Clinical trials for Rare genetic disease
9 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGN/ANCT06926127Genomic Profiling of Genetic and Rare DiseasesFondazione Policlinico Universitario Agostino Gemelli IRCCS · PI: Giovanni Scambia
- RECRUITINGNCT06796751PREcision Diagnostics in Rare genetIC Diseases and Tumors - Long Read SequencingIRCCS Azienda Ospedaliero-Universitaria di Bologna · PI: Tommaso Pippucci, Biologist
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT06314490Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2AUniversity of California, San Diego · PI: Olivia Kim-McManus, M.D.
- ACTIVE NOT RECRUITINGN/ANCT05064241Overcoming Barriers to Accessing Genetic MedicineBoston Children's Hospital
- RECRUITINGN/ANCT05499091Functional Study to Indentify Genetic Etiology of Rare Diseases - ORIGINUniversity Hospital, Angers · PI: Estelle COLIN, MD-PhD
- ENROLLING BY INVITATIONNCT05236595Research for Individualized Therapeutics in Rare Genetic DiseaseMayo Clinic · PI: Margot A Cousin, Ph.D.
- RECRUITINGNCT04586075UW Undiagnosed Genetic Diseases ProgramUniversity of Wisconsin, Madison · PI: Bryn Webb, MD
- RECRUITINGNCT03967743Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic DisordersBoston Children's Hospital · PI: Monica Wojcik, MD
- RECRUITINGNCT02450851Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases NetworkNational Human Genome Research Institute (NHGRI) · PI: William A Gahl, M.D.