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 2, Phase 2, Phase 2/Phase 3, Phase 3 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies near Boston
- NCT05127226RecruitingPhase 1/Phase 2
Test drug safety for Angelman syndrome in young children
This early-phase study tests a new experimental medicine called ION582 to see if it is safe, tolerable, and how the body processes it in people with Angelman syndrome. It may also look at early signs of how the medicine affects the brain and body, which could guide future treatments.
Boston, MassachusettsAges birth–50 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 - NCT05561647Recruiting
Survey of GATTEX knowledge for patients and prescribers
This study uses a survey to check how much people and doctors know about using GATTEX. It may help improve education and support for safer, more informed use of the medicine.
Cambridge, 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 - 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+ - 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 - 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 - 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 - 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+ - 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 - 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+ - NCT06999954Recruiting
Global survey for Shwachman-Diamond syndrome and related conditions
This trial is a global survey and registry for people with Shwachman-Diamond Syndrome and related inherited blood disorders. It aims to connect patients, families, and researchers to better understand these conditions and improve care.
Woburn, MassachusettsAges Any age - NCT07531251RecruitingPhase 4
Barth Syndrome Heart Treatment Study
This trial tests a medicine called elamipretide (4TAZ) in boys with Barth Syndrome, a genetic condition affecting the heart and muscles. The goal is to see if this medicine can help improve heart function and how the body uses energy.
Needham, MassachusettsAges 5–55 - 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 - NCT07417137Recruiting
Long-term study of Angelman syndrome
This study follows people with Angelman syndrome over time to learn more about the condition. It does not test any new treatments, but you need a parent or caregiver to come with you to all visits.
Lexington, MassachusettsAges 1 year+ - NCT07565636Recruiting
Custom 3D-Printed Wrist Support for Ehlers-Danlos Syndrome
This trial tests a custom-made wrist brace designed with 3D printing technology to help stabilize loose, overly flexible wrists in people with Ehlers-Danlos syndrome. The goal is to see if this personalized support can improve wrist function and reduce pain or instability.
Lowell, MassachusettsAges 21 years+ - NCT06810518Recruiting
Studying stool test for digestive disease diagnosis
This trial tests a new stool test to measure calprotectin levels, which could help doctors tell the difference between inflammatory bowel disease (IBD) and irritable bowel syndrome (IBS). If you have symptoms like stomach pain, diarrhea, or weight loss and are about to have a colonoscopy, you may be able to join.
Salem, New HampshireAges 22 years+ - NCT05604430Enrolling by invitation
Study of pre-hospital breathing support with a specific ventilator
This study looks at breathing support given outside the hospital using a particular ZOLL ventilator model and software version. If you were treated with the right equipment, the team will use the ventilator’s recorded data to evaluate how ventilation was delivered.
Uxbridge, MassachusettsAges Any age - NCT07038200RecruitingPhase 3
Study of Del-brax (AOC 1020) for People with FSHD
This study tests an experimental medicine called Del-brax (AOC 1020) for people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to see if it can help improve muscle strength and slow the disease. You may be able to join if you have a confirmed FSHD diagnosis and can walk at least 10 meters on your own.
Worcester, MassachusettsAges 16–70 - NCT06888024Recruiting
Measuring pelvic vein blood flow with MRI
This study uses MRI scans to measure blood flow in the pelvic veins. It’s for people who either have no vein problems, or have symptoms of a compressed left iliac vein, or already have a stent in that vein. The goal is to learn more about how blood flows in these conditions.
Worcester, MassachusettsAges 18–89
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 · 2 studies
- Neuren PMS-301 Site#104 · 2 studies
- University of Massachusetts Chan Medical School · 2 studies
- ALPCO
- Bay State Clinical Trials
- Beacon Clinical Research, LLC
- Boston College
- 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.
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.