Clinical trials · Massachusetts
Down Syndrome clinical trials in Massachusetts
We’re tracking 156 recruiting down syndrome studies with a site in Massachusetts — 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 Massachusetts
- NCT05703542RecruitingPhase 1
Testing BXCL701 for relapsed or resistant AML or MDS
This Phase 1 study tests BXCL701 in adults whose acute myeloid leukemia (AML) or high-blast myelodysplastic syndrome (MDS-EB-2) has come back or not responded to prior treatment. The goal is to find a safe dose and see how the treatment affects cancer cells.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges 18 years+ - 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.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges Up to 3 months - 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.
Boston, MassachusettsAges 18 years+ - NCT06451874Recruiting
Exercise for older veterans with memory and walking concerns
This study tests whether personalized exercise programs can help older veterans who walk slowly and have mild memory or thinking concerns. The goal is to improve your physical and mental function.
Boston, MassachusettsAges 65 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.
Boston, MassachusettsAges 2–65 - NCT01522183Recruiting
Registry for people with atypical hemolytic uremic syndrome
This is a registry that collects information from people diagnosed with atypical hemolytic uremic syndrome (aHUS). It helps researchers better understand the condition across different genetic causes and treatment needs.
Boston, MassachusettsAges Any age - 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.
Boston, MassachusettsAges 1–18 - NCT06219863Recruiting
Harness walking help for babies with Down syndrome
This study tests if a special harness can help babies with Down syndrome learn to walk earlier. It is designed for infants under 2 years old who can sit but are not yet walking.
Boston, MassachusettsAges Up to 2 years - NCT02000089RecruitingPhase 3
Pancreas screening study for people at high cancer risk
This Phase 3 study tests a structured plan for checking the pancreas early in people with a higher-than-usual risk of pancreatic cancer. The goal is to find changes sooner, when treatment may be easier.
Boston, MassachusettsAges 18 years+ - 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.
Boston, MassachusettsAges 2 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.
Lowell, MassachusettsAges 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.
Quincy, MassachusettsAges 14 years+ - NCT05528744Recruiting
Study genes and symptoms in ANKRD17-related CAGS
This study looks at how a specific gene change in ANKRD17 relates to the symptoms, brain/scan findings, and brain-cells in people with Chopra-Amiel-Gordon syndrome (CAGS). It may help families understand the condition better and connect gene results to what is seen clinically.
Boston, MassachusettsAges Any age - NCT05419492RecruitingPhase 1/Phase 2
ETX101 for children with SCN1A Dravet syndrome
This early-phase study tests how safe ETX101 is and whether it can help reduce seizures in young children with Dravet syndrome caused by an SCN1A gene change. It mainly enrolls infants and children with a specific type of seizure history and genetic result.
Boston, MassachusettsAges 6 months–17 years - 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.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges 18–75 - 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.
Boston, MassachusettsAges 18 years+ - NCT05196984Recruiting
Gamma brain stimulation study for adults with Down syndrome
This trial tests a brain stimulation approach aimed at affecting brain activity in people with Down syndrome. It may help researchers understand whether this kind of stimulation can improve certain brain or thinking-related outcomes.
Cambridge, MassachusettsAges 25–65 - NCT04968834Recruiting
Collecting children’s and young adults’ blood and marrow for genetic testing
This study collects and stores blood and bone marrow samples from young people with certain blood cancers so doctors can do genetic (“genomic”) testing now and in the future. It may help improve understanding of the disease and support better matching of care for patients.
Boston, MassachusettsAges Up to 30 years - NCT04541654Recruiting
Study of families with TP53 gene changes in Li-Fraumeni syndrome
This study is looking at how people in families with certain TP53 gene changes (including Li-Fraumeni syndrome) develop health problems over time. It may help doctors better understand risk and improve care for affected families.
Boston, MassachusettsAges Any age - 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.
Lexington, MassachusettsAges birth–20 years - NCT04206215Recruiting
Noninvasive brain stimulation for carpal tunnel pain relief
This study tests whether a noninvasive brain stimulation treatment can reduce chronic carpal tunnel (CTS) pain, especially when pain spreads beyond the usual nerve area. If you have long-lasting CTS pain that hasn’t improved with standard treatments, you may be able to try this therapy while being closely monitored for safety.
Charlestown, MassachusettsAges 18–80
Down Syndrome trials by city in Massachusetts
Where these studies are running in Massachusetts
Institutions with a site for the recruiting down syndrome studies listed above.
- Boston Children's Hospital · 7 studies
- Dana-Farber Cancer Institute · 4 studies
- Beth Israel Deaconess Medical Center · 3 studies
- Massachusetts General Hospital · 3 studies
- Tufts Medical Center · 3 studies
- Brigham and Women's Hospital · 2 studies
- Boston University
- Clinical Trial 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 Massachusetts?
- Yes. We're currently tracking 156 recruiting down syndrome studies with a site in Massachusetts, 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 Massachusetts?
- 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 Massachusetts to take part?
- Not necessarily. These studies have a site in Massachusetts, 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 Massachusetts 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.