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 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- 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 - 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+ - NCT06583395Enrolling by invitation
Meditation retreat study with fitness tracker
This trial studies people attending a 7-day meditation retreat to see how meditation affects well-being. You will wear a fitness tracker and answer surveys.
Palm Desert, CaliforniaAges 21 years+ - NCT06515184RecruitingPhase 3
Coenzyme Q10 for Gulf War illness study
This study tests whether coenzyme Q10 (a natural supplement) can improve symptoms of Gulf War illness, such as fatigue and body pain. It involves taking the supplement or placebo for several months, with regular check-ins via Zoom and a few blood draws at a local lab.
La Jolla, CaliforniaAges 50 years+ - NCT06499285RecruitingPhase 3
Study of a new drug to treat anemia in adults with low to intermediate risk MDS who need transfusions
This study tests a new drug called elritercept for people with myelodysplastic syndromes (MDS) who have low blood counts and need regular blood transfusions. The drug aims to boost red blood cell production and reduce or eliminate the need for transfusions, which can improve energy and quality of life.
Duarte, CaliforniaAges 18 years+ - NCT06501196RecruitingPhase 1
Study of BH-30236 for leukemia and high-risk MDS
This trial tests an experimental drug, BH-30236, for people with acute myeloid leukemia (AML) or a high-risk form of myelodysplastic syndrome (MDS) whose cancer has come back or stopped responding to treatment. It aims to see if the drug can help control the disease.
Palo Alto, CaliforniaAges 18 years+ - NCT06421350Recruiting
Closed-loop spinal cord stimulation for complex regional pain syndrome
This trial tests a new type of spinal cord stimulator that automatically adjusts to your pain signals. It may help people with complex regional pain syndrome (CRPS) who have not tried this kind of therapy before.
La Jolla, CaliforniaAges 18 years+ - NCT06430385RecruitingPhase 1/Phase 2
Study of ION440 for MECP2 Duplication Syndrome
This trial tests an experimental medicine called ION440 for people with MECP2 Duplication Syndrome. The medicine is given as an injection into the spinal fluid, and the study aims to check its safety and how well it works.
San Diego, CaliforniaAges 2–65 - NCT06394830RecruitingPhase 2
Testing VNX001 for bladder pain syndrome
This study tests a new treatment (VNX001) for people with interstitial cystitis or bladder pain syndrome (IC/BPS). You must have already taken part in an earlier study of this drug to join. The goal is to see if it safely reduces bladder pain.
Fresno, CaliforniaAges 18 years+ - 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 - NCT06366464RecruitingPhase 3
Study of a drug for sleepiness in Prader-Willi syndrome
This trial tests a medication called pitolisant to see if it helps reduce excessive daytime sleepiness in people with Prader-Willi syndrome. You or your loved one may be able to join if they have a confirmed diagnosis and a caregiver who can regularly help with study tasks.
Los Angeles, CaliforniaAges 6 years+ - NCT06333041RecruitingPhase 2/Phase 3
CBD study for Sanfilippo syndrome
This study tests whether a cannabis-derived medicine called cannabidiol (CBD) can help children with Sanfilippo syndrome, a rare genetic disorder. It may be a good fit for children who have already tried other treatments or whose disease is more advanced.
Torrance, CaliforniaAges 4 years+ - 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 - NCT06247787RecruitingPhase 1
Imetelstat plus chemo for relapsed leukemia in kids
This trial tests a new drug called imetelstat combined with two chemotherapy drugs (fludarabine and cytarabine) for children and teens with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) that has come back or not responded to treatment. The goal is to find the safest dose of imetelstat and see if it helps control the cancer.
Orange, CaliforniaAges 1–18 - NCT06146257RecruitingPhase 1
Study of GLB-001 for Relapsed or Refractory AML or High-Risk MDS
This trial tests an oral investigational drug (GLB-001) for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that has returned or hasn't responded to other treatments. The goal is to see if it's safe and effective for those who have run out of standard options.
Irvine, CaliforniaAges 18 years+ - NCT06128070RecruitingPhase 2
Preventing GVHD after stem cell transplant for leukemia
This trial tests adding ruxolitinib to standard drugs (tacrolimus and methotrexate) to prevent graft-versus-host disease in children and young adults undergoing a stem cell transplant for acute leukemia or myelodysplastic syndrome. The goal is to lower the risk of GVHD and improve transplant outcomes.
Duarte, CaliforniaAges 2–22 - 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+ - NCT06063486RecruitingPhase 2
Curcumin for inflammation and symptoms in blood disorders
This trial tests whether curcumin, a natural compound found in turmeric, can reduce inflammation and improve symptoms like fatigue in people with certain blood disorders that cause low blood counts or overproduction of blood cells. It is for patients with clonal cytopenia of uncertain significance, low-risk myelodysplastic syndrome, or myeloproliferative neoplasms (polycythemia vera, essential thrombocytosis, or myelofibrosis) who have bothersome symptoms.
Los Angeles, CaliforniaAges 18 years+ - NCT06066502RecruitingPhase 3
Precision ventilation for ARDS
This study tests whether a customized breathing machine approach ("precision ventilation") is better than usual care for patients with ARDS. The goal is to improve oxygen levels and recovery while avoiding lung damage.
Los Angeles, CaliforniaAges 18 years+ - NCT06073886RecruitingPhase 2
Better brain stimulation for long-lasting concussion symptoms
This trial tests whether personalized brain stimulation can help people who have ongoing symptoms more than 20 on a standard concussion scale, such as headaches, dizziness, or trouble concentrating, after a mild head injury in the past year.
Westwood, Los Angeles, CaliforniaAges 18–65 - NCT06043440RecruitingPhase 2
Oxygen therapy for sleep apnea in Down syndrome
This study tests whether oxygen therapy can help children with Down syndrome who have sleep apnea. It compares oxygen treatment to no treatment to see if it improves breathing during sleep and thinking skills.
Los Angeles, CaliforniaAges 5–18 - NCT06018766RecruitingPhase 2
LAM-001 for lung transplant patients with BOS
This study tests whether the drug LAM-001 can help people who have developed a condition called bronchiolitis obliterans syndrome (BOS) after a double lung transplant. BOS causes a slow decline in lung function, and this trial aims to see if LAM-001 can slow or stop that decline.
San Francisco, CaliforniaAges 18 years+ - NCT05947955RecruitingPhase 2
Study drug for infection-related lung failure in adults
This Phase 2 trial tests Rhu-pGSN in adults with acute respiratory distress syndrome (ARDS) caused by an infection. If you qualify, the study may help determine whether the drug improves outcomes in this short-term, life-threatening lung condition.
Los Angeles, CaliforniaAges 18 years+ - NCT05789056RecruitingPhase 2/Phase 3
Study lotion for Netherton syndrome safety and symptom improvement
This trial tests a prescription lotion (QRX003) to see if it is safe and helpful for people with Netherton syndrome. Because the study checks skin symptoms on certain body areas and closely controls other treatments, it may be best for patients who can keep their current regimen stable.
San Diego, CaliforniaAges 14 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.
- City of Hope · 5 studies
- UC San Diego Moores Cancer Center · 2 studies
- University of California, San Francisco · 2 studies
- Alta Bates Summit Medical Center-Herrick Campus
- Cedar-Sinai Medical Center
- Cedars-Sinai Medical Center
- Center of Excellence in Diabetes and Endocrinology
- Children's Hospital of Los Angeles
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.