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Rare Diseases clinical trials in Chicago, IL

8 studies for Rare Diseases are looking for participants at sites in and around Chicago (within about 30 miles). Open a study to see who can join and how to contact the study team.

Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network

RecruitingObservational studyHealthy volunteers welcome
  • Lurie Children s Hospital, Chicago
1 Month – 100 Years · All sexes · Updated Sep 18, 2026 · NCT02450851

International Rare Brain Tumor Registry

RecruitingObservational study
  • Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
Up to age 45 · All sexes · Updated Aug 7, 2026 · NCT05697874

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

RecruitingObservational study
  • Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago
Ages 6 and older · All sexes · Updated Jul 2, 2026 · NCT07127978

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

RecruitingPhase 1/2
  • Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
30 Months · All sexes · Updated Apr 17, 2026 · NCT04998396

MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)

RecruitingObservational study
  • MPN Research Foundation, Chicago
Ages 18 and older · All sexes · Updated Jan 23, 2026 · NCT07362225

CCHS Secure Health-hub Advancing Research Efforts (CCHS SHARE)

RecruitingObservational study
  • Ann & Robert H Lurie Children's Hospital of Chicago, Chicago
All ages · All sexes · Updated Jul 16, 2025 · NCT06554275

The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis

RecruitingPhase 2
  • Ann and Robert H. Lurie Childrens Hospital of Chicago, Chicago
  • Northwestern University Feinberg School of Medicine, Chicago
Ages 12 and older · All sexes · Updated Jul 1, 2025 · NCT04372615

Pyrimethamine, Sulfadiazine, and Leucovorin in Treating Patients With Congenital Toxoplasmosis

RecruitingPhase 4
  • University of Chicago, Chicago
All ages · All sexes · Updated May 14, 2009 · NCT00004317

About Rare Diseases

A rare disease is one that affects fewer than 200,000 people in the United States. There are nearly 7,000, affecting more than 25 million Americans.

More about Rare Diseases on MedlinePlus

Rare Diseases trials in other cities

Other studies in Chicago

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.