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Rare Diseases clinical trials in California
10 studies for Rare Diseases are looking for participants at sites in California.
Cities in California
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
- University of California, Los Angeles, Los Angeles
- University of California, Irvine Medical Center, Orange
- Stanford University, Stanford
- and 2 more
RecruitingObservational studyHealthy volunteers welcome
1 Month – 100 Years · All sexes · Updated Oct 2, 2026 · NCT02450851A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
- The University of California, Los Angeles (UCLA), Los Angeles
RecruitingPhase 1
Ages 18 to 40 · All sexes · Updated Sep 29, 2026 · NCT07180355A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
- University of California - San Francisco, San Francisco
RecruitingPhase 1/2
Ages 18 to 65 · All sexes · Updated Sep 21, 2026 · NCT06525636Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
- UCLA Ataxia Center, Los Angeles
RecruitingObservational studyHealthy volunteers welcome
All ages · All sexes · Updated Sep 1, 2026 · NCT06016946International Rare Brain Tumor Registry
- Children's Hospital Los Angeles, Los Angeles
- University of California San Francisco, San Francisco
RecruitingObservational study
Up to age 45 · All sexes · Updated Aug 7, 2026 · NCT05697874Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
- University of California - Irvine Medical Center, Irvine
RecruitingPhase 2
Ages 18 and older · All sexes · Updated Jun 23, 2026 · NCT07123155A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
- UCSF Benioff Children's Hospital Oakland, Oakland
RecruitingPhase 1/2
30 Months · All sexes · Updated Apr 17, 2026 · NCT04998396The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis
- Children's Hospital of Orange County, Orange
- UC Irvine, Orange
- UC Davis, Sacramento
RecruitingPhase 2
Ages 12 and older · All sexes · Updated Jul 1, 2025 · NCT04372615Prospective Observational International Registry of Patients With Newly Diagnosed Peripheral T Cell Lymphoma.
- Stanford University, Stanford
RecruitingObservational study
Ages 18 and older · All sexes · Updated May 6, 2025 · NCT03964480Epilepsy Learning Healthcare System (ELHS)
- Children's Hospital Los Angeles, Los Angeles
- University of Southern California, Los Angeles
RecruitingObservational study
All ages · All sexes · Updated Aug 30, 2024 · NCT06265103From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.