National Institutes of Health: Rare Diseases trials

The registry lists 12 Rare Diseases studies with National Institutes of Health as lead sponsor. 6 are open to participants now and 0 have results posted.

Phase 2 1No phase 11(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Genetic Analysis of Uncommon Disease Presentations in Non-US PopulationsRecruitingNCT06595940
• Caregiving Networks Across Disease Context and the Life CourseRecruitingNCT05007990
• Longitudinal Studies of Patient With FPDMMRecruitingNCT03854318
• Human Biospecimen Procurement Protocol: Biorepository to Support Translational Research to Identify Disease Mechanism(s)RecruitingNCT02543996
• Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases NetworkRecruitingNCT02450851
• Diagnosis and Treatment of Patients With Inborn Errors of MetabolismRecruitingNCT00369421
• SARS-COV2 Pandemic Serosurvey in a Rare Disease PopulationCompletedNCT04609085
• COV2Base-A Rare Disease by COVID StudyCompletedNCT04564274
• Nivolumab and Ipilimumab in Treating Patients With Rare TumorsActive, not recruitingNCT02834013Phase 2
• Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue BankCompletedNCT02706639
• Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995CompletedNCT02116764
• Genetic Studies in the Amish and MennonitesCompletedNCT00359580