeaux📍 Bordeaux Hospices Civils de Lyon · Tests: Questionnaire Self-questionnaire in Osteoporosis NCT07067827n/a1 site📍 Québec CHU de Quebec-Universite Laval · Tests: Self-administered questionnaire, Family tree Genetic of Intellectual Deficiency and Autism Spectrum Disorders (RaDiCo-GenIDA) NCT06871696n/a1 site📍 Paris Institut National de la Santé Et de la Recherche Médicale, … Diagnosis and Treatment of Patients With Inborn Errors of Metabolism NCT00369421n/a1 site📍 Bethesda National Human Genome Research Institute (NHGRI) PREcision Diagnostics in Rare genetIC Diseases and Tumors - Long Read Sequencing NCT06796751n/a1 site📍 Bologna IRCCS Azienda Ospedaliero-Universitaria di Bologna · Tests: DNA/RNA sequencing and bioinformatic data analysis Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases NCT06833489NA1 site📍 Marseille Assistance Publique Hopitaux De Marseille · Tests: ARN extraction from muscle biopsies Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) NCT07123155Phase 228 sites📍 Irvine📍 Gainesville📍 Atlanta Shionogi · Tests: S-606001, Placebo Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants NCT05589714n/a36 sites📍 Little Rock📍 Los Angeles📍 San Francisco Jaeb Center for Health Research The KidneyCARE (Community Access to Research Equity) Study NCT05497518n/a1 site📍 Danville Tufts Medical Center · Tests: Observational study - registry Personalized Training for People With Rare Neuromuscular Disorders NCT06708468NA5 sites📍 Bergen📍 Molde📍 Namsos Oslo University Hospital · Tests: Exercise, Usual Care Brain Aging in Phenylketonuria NCT06969209n/a1 site📍 Bern Insel Gruppe AG, University Hospital Bern
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