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Genetic Disorders clinical trials recruiting in the U.S.
44 studies for Genetic Disorders are recruiting participants at sites across the United States.
Genetic disorders are conditions caused by changes, or variants, in one or more genes. They can happen before birth or during your lifetime. Learn the types.
Cities with the most studies
- Washington, DC11 studies
- Houston, TX10 studies
- Philadelphia, PA9 studies
- Durham, NC8 studies
- Boston, MA7 studies
- Los Angeles, CA7 studies
- New York City, NY7 studies
- Mountain View, CA6 studies
- San Diego, CA6 studies
- Miami, FL5 studies
- Columbus, OH4 studies
- Aurora, CO4 studies
- Nashville, TN4 studies
- Baltimore, MD4 studies
- Cincinnati, OH4 studies
- Dallas, TX4 studies
- Chicago, IL4 studies
- St. Louis, MO3 studies
- Minneapolis, MN3 studies
- Orange, CA3 studies
- Ann Arbor, MI3 studies
- Atlanta, GA3 studies
- Oakland, CA3 studies
- Birmingham, AL3 studies
By state
- Alabama3 studies
- California17 studies
- Colorado6 studies
- District of Columbia3 studies
- Florida7 studies
- Georgia3 studies
- Illinois5 studies
- Indiana3 studies
- Maryland13 studies
- Massachusetts7 studies
- Michigan3 studies
- Minnesota7 studies
- Missouri4 studies
- New York8 studies
- North Carolina10 studies
- Ohio7 studies
- Pennsylvania11 studies
- South Carolina3 studies
- Tennessee5 studies
- Texas13 studies
- Wisconsin3 studies
Recently updated studies
A Phase 3 Evaluation of Sutacimig in Glanzmann Thrombasthenia
RecruitingPhase 3
Ages 12 to 70 · All sexes · Updated Nov 13, 2026 · NCT07935356Screening Protocol for Genetic Diseases of Lymphocyte Homeostasis and Programmed Cell Death
RecruitingObservational study
1 Month – 100 Years · All sexes · Updated Nov 12, 2026 · NCT00246857Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
RecruitingObservational studyHealthy volunteers welcome
2 Months – 115 Years · All sexes · Updated Nov 12, 2026 · NCT00001373Genome Medical Sequencing for Gene Discovery
RecruitingObservational study
4 Weeks – 99 Years · All sexes · Updated Nov 12, 2026 · NCT01087320Helix Research Network
RecruitingObservational studyHealthy volunteers welcome
Ages 18 and older · All sexes · Updated Nov 12, 2026 · NCT06057181Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes
RecruitingObservational studyHealthy volunteers welcome
Ages 18 to 70 · All sexes · Updated Nov 9, 2026 · NCT00001405Evaluation of Patients With Immune Function Abnormalities
RecruitingObservational studyHealthy volunteers welcome
All ages · All sexes · Updated Nov 9, 2026 · NCT00128973Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
RecruitingObservational study
7 Months – 15 Months · All sexes · Updated Nov 5, 2026 · NCT06805695Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
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
RecruitingObservational studyHealthy volunteers welcome
1 Month – 100 Years · All sexes · Updated Nov 3, 2026 · NCT02450851Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
RecruitingPhase 3
Ages 12 to 17 · All sexes · Updated Oct 26, 2026 · NCT07473843A 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)
RecruitingPhase 3
Ages 18 to 80 · All sexes · Updated Oct 26, 2026 · NCT07555483Inherited Reproductive Disorders
RecruitingObservational study
6 Weeks – 120 Years · All sexes · Updated Oct 26, 2026 · NCT01500447Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
RecruitingPhase 2/3
Ages 2 to 17 · All sexes · Updated Oct 22, 2026 · NCT07441876Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
RecruitingHealthy volunteers welcome
Ages 18 and older · All sexes · Updated Oct 16, 2026 · NCT05361811PKP2-ACM Natural History Study
RecruitingObservational study
Ages 12 and older · All sexes · Updated Oct 14, 2026 · NCT06644742Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria
RecruitingPhase 2
6 Months – 70 Years · All sexes · Updated Oct 13, 2026 · NCT03301038A Long-Term Observational Study of Patients With Fucosidosis
RecruitingObservational study
All ages · All sexes · Updated Sep 17, 2026 · NCT07615400Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
RecruitingObservational studyHealthy volunteers welcome
All ages · All sexes · Updated Sep 1, 2026 · NCT06016946Investigating Hereditary Risk In Thoracic Cancers (INHERIT)
RecruitingObservational study
Ages 18 and older · All sexes · Updated Aug 26, 2026 · NCT05587439Prospective Clinical Assessment Study in Children With Hypochondroplasia
RecruitingObservational study
30 Months – 16 Years · All sexes · Updated Jul 16, 2026 · NCT06410976Genetic and Metabolic Disease in Children
RecruitingObservational study
1 Day · All sexes · Updated Jul 2, 2026 · NCT02650622Rett Syndrome Registry
RecruitingObservational study
Ages 0 to 99 · All sexes · Updated Jun 30, 2026 · NCT05432349ARPKD Database Study
RecruitingObservational study
Up to age 18 · All sexes · Updated Jun 15, 2026 · NCT01401998A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
RecruitingObservational study
Ages 12 and older · All sexes · Updated Jun 3, 2026 · NCT07001280From ClinicalTrials.gov, data retrieved Nov 15, 2026. Each study sets its own eligibility; the study team decides who can join.