Clinical trials · California
Down Syndrome clinical trials in California
We’re tracking 263 recruiting down syndrome studies with a site in California — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 3 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies in California
- 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 - NCT07216443RecruitingPhase 2
Study of orca-t for leukemia or MDS with reduced intensity chemo
This trial tests a new cell therapy, orca-t, given after a lower-intensity chemo and radiation regimen for people with acute myeloid leukemia, mixed phenotype leukemia, or myelodysplastic syndrome. It aims to help patients who cannot tolerate full-intensity transplant conditioning.
Los Angeles, CaliforniaAges 18 years+ - NCT07209462RecruitingPhase 2
MRM-3379 for Fragile X Syndrome in males
This trial tests an investigational medicine called MRM-3379 in males with Fragile X syndrome. It looks at how the medicine affects thinking and behavior over time.
Irvine, CaliforniaAges 13–45 - NCT06297941RecruitingPhase 1
Study of REM-422 for AML or high-risk MDS
This trial tests an experimental oral medication called REM-422 for people with AML (a type of blood cancer) that has not responded to treatment or has come back, or for those with high-risk MDS. The goal is to see if it is safe and effective.
Duarte, CaliforniaAges 18 years+ - NCT07181837RecruitingPhase 1/Phase 2
Testing MVX-220 for Angelman Syndrome
This study tests an experimental medicine called MVX-220 to see if it is safe and effective for people with Angelman Syndrome. It includes both children (ages 4-8) and adults (ages 18-50) who can walk independently and have stable seizure medications.
Los Angeles, CaliforniaAges 4–50 - NCT06819670RecruitingPhase 2
Preventing infantile spasms from coming back
This trial tests whether a longer course of steroids can keep infantile spasms from returning in babies who have already responded to standard treatment. It may help reduce the chance of relapse.
Los Angeles, CaliforniaAges 2 months–1.5 years - NCT07135050RecruitingPhase 1/Phase 2
Gene therapy study for Pitt Hopkins syndrome
This trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins syndrome. The therapy is given as a single injection into the fluid around the brain to help replace the missing or faulty TCF4 gene.
Oakland, CaliforniaAges 2–25 - NCT05741112Recruiting
Long COVID wearable study to track symptoms and help daily management
This study uses a wearable device to track symptoms and patterns in people with Long COVID, ME/CFS, and/or POTS. The goal is to see how wearable feedback and tracking tools may help you manage your symptoms.
La Jolla, CaliforniaAges 18 years+ - NCT02502162Recruiting
Low-dose naltrexone for complex regional pain syndrome
This trial tests whether low-dose naltrexone can reduce symptoms of complex regional pain syndrome (CRPS), a long-lasting pain condition in an arm or leg. It may help by targeting pain signaling, but you must meet specific CRPS diagnosis and medication rules to join.
Palo Alto, CaliforniaAges 18–70 - NCT07020819RecruitingPhase 3
Study of tanruprubart for people with Guillain-Barré syndrome
This trial tests a new medicine, tanruprubart (also called ANX005), for people with Guillain-Barré syndrome. It aims to see if the drug can help improve recovery when given soon after symptoms start.
Carlsbad, CaliforniaAges 12–85 - NCT06874855RecruitingPhase 4
Study of sleep medication for delayed sleep phase syndrome
This trial tests whether a sleep medication called lemborexant can help people with delayed sleep phase syndrome fall asleep earlier. It may improve sleep timing and reduce the impact on daily life.
San Francisco, CaliforniaAges 18 years+ - NCT06872125RecruitingPhase 3
Testing zorevunersen for Dravet syndrome
This study tests an experimental drug called zorevunersen for people with Dravet syndrome. It aims to see if the drug can reduce seizures and improve safety.
San Francisco, CaliforniaAges 2–17 - NCT06850038Recruiting
Long-term study of odevixibat in Alagille syndrome
This study follows people with Alagille syndrome who are already taking or starting odevixibat (Bylvay). It looks at how well the medicine works and how safe it is over a long period of time.
Los Angeles, CaliforniaAges Any age - NCT06847867RecruitingPhase 2
Testing momelotinib for low-risk myelodysplastic syndrome
This study tests a new drug, momelotinib, for people with a low-risk form of myelodysplastic syndrome (MDS) who need red blood cell transfusions and have already tried one other treatment (like ESAs or luspatercept). The goal is to see if momelotinib can help reduce transfusion needs.
Duarte, CaliforniaAges 18 years+ - NCT06846606RecruitingPhase 1
Study of AUTX-703 for advanced blood cancers
This trial tests an experimental drug, AUTX-703, for people with certain blood cancers (AML or MDS) that have returned or not improved after other treatments. The goal is to see if the drug is safe and helps control the disease.
Duarte, CaliforniaAges 18 years+ - NCT06840496RecruitingPhase 3
Study of oral NA-921 vs placebo for girls and women with Rett syndrome
This trial tests an oral medication called NA-921 in females with Rett syndrome to see if it works better than a placebo. It may be an option for those who have a confirmed MECP2 gene mutation and stable seizure patterns.
La Jolla, CaliforniaAges 5–20 - NCT06778070Recruiting
Pelvic pain study testing yoga therapy for relief
This trial tests whether yoga therapy can help reduce chronic pelvic pain in women and non-binary individuals over 6 months. You'll join online yoga classes and see if they improve your pain and daily life.
San Francisco, CaliforniaAges 18 years+ - NCT06747949RecruitingPhase 3
Long term extension study of dazodalibep for Sjogren's syndrome
This study gives continued access to the study drug dazodalibep for people with Sjogren's syndrome who completed a prior phase 3 study. The goal is to see how safe and effective the medication is over a longer period.
Covina, CaliforniaAges 18 years+ - NCT06726785Recruiting
Gene changes in nasal cells of RLS patients on long-term dopamine agonist
This study looks at changes in a gene (DRD2) in nasal cells of people with Restless Legs Syndrome who are taking a dopamine agonist medication for a long time. It aims to understand how this gene might be involved in RLS.
San Francisco, CaliforniaAges 18–60 - NCT06716411Recruiting
Acupuncture for Gulf War Illness relief
This study tests whether acupuncture can help relieve Gulf War Illness symptoms like fatigue, mood or thinking problems, and muscle or joint pain. You may be able to join if you were deployed to the Gulf region and have had these symptoms for more than 6 months.
Los Angeles, CaliforniaAges 18 years+ - NCT06679855RecruitingPhase 3
Milrinone to prevent heart problems after PDA closure
This trial tests if giving milrinone before surgery to close a common heart vessel (PDA) can prevent heart issues in premature babies. It may help very premature infants who need surgery for a large PDA.
San Diego, CaliforniaAges Up to 3 months - NCT06669949Recruiting
Studying the natural history of SPLIS disease
This study collects health information from people with SPLIS to better understand the condition over time. It may include your siblings if they also have SPLIS, and data from patients who have passed away.
San Francisco, CaliforniaAges Any age - NCT06668922Recruiting
Diet and irritable bowel syndrome study
This study compares how people with and without irritable bowel syndrome (IBS) react to different foods. It may help find which foods trigger symptoms and which are safe to eat.
Los Angeles, CaliforniaAges 18–75 - NCT06641414RecruitingPhase 3
Lisaftoclax with azacytidine for higher-risk myelodysplastic syndrome
This trial tests a new drug called lisaftoclax combined with a standard treatment, azacytidine, for people newly diagnosed with higher-risk myelodysplastic syndrome (MDS). The goal is to see if the combination helps control the disease better than what's currently available.
Los Angeles, CaliforniaAges 18 years+
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.
- Children's Hospital of Orange County · 3 studies
- University of California, San Francisco · 3 studies
- Cedars-Sinai Medical Center · 2 studies
- City of Hope · 2 studies
- Stanford University · 2 studies
- Alethios Inc
- Amnova Clinical Research, LLC
- Ascentage Investigative Site
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 263 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.