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Genetic Disorders clinical trials in California
17 studies for Genetic Disorders are looking for participants at sites in California.
Cities in California
A Phase 3 Evaluation of Sutacimig in Glanzmann Thrombasthenia
- University of California San Diego (UC San Diego) Health - Jacobs Medical Center, La Jolla
- Children's Hospital Los Angeles, Los Angeles
- University of California Davis (UC Davis) Medical Center, Sacramento
- and 1 more
RecruitingPhase 3
Ages 12 to 70 · All sexes · Updated Nov 13, 2026 · NCT07935356Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
- University of California Irvine, Orange
RecruitingPhase 2
Ages 18 and older · All sexes · Updated Nov 3, 2026 · NCT07224360Clinical 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 Nov 3, 2026 · NCT02450851A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)
- GC2501 Study Site 100, Los Angelas
RecruitingPhase 3
Ages 18 to 80 · All sexes · Updated Oct 26, 2026 · NCT07555483Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
- UCSF Benioff Children's Hospital Oakland, Oakland
RecruitingPhase 2/3
Ages 2 to 17 · All sexes · Updated Oct 22, 2026 · NCT07441876A Long-Term Observational Study of Patients With Fucosidosis
- Children's Hospital of Orange County, Orange
RecruitingObservational study
All ages · All sexes · Updated Sep 17, 2026 · NCT07615400Friedreich 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 · NCT06016946Prospective Clinical Assessment Study in Children With Hypochondroplasia
- UCSF Benioff Children's Hospital, Oakland
RecruitingObservational study
30 Months – 16 Years · All sexes · Updated Jul 16, 2026 · NCT06410976Rett Syndrome Registry
- Children's Hospital Los Angeles, Los Angeles
- UCSF Benioff Children's Hospital, Oakland
- Rady Children's Hospital - San Diego, San Diego
RecruitingObservational study
Ages 0 to 99 · All sexes · Updated Jun 30, 2026 · NCT05432349A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
- Ronald Reagan UCLA Medical Center, Los Angeles
- University of Stanford Medical Center, Palo Alto
RecruitingPhase 1/2
Ages 1 and older · All sexes · Updated Mar 23, 2026 · NCT05130437Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
- UCSD Altman Clinical and Transalational Research Institute Building, Los Angeles
- Ronald Reagan UCLA Medical Center, Los Angeles
- Lucile Packard Children's Hospital Stanford, Stanford
RecruitingPhase 1/2
All ages · All sexes · Updated Jan 22, 2026 · NCT04159103An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
- UCLA Medical Center, Los Angeles
- Lucile Packard Children's Hospital at Stanford, Palo Alto
- Altman Clinical and Translational Research Institution, San Diego
RecruitingPhase 1/2
Ages 1 and older · All sexes · Updated Dec 23, 2025 · NCT05295433STXBP1 and SYNGAP1 Related Disorders Natural History Study
- Stanford Medicine Children's Health, Palo Alto
RecruitingObservational study
All ages · All sexes · Updated Oct 29, 2025 · NCT06555965Large Language Models To Improve the Quality of Care of Cardiology Patients
- Stanford, Palo Alto
Recruiting
Ages 18 and older · All sexes · Updated May 15, 2025 · NCT06935253Natural History Study of Patients with HPDL Mutations
- Eun Hae Lee, San Diego
RecruitingObservational study
All ages · All sexes · Updated Mar 30, 2025 · NCT05848271An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing
- Rady Children's Hospital San Diego, San Diego
RecruitingHealthy volunteers welcome
1 Day – 28 Days · All sexes · Updated Mar 12, 2024 · NCT06306521Rady Children's Institute Genomic Biorepository
- Rady Pediatric Genomics & Systems Medicine Institute, San Diego
RecruitingHealthy volunteers welcome
All ages · All sexes · Updated Dec 12, 2022 · NCT02917460From ClinicalTrials.gov, data retrieved Nov 15, 2026. Each study sets its own eligibility; the study team decides who can join.