Clinical trials · Massachusetts
Down Syndrome clinical trials in Massachusetts
We’re tracking 156 recruiting down syndrome studies with a site in Massachusetts — including Early Phase 1, Phase 1, 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 in Massachusetts
- NCT06782373RecruitingPhase 2
Pacritinib for VEXAS syndrome
This trial tests whether the drug pacritinib can safely reduce inflammation and improve symptoms in people with VEXAS syndrome, a rare genetic condition that causes inflammation in various parts of the body. It may be a new treatment option for those who still have inflammation despite taking steroids.
Boston, MassachusettsAges 18 years+ - NCT06776783RecruitingPhase 3
Testing a new surfactant for breathing problems in premature babies
This study tests a new medicine called APC-0101, a type of surfactant, for premature babies with breathing problems (respiratory distress syndrome). It may help their lungs work better and reduce the need for more breathing support.
Boston, MassachusettsAges 1 hour–1 day - 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 - NCT06056635Recruiting
Study of special telescopes for fetal surgery
This study tests two types of small cameras (fetoscopes) to see how well they work for surgery on a baby before birth. Doctors want to learn if these tools can safely perform fetal procedures.
Boston, MassachusettsAges 18–45 - NCT06690801Recruiting
Nitric oxide breathing gas for children with lung failure
This trial tests whether adding a breathing gas called nitric oxide to standard care helps children with severe lung failure (ARDS) recover faster. It is for children on a breathing machine who still have low oxygen levels.
Boston, MassachusettsAges birth–21 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 - 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 - 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+ - 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 - 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+ - 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+ - NCT07403812Recruiting
Checking thinking skills after CAR-T therapy
This study uses a simple iPad test to check for thinking problems that can happen after CAR-T cell therapy. It aims to catch these issues early so they can be managed better.
Boston, MassachusettsAges 18 years+ - 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 - NCT07277582RecruitingPhase 2/Phase 3
Study of a new medicine for long QT syndrome types 2 and 3
This study tests a new medicine called THRV-1268 to see if it can safely improve heart rhythm in people with Long QT syndrome types 2 and 3. It may help reduce the risk of dangerous heart rhythm problems.
Boston, MassachusettsAges 15 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 - NCT07220239Recruiting
Menstrual cup for early cancer detection in Lynch syndrome
This study tests whether a menstrual cup can collect cells from the uterus to detect early endometrial cancer in women with Lynch syndrome. If you have Lynch syndrome and are due for a routine biopsy, you may be able to use a menstrual cup instead.
Boston, MassachusettsAges 18 years+ - NCT07209462RecruitingPhase 2
MRM-3379 for Fragile X Syndrome in males
This trial tests an investigational medicine called MRM-3379 in males with Fragile X syndrome. It looks at how the medicine affects thinking and behavior over time.
Worcester, MassachusettsAges 13–45 - NCT06297941RecruitingPhase 1
Study of REM-422 for AML or high-risk MDS
This trial tests an experimental oral medication called REM-422 for people with AML (a type of blood cancer) that has not responded to treatment or has come back, or for those with high-risk MDS. The goal is to see if it is safe and effective.
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 - NCT07020819RecruitingPhase 3
Study of tanruprubart for people with Guillain-Barré syndrome
This trial tests a new medicine, tanruprubart (also called ANX005), for people with Guillain-Barré syndrome. It aims to see if the drug can help improve recovery when given soon after symptoms start.
Worcester, MassachusettsAges 12–85 - NCT07016555RecruitingEarly Phase 1
Mouth burning study: symptoms and management
This study looks at ways to manage the burning pain in your mouth if you have burning mouth syndrome or ongoing mouth burning without a clear cause. It tests if certain natural extracts can help ease the discomfort.
Amherst, MassachusettsAges 18 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 - NCT06840496RecruitingPhase 3
Study of oral NA-921 vs placebo for girls and women with Rett syndrome
This trial tests an oral medication called NA-921 in females with Rett syndrome to see if it works better than a placebo. It may be an option for those who have a confirmed MECP2 gene mutation and stable seizure patterns.
Boston, MassachusettsAges 5–20 - NCT06817590RecruitingPhase 1
Nucleoside therapy for telomere disorders
This trial tests a nucleoside therapy (dC/dT) for people with telomere biology disorders, which cause premature aging and problems like low blood counts, lung or liver issues. The goal is to see if the treatment can help manage these symptoms.
Boston, MassachusettsAges 1–70
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 · 2 studies
- Massachusetts General Hospital · 2 studies
- Beacon Clinical Research
- Beth Israel Deaconess Medical Center
- Biomed Testing Facility #BIO-10-02115
- Heart Center Clinical Research Program | MGH
- Mass General Brigham
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.