Clinical trials · California
Down Syndrome clinical trials in California
We’re tracking 274 recruiting down syndrome studies with a site in California — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- NCT06078553Recruiting
Natural history study of congenital myasthenic syndromes (CMS)
This study follows people with congenital myasthenic syndrome (CMS) caused by changes in certain genes (DOK7, MUSK, AGRN, or LRP4) over time. It does not test any new drug, but helps researchers understand how the condition changes and how to plan future treatments.
Sacramento, CaliforniaAges 2 years+ - NCT07277582RecruitingPhase 2/Phase 3
Study of THRV-1268 for long QT syndrome type 2
This trial tests an investigational drug called THRV-1268 in people with a specific genetic form of long QT syndrome (type 2). The goal is to see if it can safely help control the heart's electrical activity and reduce the risk of dangerous heart rhythms.
San Francisco, CaliforniaAges 15 years+ - NCT03113643RecruitingPhase 1
SL-401 with standard drugs for hard-to-treat blood cancers
This Phase 1 study tests SL-401, given with azacitidine and sometimes venetoclax, to treat acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (MDS), or blastic plasmacytoid dendritic cell neoplasm (BPDCN). It aims to find a safe dose and see whether the combination can improve blood cancer control.
Duarte, CaliforniaAges 18 years+ - NCT03339128RecruitingPhase 2
Eluxadoline for diarrhea-predominant IBS in children
This trial tests whether eluxadoline can reduce symptoms of IBS with diarrhea (IBS-D) in children ages 6 to 17. It also asks families to track symptoms with a daily diary to measure whether treatment helps over the study period.
Garden Grove, CaliforniaAges 6–17 - NCT03568669Recruiting
Testing thinking and learning in congenital central hypoventilation syndrome
This study looks at thinking and memory in people with congenital central hypoventilation syndrome (CCHS) to better understand how the condition affects the brain. It may help researchers measure changes over time and guide future care.
Los Angeles, CaliforniaAges 3–85 - NCT03587844RecruitingPhase 2
Test dosing of brentuximab for skin lymphoma
This Phase 2 study tests how brentuximab vedotin should be dosed for people with mycosis fungoides or Sezary syndrome (skin T-cell lymphoma), and also for lymphomatoid papulosis. It may help improve how well the treatment works while tracking side effects.
Stanford, CaliforniaAges 18 years+ - NCT03708900RecruitingPhase 2
Osilodrostat study for children with Cushing’s syndrome
This Phase 2 study tests how osilodrostat works in children and teens with Cushing’s syndrome—what the drug does in the body, how it affects hormones, and how safely it’s tolerated. It may help families who cannot have immediate surgery or whose surgery has already failed or is still pending.
San Francisco, CaliforniaAges 2–17 - NCT03801434RecruitingPhase 2
Ruxolitinib for people with certain eosinophil overgrowth diseases
This trial studies ruxolitinib to treat illnesses where the body makes too many eosinophils (a type of white blood cell) and symptoms or organ damage happen. It may help shrink disease activity when steroids are not enough, cannot be tolerated, or the illness comes back.
Palo Alto, CaliforniaAges 18 years+ - NCT04165109Recruiting
Down syndrome study to test memory and thinking changes
This study enrolls adults with Down syndrome to better understand how thinking and memory develop and change over time. It may help by building a “trial-ready” group and by measuring thinking in a careful, consistent way.
Orange, CaliforniaAges 25–55 - NCT04243785RecruitingPhase 1
Testing BTX-A51 for hard-to-treat AML or high-risk MDS
This Phase 1 study tests BTX-A51 in adults with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). It’s designed to see if the treatment can be given safely and to learn how it works in people whose standard treatments have not helped or are no longer an option.
Duarte, CaliforniaAges 18 years+ - NCT04256018RecruitingPhase 2
Mogamulizumab with low-dose skin radiation for lymphoma skin disease
This Phase 2 study tests mogamulizumab together with low-dose total skin electron beam radiation to treat mycosis fungoides or Sézary syndrome, two types of skin lymphoma. It may help shrink skin disease and control symptoms by combining an immune-targeting medicine with skin-focused radiation.
Stanford, CaliforniaAges 18 years+ - NCT04278404Recruiting
Drug safety study for children already on certain medicines
This study measures how certain medicines move through the body and checks their safety in children. It may help doctors better understand dosing and side effects in kids who are already receiving these treatments, or in some kids who recently tested positive for COVID-19.
Los Angeles, CaliforniaAges birth–20 years - NCT04419649RecruitingPhase 2
Elritercept for anemia in lower-risk myelodysplastic syndromes
This trial tests elritercept, a medicine meant to improve anemia (low blood count) in adults with certain lower-risk bone marrow disorders called MDS. If you qualify, the study may help reduce the need for blood transfusions or improve anemia symptoms.
Duarte, CaliforniaAges 18 years+ - NCT04507997Recruiting
Study of the natural course of Angelman syndrome
This study follows people with Angelman syndrome to understand how their symptoms and development change over time. It may help researchers better describe the condition and support future treatments.
Los Angeles, CaliforniaAges Any age - NCT04978571Recruiting
Electrical nerve stimulation for long-lasting concussion symptoms
This study tests whether a device that delivers gentle electrical signals to nerves through the skin can help children and teens with concussion symptoms that have lasted at least 3 months. You may be asked to attend clinic visits for the nerve stimulation setup if you meet the safety and scheduling requirements.
Orange, CaliforniaAges 11–18 - NCT05123755RecruitingPhase 2
Testing a new medicine in hospitalized pneumonia patients needing oxygen
This Phase 2 trial tests an investigational medicine (given in increasing doses) for hospitalized people with pneumonia who need supplemental oxygen. It aims to see if the medicine is safe and potentially helps the pneumonia process in the early hospital period.
Newport Beach, CaliforniaAges 18 years+ - NCT05127226RecruitingPhase 1/Phase 2
Test drug safety for Angelman syndrome in young children
This early-phase study tests a new experimental medicine called ION582 to see if it is safe, tolerable, and how the body processes it in people with Angelman syndrome. It may also look at early signs of how the medicine affects the brain and body, which could guide future treatments.
San Diego, CaliforniaAges birth–50 years - NCT05143996RecruitingPhase 1
First study drug for relapsed AML or MDS
This Phase 1 trial tests CLN-049 in adults whose acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) has come back or did not respond to treatment. The goal is to find out how well the drug can work and how safe it is in people with limited options.
Los Angeles, CaliforniaAges 18 years+ - NCT05204459Recruiting
MRI and eye-scan study for MS and related conditions
This trial uses brain MRI and eye imaging tests to study research markers in people with multiple sclerosis (MS) and related neurologic conditions. It may help researchers better understand these diseases and how they change over time.
Los Angeles, CaliforniaAges 18 years+ - NCT05237505RecruitingPhase 2
Sleep apnea plus COPD study to check heart and breathing effects
This Phase 2 study looks at the cardiovascular (heart/blood vessel) effects of having both sleep apnea and COPD. It may help doctors better understand who is at risk and how to manage these breathing problems together.
La Jolla, CaliforniaAges 40–79 - NCT05308264RecruitingPhase 1/Phase 2
R289 for lower-risk MDS that still needs blood support
This Phase 1/2 study tests R289 in adults with lower-risk myelodysplastic syndromes (MDS) whose disease keeps causing anemia and/or ongoing need for red blood cell transfusions. It may help reduce blood problems and treat MDS when standard options haven’t worked well enough or aren’t suitable.
Los Angeles, CaliforniaAges 18 years+ - NCT05354141RecruitingPhase 3
Tested treatment with tiny lung particles for moderate to severe ARDS
This Phase 3 trial studies an “extracellular vesicle” treatment in people with moderate to severe ARDS, a serious lung breathing problem. The goal is to see whether the treatment can improve outcomes compared with standard care.
Irvine, CaliforniaAges 18–75 - NCT05410977Recruiting
Testing blood and stool for colon cancer in Lynch syndrome
This study collects blood and stool samples to see how well they can detect colorectal cancer or advanced growths in people with Lynch syndrome (an inherited colon cancer risk) or suspected Lynch syndrome. It may help find cancer or precancer earlier, which can improve next-step decisions.
San Francisco, CaliforniaAges 18 years+ - NCT05432466RecruitingPhase 3
Celiprolol vs placebo for vascular Ehlers-Danlos heart vessel fragility
This Phase 3 study compares celiprolol (a blood vessel–related medicine) versus placebo to see which better reduces vascular risks in people with vascular Ehlers-Danlos syndrome. It requires confirmed COL3A1 genetic findings and specific imaging before starting.
Culver City, CaliforniaAges 15–64
Down Syndrome trials by city in California
Where these studies are running in California
Institutions with a site for the recruiting down syndrome studies listed above.
- City of Hope Comprehensive Cancer Center · 3 studies
- University of California, Los Angeles (UCLA) · 3 studies
- University of California, San Francisco · 3 studies
- Cedars-Sinai Medical Center · 2 studies
- Rady Children's Hospital San Diego · 2 studies
- Stanford University · 2 studies
- Children's Hospital Los Angeles
- CHOC Children's Hospital
What taking part in a down syndrome 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.
Down Syndrome trials in other states
Common questions
- Are there down syndrome clinical trials in California?
- Yes. We're currently tracking 274 recruiting down syndrome 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 down syndrome 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 down syndrome 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 down syndrome 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.