Clinical trials · Boston, MA
Down Syndrome clinical trials in Boston
We’re tracking 182 recruiting down syndrome studies with a site in or near Boston — 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 near Boston
- NCT06462365RecruitingPhase 1
Preventing GvHD after stem cell transplant for blood cancers
This trial tests a new way to prevent graft-versus-host disease (GvHD), a common complication after a stem cell transplant. It is for people with certain blood cancers who are getting a stem cell transplant from a half-matched family member or unrelated donor.
Boston, MassachusettsAges 18 years+ - NCT06585618Recruiting
Pediatric Deep Brain Stimulation Registry
This study creates a list of children who already have or plan to get Deep Brain Stimulation (DBS) to see how it helps with movement problems. Joining helps doctors learn more about how DBS works in kids.
Boston, MassachusettsAges birth–18 years - NCT01186913Enrolling by invitation
Natural history study for severe combined immune deficiency (SCID)
This study follows children and adults with SCID and related immune disorders to understand how the illness behaves over time. It also helps researchers learn which testing results and genetic types of SCID match different treatment plans.
Boston, MassachusettsAges Any age - 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 - NCT06703073RecruitingPhase 2
Testing new treatments for acute respiratory distress syndrome (ARDS)
This trial is testing new treatments for people with ARDS, a serious lung condition. The goal is to find therapies that help patients breathe better and recover faster. If you or a loved one has ARDS, this study may offer access to experimental treatments.
Boston, MassachusettsAges 18 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+ - NCT03850574RecruitingPhase 1/Phase 2
Tuspetinib for relapsed or hard-to-treat AML
This early-phase study tests tuspetinib in people with AML that has come back or did not respond to treatment. The goal is to learn whether it is safe and tolerable, how the drug behaves in the body, and whether it shows signs of benefit.
Boston, MassachusettsAges 18 years+ - NCT05724173Recruiting
Test of a brain-to-computer device for ALS hand paralysis
This study checks whether a brain-computer interface (a BrainGate2 system) is safe and workable for people with ALS. If you qualify, you may be asked to try the device and complete study tasks to help researchers learn how well it can support communication or control.
Boston, MassachusettsAges 18–80 - 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 - NCT04678401RecruitingPhase 1
Trial tests transplant using a half-matched donor for high-risk leukemia
This Phase 1 trial tests a special bone marrow transplant using a related “half-matched” donor (haploidentical), without a specific immune-suppressing medicine (“IS-free”). It’s for adults with certain high-risk AML or MDS to see if this approach can improve control of the disease while tracking safety.
Boston, MassachusettsAges 18–75 - 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+ - NCT06059391RecruitingPhase 2
Vaccine for stem cell donors to prevent CMV
This trial tests whether giving a CMV vaccine to stem cell donors can help protect recipients from CMV infection after transplant. It is for adults with specific blood cancers who are CMV-positive and have a matched related donor.
Boston, MassachusettsAges 18–75 - NCT03471260RecruitingPhase 1/Phase 2
Ivosidenib and venetoclax for IDH1-mutated leukemia or MDS
This Phase 1/2 study tests whether the medicines ivosidenib and venetoclax (with or without azacitidine) can help people with certain “IDH1-mutated” blood cancers like AML or high-risk MDS. It’s meant for patients whose disease has come back, doesn’t respond, or who can’t safely get standard chemotherapy.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges 18 years+ - NCT05675878RecruitingPhase 3
Testing a specific diet for Gulf War illness
This Phase 3 study tests whether following a study diet can improve symptoms of Gulf War illness. It may help people by confirming whether diet changes are an effective treatment.
Boston, MassachusettsAges 50–75 - 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 - NCT07391605RecruitingPhase 2
Descartes-08 for treatment-resistant myositis
This trial tests a new treatment called Descartes-08 for people with dermatomyositis or antisynthetase syndrome whose disease hasn't responded well to standard therapies. It aims to see if this treatment can help control muscle and skin symptoms.
Boston, MassachusettsAges 18 years+ - NCT04024761RecruitingPhase 1
NK cell therapy for leukemia relapse after transplant
This Phase 1 study tests whether an infusion of special immune cells (NK cells) from a donor can help control leukemia or related blood cancers that come back or do not fully go away after a stem cell transplant. It also uses short chemotherapy to “make space” for the NK cells to work, and IL-2 to help them function.
Boston, MassachusettsAges 12 years+ - 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+ - NCT04256317RecruitingPhase 2/Phase 3
Azacitidine pills with or without venetoclax for certain blood cancers
This study tests whether a pill form of azacitidine (ASTX030) helps people with myelodysplastic syndromes (MDS), CMML, or AML. Some groups may also receive venetoclax; the goal is better blood cancer control for people who aren’t good candidates for intensive chemotherapy.
Boston, MassachusettsAges 18 years+ - NCT04775706RecruitingPhase 2
Test of sonefpeglutide for people with short bowel syndrome
This Phase 2 study tests whether sonefpeglutide is safe and how the body handles it (pharmacokinetics) in people with short bowel syndrome. It may help people who still need nutrition through a tube because their intestinal condition is stable.
Boston, MassachusettsAges 18 years+ - NCT06701682RecruitingPhase 2
Study of vilobelimab for moderate to severe ARDS
This trial tests a drug called vilobelimab for people with moderate or severe ARDS (a serious lung condition). It aims to find if this treatment can help improve breathing and outcomes.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges 4–50 - NCT06668805RecruitingPhase 2
Testing vosoritide for children with Noonan syndrome and slow growth
This study tests a medication called vosoritide for children with Noonan syndrome who are still growing slowly despite using human growth hormone. The goal is to see if vosoritide can help them grow taller.
Boston, MassachusettsAges 3–11
Hear when a new Down Syndrome trial opens near Boston, MA
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Down Syndrome trials in other cities
Where these studies are running near Boston
Institutions with a site for the recruiting down syndrome studies listed above.
- Boston Children's Hospital · 7 studies
- Dana-Farber Cancer Institute · 6 studies
- Brigham and Women's Hospital · 3 studies
- Massachusetts General Hospital · 3 studies
- Tufts Medical Center · 3 studies
- Lahey Hospital and Medical Center · 2 studies
- Boston University
- GSK Investigational 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.
Looking wider? See all recruiting down syndrome studies in Massachusetts.
Common questions
- Are there down syndrome clinical trials near Boston?
- Yes. We're currently tracking 182 recruiting down syndrome studies with a site near Boston, 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 near Boston?
- 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 Boston to take part?
- Not necessarily. These studies have a site near Boston, 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 near Boston regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.