Clinical trials · Boston, MA
Down Syndrome clinical trials in Boston
We’re tracking 166 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
- 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+ - NCT03530852Recruiting
RELIZORB test for children needing tube feeding and IV nutrition
This study tests whether RELIZORB helps children with short bowel syndrome (SBS), who rely on IV nutrition (and also tube feeding), absorb fats better. It is an open-label study over 90 days, meaning everyone gets the study treatment and outcomes are compared over time.
Boston, MassachusettsAges 2–18 - 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+ - NCT03816345RecruitingPhase 1
Nivolumab for advanced cancer with autoimmune disease
This trial tests whether the immunotherapy drug nivolumab can safely treat advanced cancer in people who also have an autoimmune disease like rheumatoid arthritis, lupus, or multiple sclerosis. It aims to find out if the drug may help control cancer while managing their autoimmune condition.
Boston, MassachusettsAges 18 years+ - NCT02269592Recruiting
Study blood marker changes in early, symptom-free blood cancers
This study follows people with early, “precursor” blood conditions to learn what changes in their blood or bone marrow signal progression. It may help doctors better predict outcomes and understand why some people develop symptom-causing cancer.
Boston, MassachusettsAges 18 years+ - 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 - NCT05587127Recruiting
CBT therapy for eating anxiety in functional stomach pain
This trial tests an “exposure-based” type of cognitive behavioral therapy (CBT) to help people with functional dyspepsia (stomach discomfort after eating) who avoid or restrict food. The goal is to reduce food fear and improve eating and weight, after other medical causes have been ruled out.
Boston, MassachusettsAges 18 years+ - NCT05292664RecruitingPhase 1
Venetoclax trial for high-risk blood cancers in young patients
This Phase 1 study tests venetoclax to treat certain high-risk blood cancers in children, teens, or young adults. It is designed to see if the drug can work safely for patients whose disease has returned or did not respond to prior treatment.
Boston, MassachusettsAges 1–40 - NCT04988555RecruitingPhase 1/Phase 2
Study of an investigational drug for high-risk acute leukemia
This Phase 1/2 study tests enzomenib (DSP-5336) in people with relapsed or hard-to-treat acute leukemia (and, in some places, certain other blood cancers). The goal is to see how the drug works and how safe it is, especially for leukemia cases with specific genetic changes (KMT2A/MLL or NPM1).
Boston, MassachusettsAges 12 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 - 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 - 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 - NCT07102797Recruiting
ActiveGirls: Activity, hormones, and diabetes risk in young girls
This study looks at how physical activity and hormone health affect diabetes risk in young girls. It is for girls ages 8 to 12 who are at risk for PCOS (a hormone condition) and insulin resistance, and who have not yet started their period.
Somerville, MassachusettsAges 8 years+ - 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.
Brookline, 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 - NCT05932589Recruiting
Study of brain signals as signs of Rett syndrome
This study measures brain and nerve activity (brain signals) to find patterns that match Rett syndrome. It may help researchers better track Rett changes over time and develop more precise ways to measure response in future treatments.
Brookline, MassachusettsAges 1–18 - NCT06821568Recruiting
Home brain stimulation for memory and walking concerns
This study tests if a gentle brain-stimulation device used at home can help older adults who have both memory worries and slower walking. A study partner will help you use the device.
Roslindale, MassachusettsAges 65–90 - 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.
Chestnut Hill, MassachusettsAges 22–80 - NCT07008989Recruiting
Phone app to improve memory in Down syndrome
This study tests a smartphone app called HippoCamera to see if it can help teenagers and young adults with Down syndrome remember everyday events better. You would use the app at home with some help from a parent or guardian.
Chestnut Hill, MassachusettsAges 15–35 - NCT07464093Recruiting
Pilates for joint hypermobility pain
This trial tests if a special Pilates program can reduce pain in people with hypermobile Ehlers-Danlos syndrome or hypermobility spectrum disorder. You must have had pain for at least 3 months and be able to get on and off the floor on your own.
Chestnut Hill, MassachusettsAges 18 years+ - NCT05737121RecruitingPhase 2
Study drug for bladder pain compared with lidocaine, heparin, or placebo
This Phase 2 trial tests VNX001 to see if it helps people with interstitial cystitis/bladder pain syndrome (IC/BPS) compared with its separate ingredients (lidocaine and heparin) or a placebo (inactive treatment). You may be eligible if you have had IC/BPS for months and recently had a moderate-to-severe bladder pain flare.
Watertown, MassachusettsAges 18 years+ - NCT05938543Recruiting
Brain stimulation and appetite in Prader-Willi syndrome
This trial studies whether targeted brain stimulation (TMS) can help with appetite and eating feelings in people with Prader-Willi syndrome. It also includes an MRI scan to measure brain-related effects, but only if you can safely have these scans.
Belmont, MassachusettsAges 18–64 - NCT07538583RecruitingPhase 2/Phase 3
QRX003 Lotion for Netherton Syndrome
This study tests a new lotion (QRX003) designed to treat Netherton Syndrome, a rare genetic skin condition. The lotion is applied to affected skin areas to see if it can reduce symptoms and improve skin health.
Quincy, MassachusettsAges 4 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+
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.
- Massachusetts General Hospital · 5 studies
- Boston Children's Hospital · 3 studies
- Dana-Farber Cancer Institute · 3 studies
- Bay State Clinical Trials
- Beacon Clinical Research
- Boston College ONLINE STUDY
- Brigham and Women's Hospital
- 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.
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 166 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.