Clinical trials · Massachusetts
Down Syndrome clinical trials in Massachusetts
We’re tracking 173 recruiting down syndrome studies with a site in Massachusetts — including 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
- NCT04689646Recruiting
Mind-body therapy for chronic back pain
This study tests whether a mind-body therapy approach can reduce disability and how bothersome chronic back pain feels. It may help people whose pain has lasted for months and affects daily life.
Boston, MassachusettsAges 18–67 - 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 - 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 - 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+ - 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 - NCT05354141RecruitingPhase 3
Tested treatment with tiny lung particles for moderate to severe ARDS
This Phase 3 trial studies an “extracellular vesicle” treatment in people with moderate to severe ARDS, a serious lung breathing problem. The goal is to see whether the treatment can improve outcomes compared with standard care.
Boston, MassachusettsAges 18–75 - NCT05410977Recruiting
Testing blood and stool for colon cancer in Lynch syndrome
This study collects blood and stool samples to see how well they can detect colorectal cancer or advanced growths in people with Lynch syndrome (an inherited colon cancer risk) or suspected Lynch syndrome. It may help find cancer or precancer earlier, which can improve next-step decisions.
Boston, MassachusettsAges 18 years+ - NCT05528744Recruiting
Study genes and symptoms in ANKRD17-related CAGS
This study looks at how a specific gene change in ANKRD17 relates to the symptoms, brain/scan findings, and brain-cells in people with Chopra-Amiel-Gordon syndrome (CAGS). It may help families understand the condition better and connect gene results to what is seen clinically.
Boston, MassachusettsAges Any age - NCT05703542RecruitingPhase 1
Testing BXCL701 for relapsed or resistant AML or MDS
This Phase 1 study tests BXCL701 in adults whose acute myeloid leukemia (AML) or high-blast myelodysplastic syndrome (MDS-EB-2) has come back or not responded to prior treatment. The goal is to find a safe dose and see how the treatment affects cancer cells.
Boston, MassachusettsAges 18 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+ - NCT05796570RecruitingPhase 2
Decitabine plus filgrastim to prevent relapse after transplant
This trial tests whether giving decitabine with filgrastim soon after a stem cell transplant can be done safely and may help prevent the leukemia or related cancer from coming back. It is aimed at children and young adults with AML, MDS, or related myeloid cancers.
Boston, MassachusettsAges 1–39 - 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 - NCT06247787RecruitingPhase 1
Imetelstat plus chemo for relapsed leukemia in kids
This trial tests a new drug called imetelstat combined with two chemotherapy drugs (fludarabine and cytarabine) for children and teens with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) that has come back or not responded to treatment. The goal is to find the safest dose of imetelstat and see if it helps control the cancer.
Boston, MassachusettsAges 1–18 - 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+ - NCT06338267Recruiting
Testing new sensors for Rett syndrome symptoms at home
This study tests new wearable sensors to track symptoms like heart rate and breathing in people with Rett syndrome. If you join, you'll use the sensors at home to help researchers see how symptoms change day to day.
Boston, MassachusettsAges Any age - NCT06419634RecruitingPhase 1
New drug combo for returning AML or MDS
This study tests a new drug (BMS-986497) alone or combined with two other medicines (azacitidine and venetoclax) for people with AML or MDS that has not responded to or returned after prior treatments. The goal is to see if this combination can help control the disease.
Boston, MassachusettsAges 18 years+ - NCT06430385RecruitingPhase 1/Phase 2
Study of ION440 for MECP2 Duplication Syndrome
This trial tests an experimental medicine called ION440 for people with MECP2 Duplication Syndrome. The medicine is given as an injection into the spinal fluid, and the study aims to check its safety and how well it works.
Boston, MassachusettsAges 2–65 - NCT05419492RecruitingPhase 1/Phase 2
ETX101 for children with SCN1A Dravet syndrome
This early-phase study tests how safe ETX101 is and whether it can help reduce seizures in young children with Dravet syndrome caused by an SCN1A gene change. It mainly enrolls infants and children with a specific type of seizure history and genetic result.
Boston, MassachusettsAges 6 months–17 years - 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 - 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 - NCT06716411Recruiting
Acupuncture for Gulf War Illness relief
This study tests whether acupuncture can help relieve Gulf War Illness symptoms like fatigue, mood or thinking problems, and muscle or joint pain. You may be able to join if you were deployed to the Gulf region and have had these symptoms for more than 6 months.
Boston, MassachusettsAges 18 years+ - NCT06747949RecruitingPhase 3
Long term extension study of dazodalibep for Sjogren's syndrome
This study gives continued access to the study drug dazodalibep for people with Sjogren's syndrome who completed a prior phase 3 study. The goal is to see how safe and effective the medication is over a longer period.
Boston, MassachusettsAges 18 years+ - NCT06782373RecruitingPhase 2
Pacritinib for treating VEXAS syndrome
This trial tests a drug called pacritinib for people with VEXAS syndrome. It aims to see if pacritinib can help control inflammation and reduce the need for steroids. You may qualify if you have a specific UBA1 gene mutation and are currently on a stable steroid dose.
Boston, MassachusettsAges 18 years+
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 · 8 studies
- Dana-Farber Cancer Institute · 5 studies
- Beth Israel Deaconess Medical Center · 3 studies
- Massachusetts General Hospital · 3 studies
- Direct Biologics Investigational Site
- Local Institution - 0007
- Massachusetts Institute of Technology
- McLean 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 173 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.