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Genetic Disorders clinical trials in California
18 studies for Genetic Disorders are looking for participants at sites in California.
Cities in California
Study 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 Sep 24, 2026 · NCT07441876Clinical 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 Sep 18, 2026 · NCT02450851A 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 · NCT06016946RNA to the RESCUE: Evaluation to Assess the Clinical Utility of RNA Sequencing in Establishing a Genetic Diagnosis or Adjudicating a Previously Established Genetic Diagnosis.
- University of California, San Francisco, San Francisco
RecruitingObservational study
Ages 2 and older · All sexes · Updated Aug 26, 2026 · NCT07787975An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
- UCLA Mattel Children's Hospital, Los Angeles
RecruitingPhase 3
24 Hours – 7 Months · Men · Updated Aug 12, 2026 · NCT06255782A 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 Aug 11, 2026 · NCT07555483Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
- University of California Irvine, Orange
RecruitingPhase 2
Ages 18 and older · All sexes · Updated Aug 5, 2026 · NCT07224360Prospective 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 Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.