Clinical trials · California
Genetic Disease clinical trials in California
We’re tracking 24 recruiting genetic disease studies with a site in California — including Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- NCT05432349Recruiting
Rett syndrome genetic registry for MECP2 changes
This trial is a registry that collects health and genetic information from people with Rett syndrome. It may help researchers better understand how MECP2 loss-of-function changes lead to symptoms and how to support patients.
San Diego, CaliforniaAges birth–99 years - NCT06306521Recruiting
Newborn screening for hundreds of genetic diseases by genome sequencing
This research study tests a new way to screen newborns for hundreds of genetic diseases using a sample of their blood or saliva. It aims to find conditions early so your baby can get the right care sooner.
San Diego, CaliforniaAges 1 day–4 weeks - NCT04208529Enrolling by invitationPhase 3
Long-term follow-up after receiving CTX001 infusion
This study follows people for a long time after they received CTX001 in an earlier (parent) study. It helps researchers learn about long-term safety and outcomes of the treatment.
Palo Alto, CaliforniaAges 2 years+ - NCT02450851Recruiting
Find answers for undiagnosed medical conditions using genetic testing
This study helps look for causes of health problems when no one has been able to give a diagnosis. It tests and shares medical and genetic information (with privacy protections) so researchers can better match findings to possible causes.
Los Angeles, CaliforniaAges 1 month–100 years - NCT03385876Enrolling by invitation
Rapid whole genome sequencing for children with suspected genetic illness
This study quickly reads a child’s DNA using whole genome sequencing to look for genetic causes of symptoms. It may help families and doctors better understand an underlying genetic condition and guide medical care.
San Diego, CaliforniaAges Any age - NCT04994015RecruitingTeam says not enrolling
Genetic registry for people with Parkinson’s
This study builds a genetics “registry” of people diagnosed with Parkinson’s disease who agree to genetic testing. Your information may help researchers understand why Parkinson’s happens and improve future treatments.
La Jolla, CaliforniaAges 18 years+ - NCT03277365Recruiting
Share your 23andMe data with a digital genetics study
This study tests a digital platform that uses people’s existing genetic information to support genetic research. If you are an adult and willing to share your 23andMe results from an Apple phone, you can likely participate.
La Jolla, CaliforniaAges 18 years+ - NCT05848271Recruiting
Natural history study for people with HPDL gene changes
This study follows people who have an HPDL gene change to better understand how symptoms and health progress over time. It may help clinicians learn more about the condition and plan future treatments.
San Diego, CaliforniaAges Any age - NCT05552157RecruitingPhase 2/Phase 3
Study of treatments for early onset Alzheimer's caused by genetic mutations
This trial tests potential treatments for people who are at risk for or have early onset Alzheimer's disease due to a specific genetic mutation. It aims to find treatments that could slow or prevent symptoms.
La Jolla, CaliforniaAges 18 years+ - NCT06647498RecruitingPhase 2/Phase 3
Study of a treatment for early-onset Alzheimer's caused by a gene change
This study tests a potential disease-modifying treatment for people who have a specific gene mutation that causes early-onset Alzheimer's disease. The goal is to see if the treatment can delay or prevent symptoms.
La Jolla, CaliforniaAges 18 years+ - NCT06555965Recruiting
STXBP1 and SYNGAP1 natural history study
This study follows people with STXBP1 or SYNGAP1 gene changes over time to learn more about how these conditions affect the body and mind. It does not test a treatment but helps researchers understand the natural course of the disorder.
Palo Alto, CaliforniaAges Any age - NCT06935253Recruiting
AI to Improve Cardiology Care
This trial tests whether large language models (a type of AI that understands human language) can help cardiologists give better care. If you are a qualified cardiologist who currently treats patients, you may be eligible.
Palo Alto, CaliforniaAges 18 years+ - NCT02917460Recruiting
Join a children’s genomic sample and data library
This study collects and stores DNA samples and related health information from people of all ages. It helps researchers understand childhood diseases better and may support future discoveries and treatments.
San Diego, CaliforniaAges Any age - NCT05589714Recruiting
Study your rare eye gene and track retinal disease over time
This study builds a registry and tracks the natural history of rare retinal dystrophies while also testing people for disease-causing gene variants. It may help researchers better understand what causes retinal degeneration and how it progresses.
Los Angeles, CaliforniaAges 4 years+ - NCT01694940Recruiting
Mitochondrial disease registry and sample collection study
This trial builds a large database and collection of lab results, and possibly tissue samples, from people with suspected or diagnosed mitochondrial conditions. It helps researchers learn more about these disorders and mitochondrial DNA changes, which may improve future diagnosis and treatments.
Stanford, CaliforniaAges Any age - NCT05020574RecruitingPhase 2
Study how the microbiome may affect breast implant infections
This Phase 2 study looks at whether changes in the body’s “microbiome” (the normal germs in and on the body) are linked to infections after breast surgery with implants. It may help doctors better predict and prevent implant infections in people undergoing mastectomy with immediate implant placement.
San Francisco, CaliforniaAges 18 years+ - NCT06144957Enrolling by invitation
SLC13A5 deficiency natural history study (US only)
This study follows people with SLC13A5 deficiency (a rare genetic condition that affects brain development) to learn more about the disease over time. It collects health information, assessments, and samples to help understand the condition and plan future treatments.
Palo Alto, CaliforniaAges Any age - NCT04681781Enrolling by invitation
Remote study of SLC13A5 deficiency history
This study follows people with SLC13A5 deficiency to better understand how the condition changes over time, using surveys and clinical check-ins done remotely. If you (or your child) have this genetic condition, it may help researchers learn what symptoms to expect and how to track them.
Palo Alto, CaliforniaAges Any age - NCT04472338Recruiting
Prostate cancer screening for people with higher genetic risk
This study looks at prostate cancer screening for people who may be at higher risk because of a specific inherited (genetic) change. It may help your care team find the best screening approach to catch aggressive prostate cancer earlier.
Duarte, CaliforniaAges 40 years+ - NCT02910180Recruiting
Biobank study of genetic and metabolic data in brain blood vessel disease
This study collects genetic and other health information from people with brain blood vessel (cerebrovascular) problems. It aims to help researchers understand causes and patterns that could lead to better care in the future.
Los Angeles, CaliforniaAges 18 years+ - NCT04924075RecruitingPhase 2
Belzutifan for cancers linked to VHL or HIF-2α changes
This Phase 2 study tests belzutifan (MK-6482) to treat certain advanced cancers caused by specific gene or disease changes, including VHL-related tumors and HIF-2α related tumors. It may help shrink tumors or slow their growth in people whose cancer matches these genetic or disease patterns.
Los Angeles, CaliforniaAges 12 years+ - NCT05519475RecruitingPhase 2
Gene-silencing medicine trial for adults with MASH and liver scarring
This Phase 2 study tests a gene-silencing treatment to improve chronic liver disease called MASH, especially in people with certain genetic risk. You may be eligible if you have MASH with moderate-to-advanced scarring and specific test results, and if other liver causes and recent substance/alcohol issues don’t apply.
Pasadena, CaliforniaAges 18–75 - NCT01193088Recruiting
Study genetics to find who modifies Charcot-Marie-Tooth (CMT)
This study looks at DNA changes in families with Charcot-Marie-Tooth (CMT), especially a known gene duplication called PMP22. It aims to understand what other genetic factors may change how CMT shows up, which could help future diagnosis and care.
Los Angeles, CaliforniaAges Any age - NCT06991114RecruitingPhase 2
AlloNK and rituximab for relapsing rheumatologic diseases
This trial tests a new natural killer (NK) cell therapy called AlloNK given with rituximab for people with certain autoimmune diseases (rheumatoid arthritis, Sjögren's disease, inflammatory myopathies, or systemic sclerosis) that have not responded well to standard treatments. The goal is to see if this combination can better control the disease.
Chula Vista, CaliforniaAges 18 years+
Genetic Disease trials by city in California
Where these studies are running in California
Institutions with a site for the recruiting genetic disease studies listed above.
- University of California, San Francisco · 4 studies
- Cedars-Sinai Medical Center · 3 studies
- Lucile Packard Children's Hospital at Stanford · 3 studies
- University of California San Diego Health · 3 studies
- Lucile Packard Children's Hospital · 2 studies
- Rady Children's Hospital San Diego · 2 studies
- Stanford University · 2 studies
- University of California, Los Angeles (UCLA) · 2 studies
What taking part in a genetic disease study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Genetic Disease trials in other states
Common questions
- Are there genetic disease clinical trials in California?
- Yes. We're currently tracking 24 recruiting genetic disease studies with a site in California, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a genetic disease study in California?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in California to take part?
- Not necessarily. These studies have a site in California, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a genetic disease trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New genetic disease studies open in California regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.