Rare Disorder clinical trials
19 active trials name this condition. Describe your situation to see which ones you may be eligible for.
By location
- Genetic Study to Determine the Cause of Birth Defects in Newborns in Texas
- A Multi-Site Leukopak Repository Providing Annotated Biospecimens for Approved Investigator-Directed Biomedical Research Initiatives
- Gene Discovery Core, The Manton Center
- Human Biospecimen Procurement Protocol: Biorepository to Support Translational Research to Identify Disease Mechanism(s)
- Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
- Genome Medical Sequencing for Gene Discovery
- Diagnosis and Treatment of Patients With Inborn Errors of Metabolism
- Direct to Patient Minimal Risk Biospecimen and Data Collection Research
- FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
- Caregiving Networks Across Disease Context and the Life Course
- Longitudinal Studies of Patient With FPDMM
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
- Evaluation of an Advanced Lower Extremity Neuroprostheses
- The Mayo Clinic Rare and Undiagnosed Disease Hackathon
- Rare and Undiagnosed Disease Research Biorepository
- Idiopathic Diseases of Man
- SLC13A5 Deficiency Natural History Study - United States Only
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
- Palliative Care Needs of Children With Rare Diseases and Their Families
Guides for people searching for a trial
- Why people get turned down — and what to do nextWhat eligibility criteria are for, what keeps people out, and where to go when the first door closes.
- How your biomarkers decide which trials you qualify forEGFR, HER2, BRAF, PD-L1 and the rest — what they are and why they increasingly gate access.
- How to search for a trial without losing your mindWhere to look, what to gather first, and how to read what you find.
- What Phase 1, 2 and 3 actually mean for youWhat each phase is testing, and how to weigh an early-phase trial against a later one.
- 10 questions to ask before you say yesTreatment, time, cost, safety and your rights. Worth bringing to a screening visit.
- You matched with a trial. What happens now?Phone screens, the screening visit, informed consent, and day one.
Showing active trials that list “Rare Disorder” as a condition. Eligibility shown on each trial is an estimate — the trial's study team makes the final decision.