Clinical trials · California
Down Syndrome clinical trials in California
We’re tracking 273 recruiting down syndrome studies with a site in California — including Phase 1, Phase 1/Phase 2, Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- NCT07593391RecruitingPhase 3
NNZ-2591 Treatment for Children with Phelan-McDermid Syndrome
This study tests whether a medication called NNZ-2591 can help children with Phelan-McDermid syndrome, a rare genetic condition that affects brain development and learning. Researchers want to see if this drug can improve how children with this condition develop and learn.
Glendale, CaliforniaAges 3–12 - 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 - NCT07281079RecruitingPhase 3
Testing NNZ-2591 for children with Phelan-McDermid syndrome
This trial tests a new medicine, NNZ-2591, to see if it helps children with Phelan-McDermid syndrome. It is for children aged 3 to 12 who have a specific genetic change (SHANK3) and weigh at least 10 kg.
Glendale, CaliforniaAges 3–12 - NCT06660394RecruitingPhase 3
LP352 for Dravet syndrome in children and adults
This study tests a new medicine called LP352 for children and adults with Dravet syndrome. It compares the study drug to a placebo (a pill with no medicine) to see if it helps reduce seizures.
Los Angeles, CaliforniaAges 2–65 - NCT06776783RecruitingPhase 3
Testing a new surfactant for breathing problems in premature babies
This study tests a new medicine called APC-0101, a type of surfactant, for premature babies with breathing problems (respiratory distress syndrome). It may help their lungs work better and reduce the need for more breathing support.
La Jolla, CaliforniaAges 1 hour–1 day - NCT05672901Recruiting
Silicone gel vs estrogen cream for menopause discomfort
This study compares a silicone gel with an estrogen vaginal cream to treat genital and urinary symptoms caused by menopause. It may help you see which treatment works better and is more comfortable for your symptoms.
Laguna Hills, CaliforniaAges 18 years+ - NCT06196203RecruitingPhase 2
Study of AK117 with Azacitidine for High-Risk MDS
This trial tests a new drug, AK117, combined with azacitidine for people newly diagnosed with high-risk myelodysplastic syndromes (MDS), a blood disorder. The goal is to see if the combination can help improve outcomes.
Los Angeles, CaliforniaAges 18 years+ - NCT06420297Enrolling by invitationPhase 3
Carbetocin nasal spray for Prader-Willi syndrome eating behaviors (open-label extension)
This is a long-term follow-up study for people who completed a previous study of carbetocin nasal spray for hyperphagia (extreme hunger) in Prader-Willi syndrome. The trial gives everyone the study drug openly to see if it continues to help manage eating behaviors over a longer period.
Orange, CaliforniaAges 5–30 - 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+ - NCT07727538RecruitingPhase 3
Olezarsen for children with familial chylomicronemia syndrome
This trial tests a drug called olezarsen in children with a genetic condition that causes extremely high triglycerides. It aims to lower triglyceride levels and prevent pancreatitis.
San Francisco, CaliforniaAges 2–17 - 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.
Orange, CaliforniaAges Up to 3 months - 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 - 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.
Los Angeles, CaliforniaAges 2–17 - NCT07742735RecruitingPhase 3
Study of apraglutide for short bowel syndrome with intestinal failure
This study tests a new medicine called apraglutide, given as a shot, to see if it can help adults with short bowel syndrome and intestinal failure reduce their need for intravenous nutrition (parenteral support). The study compares apraglutide to a placebo (a shot with no medicine) to check if it is safe and effective.
Los Angeles, CaliforniaAges 18 years+ - NCT06577441RecruitingPhase 2
Adding enasidenib to usual care for higher-risk MDS with IDH2 mutation
This trial tests whether adding enasidenib (a targeted pill) to the standard chemotherapy (cedazuridine-decitabine) works better than the chemotherapy alone for people with higher-risk myelodysplastic syndrome (MDS) whose cells have a specific change in the IDH2 gene.
Berkeley, CaliforniaAges 18 years+ - NCT05677100Recruiting
Aortix device for acute heart failure
This trial is testing a small pump placed in the aorta to help the kidneys remove extra fluid when standard IV diuretics aren't working well enough for severe heart failure. It may help you if you're hospitalized and still very congested (swollen, fluid buildup) despite strong water pills.
Concord, CaliforniaAges 21 years+ - 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 - NCT06287944RecruitingPhase 1
Targeted radiation and chemo before donor stem cell transplant
This trial tests a new approach: giving a targeted radioactive antibody (attached to a drug that finds CD38 on cancer cells) along with low-dose chemotherapy and targeted radiation to the bone marrow before a donor stem cell transplant. The goal is to prepare the body for the transplant while killing remaining cancer cells more precisely.
Duarte, CaliforniaAges 18–70 - NCT05334368RecruitingPhase 3
Depemokimab for hypereosinophilic syndrome with frequent flares
This trial studies depemokimab, a medicine meant to reduce flares in people with hypereosinophilic syndrome (HES). It also checks how safe the treatment is and how well it works over time.
San Diego, CaliforniaAges 18 years+ - NCT07605429RecruitingPhase 3
Rugonersen treatment for Angelman syndrome
This study tests a new medicine called rugonersen for people with Angelman syndrome. The medicine is given as an injection into the fluid around the spinal cord and may help improve symptoms by targeting the genetic cause of the condition.
San Diego, CaliforniaAges 1–50 - NCT06415344Enrolling by invitationPhase 3
Long-term extension of GTX-102 in Angelman syndrome
This study is a long-term follow-up for people with Angelman syndrome who have already taken part in a GTX-102 trial. It tests whether the treatment continues to be safe and helpful over a longer period.
Los Angeles, CaliforniaAges Any age - 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 - NCT06371417RecruitingPhase 1
Testing RAY121 for several immune diseases (RAINBOW trial)
This trial tests a new medicine called RAY121 (an antibody) for people with certain autoimmune diseases. It aims to see if RAY121 is safe and how the body handles it. If you have one of these conditions and meet the basic health requirements, you might be able to join.
Orange, CaliforniaAges 18–85 - NCT07422480RecruitingPhase 3
Comparing elritercept to epoetin alfa for anemia in MDS needing transfusions
This trial tests a new drug called elritercept against a standard treatment, epoetin alfa, for people with a low-risk form of MDS who need regular blood transfusions for anemia. The goal is to see if elritercept works better to reduce the need for transfusions.
Duarte, 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.
- University of California, Los Angeles (UCLA) · 5 studies
- University of California, San Francisco · 5 studies
- Rady Children's Hospital San Diego · 3 studies
- Stanford University · 3 studies
- University of California, Irvine Medical Center / UC Irvine Health · 3 studies
- Cedars-Sinai Medical Center · 2 studies
- Children's Hospital Los Angeles · 2 studies
- Children's Hospital of Orange County · 2 studies
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 273 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.