Clinical trials · Massachusetts
Down Syndrome clinical trials in Massachusetts
We’re tracking 172 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
- NCT07450664Enrolling by invitation
Observational study of VYKAT XR for Prader-Willi syndrome
This study follows people with Prader-Willi syndrome who are already taking or starting VYKAT XR, including those switching from an earlier version of the drug (DCCR). It aims to understand how the treatment works in everyday life.
Boston, MassachusettsAges 4 years+ - NCT07211685RecruitingPhase 2
Testing a new drug for adults with Alport syndrome
This study tests a new medicine called BAY 3401016 to see if it can help adults with Alport syndrome, a genetic condition that affects the kidneys. The drug is given as an infusion or injection and the study will check how well it works and if it is safe.
Boston, MassachusettsAges 18–45 - NCT06914609RecruitingPhase 3
Study of ION582 for people with Angelman syndrome
This trial tests a new medicine called ION582 for people with Angelman syndrome. It aims to see if it can help with symptoms and is given as a lumbar puncture (spinal tap). You may be able to join if you have a genetic diagnosis of Angelman syndrome and are between 2 and 50 years old.
Boston, MassachusettsAges 2–50 - 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.
Lexington, MassachusettsAges 3–12 - 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.
Brookline, MassachusettsAges 3–12 - 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.
Boston, MassachusettsAges 5–30 - NCT07568015Recruiting
Nerve-targeted treatment for lateral hip pain
This study tests an experimental treatment that targets nerves around the hip to relieve pain on the outside of the hip. It may help people who have tried other treatments without enough relief.
Burlington, MassachusettsAges 22–80 - 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 - 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+ - 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.
Boston, MassachusettsAges 2–17 - 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+ - 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.
Boston, MassachusettsAges 18 years+ - 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.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges 4–50 - 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 - 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+ - 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.
Boston, MassachusettsAges 15 years+ - 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 - 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.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges 25–55 - 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 - 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.
Boston, MassachusettsAges birth–20 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.
Boston, MassachusettsAges Any age - 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
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 · 10 studies
- Massachusetts General Hospital · 4 studies
- Dana-Farber Cancer Institute · 2 studies
- Neuren PMS-301 Site#104 · 2 studies
- Tufts Medical Center · 2 studies
- Boston Children's Hospital (Harvard Collaboration)
- Boston University
- Brigham and Women'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 Massachusetts?
- Yes. We're currently tracking 172 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.