Clinical trials · Pittsburgh, PA
Down Syndrome clinical trials in Pittsburgh
We’re tracking 53 recruiting down syndrome studies with a site in or near Pittsburgh — 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 Pittsburgh
- NCT03779854RecruitingPhase 2
Preventing graft-versus-host disease in kids after stem cell transplant
This trial tests whether removing certain immune cells (T cells) from the donor transplant can lower the risk of chronic graft-versus-host disease (long-term immune attack after transplant) in children and young adults with blood cancers. If you qualify, it may help improve transplant outcomes while tracking safety and side effects.
Pittsburgh, PennsylvaniaAges 6 months–26 years - NCT06193928Recruiting
Long-term safety study of Livmarli in ALGS or PFIC
This study is following patients who are already taking Livmarli (maralixibat) for Alagille syndrome or PFIC to see how safe and effective it is over the long term.
Pittsburgh, PennsylvaniaAges Any age - NCT05256160Recruiting
Study brain excitability in people with cyclic vomiting syndrome
This study looks at how sensitive your brain is (brain “excitability”) in people with cyclic vomiting syndrome (CVS). It may help researchers understand what is happening in the brain during CVS and how it could be treated in the future.
Pittsburgh, PennsylvaniaAges 18–60 - 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.
Pittsburgh, PennsylvaniaAges 1–18 - NCT05701917Recruiting
Clot-busting device versus blood thinners for leg clot
This study compares a clot-removal device (ClotTriever) plus standard care versus standard blood thinners alone for a deep vein clot in the leg. It aims to see which approach better improves leg symptoms and outcomes.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT07519395RecruitingPhase 2
Camlipixant for abdominal symptoms in IBS-D and IBS-M
This study tests whether a new drug called camlipixant can reduce belly pain and other uncomfortable symptoms in people with irritable bowel syndrome that causes diarrhea or mixed symptoms. The trial compares camlipixant to a placebo (inactive pill) to see if it works better.
Pittsburgh, PennsylvaniaAges 18–80 - NCT06529731RecruitingPhase 2
Interferon-Gamma and Donor Cells for Relapsed Leukemia After Transplant
This trial tests combining a protein called interferon-gamma with an infusion of donor white blood cells to treat AML or MDS that returned after a stem cell transplant. The goal is to see if this combination helps the donor cells attack the cancer more effectively.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT07276035Recruiting
Brain-stomach circuits in chronic nausea
This study looks at how the brain and stomach communicate in people with chronic nausea. It uses a noninvasive brain stimulation method called TMS to understand brain activity. You may qualify if you have chronic nausea or are a healthy volunteer.
Pittsburgh, PennsylvaniaAges 21–60 - NCT07618364Recruiting
Rinsing to reduce eye inflammation in dry eye disease
This study tests whether rinsing the eye surface helps reduce inflammation and symptoms in people with dry eyes. Participants must have a specific inflammatory marker (MMP-9) in both eyes.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT04419649RecruitingPhase 2
Elritercept for anemia in lower-risk myelodysplastic syndromes
This trial tests elritercept, a medicine meant to improve anemia (low blood count) in adults with certain lower-risk bone marrow disorders called MDS. If you qualify, the study may help reduce the need for blood transfusions or improve anemia symptoms.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT02716246RecruitingPhase 2/Phase 3
Gene therapy for MPS IIIA to slow brain decline
This trial tests a gene-transfer treatment (delivered into the spinal fluid) for children with MPS IIIA, a rare genetic condition. It aims to improve or slow down brain and developmental changes caused by a missing enzyme.
Pittsburgh, PennsylvaniaAges Any age - NCT06825390Recruiting
Ear stimulation and brain imaging for chronic low back pain
This study tests if a non-invasive ear stimulation technique can change brain activity linked to chronic low back pain. You would have a brain scan before and after the treatment to see how your brain responds.
Pittsburgh, PennsylvaniaAges 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.
Pittsburgh, PennsylvaniaAges Any age - NCT05157581Recruiting
Light-based treatment for Sezary syndrome patients
This trial tests whether a special light treatment done outside the body (extracorporeal photopheresis) can help people with Sezary syndrome. It is meant to see if the treatment is safe and effective for this specific type and stage of skin lymphoma.
Pittsburgh, PennsylvaniaAges 18–100 - NCT07284641RecruitingPhase 2
Stem cell transplant for immune disorders
This trial tests a stem cell transplant to treat common variable immunodeficiency (CVID) and other immune problems. It aims to replace a faulty immune system with a healthy one from a donor.
Pittsburgh, PennsylvaniaAges 5–40 - NCT05979441Enrolling by invitationPhase 3
Long-term safety of efgartigimod injection for myositis
This study tests the long-term safety and effectiveness of a subcutaneous (under-the-skin) version of efgartigimod for adults with active idiopathic inflammatory myopathy. It is for people who have already completed an earlier study of this medicine.
Pittsburgh, PennsylvaniaAges 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.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06827262Recruiting
A procedure for vertebrogenic back pain relief
This trial tests a minimally invasive procedure that uses a special device to target and treat nerve-related back pain coming from the vertebrae (the bones in your spine). It may offer lasting relief without more major surgery.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT05947955RecruitingPhase 2
Study drug for infection-related lung failure in adults
This Phase 2 trial tests Rhu-pGSN in adults with acute respiratory distress syndrome (ARDS) caused by an infection. If you qualify, the study may help determine whether the drug improves outcomes in this short-term, life-threatening lung condition.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT00900198Recruiting
Give cancer tissue samples to help future cancer research
This study collects tissue or other cancer-related samples to help researchers better understand cancer and develop better tests or treatments. You can join if you are being evaluated for or treated for cancer, and you agree to let your samples be used for research.
Pittsburgh, PennsylvaniaAges 2 months+ - NCT05702645Recruiting
Down syndrome long-term study after leukemia treatment
This study follows children and young adults with Down syndrome who have survived acute leukemia (ALL or AML) to learn about their longer-term health after treatment ends. It may help families and doctors understand what health problems to watch for and how to provide better follow-up care.
Pittsburgh, PennsylvaniaAges 6–39 - NCT07565220RecruitingPhase 1
Thiotepa-based stem cell transplant for blood cancers
This trial tests a new chemotherapy preparation (using thiotepa) before a stem cell transplant from a matched donor to treat blood cancers like leukemia and myelodysplastic syndrome. The goal is to improve survival while reducing severe side effects.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT05905328RecruitingPhase 1/Phase 2
Drug to help prevent and treat cytokine release syndrome
This Phase 1/2 trial tests CTO1681 to help prevent and treat cytokine release syndrome (CRS) in people with DLBCL who are receiving CD19 CAR T-cell therapy. CRS is a potentially serious immune reaction that can happen after CAR T treatment, and this study aims to make that complication less severe or easier to manage.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT07625579Recruiting
Collaboration for Down Syndrome Progress Research Study
This research study aims to better understand Down syndrome by collecting health information, family history, and sometimes brain imaging or sleep data from children and adults with Down syndrome and their families. The goal is to advance knowledge that could improve health and development for people with Down syndrome.
Pittsburgh, PennsylvaniaAges Any age
Hear when a new Down Syndrome trial opens near Pittsburgh, PA
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Down Syndrome trials in other cities
Where these studies are running near Pittsburgh
Institutions with a site for the recruiting down syndrome studies listed above.
- University of Pittsburgh Medical Center · 8 studies
- UPMC Children's Hospital of Pittsburgh · 7 studies
- UPMC Hillman Cancer Center · 3 studies
- Allegheny General Hospital · 2 studies
- University of Pittsburgh · 2 studies
- Everett and Hurite Ophthalmic Association
- 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 Pennsylvania.
Common questions
- Are there down syndrome clinical trials near Pittsburgh?
- Yes. We're currently tracking 53 recruiting down syndrome studies with a site near Pittsburgh, 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 Pittsburgh?
- 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 Pittsburgh to take part?
- Not necessarily. These studies have a site near Pittsburgh, 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 Pittsburgh regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.