Clinical trials · Pittsburgh, PA
Down Syndrome clinical trials in Pittsburgh
We’re tracking 55 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
- 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 - NCT02852031Recruiting
Care program for newborns needing the Norwood procedure
This trial focuses on improving care for babies born with certain complex heart conditions where only one pumping chamber works. It is meant for babies (including unborn babies) who are planned to have the Norwood procedure, which is a common early surgery.
Pittsburgh, PennsylvaniaAges Up to 1.3 years - NCT02848274Recruiting
Looking for factors that predict outcomes in advanced skin lymphoma
This study looks at medical and disease details to find factors that can help predict how advanced mycosis fungoides or Sézary syndrome will behave. Your information may help doctors better estimate prognosis and plan care.
Pittsburgh, PennsylvaniaAges Any age - 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+ - 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 - 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 - NCT04726241RecruitingPhase 1/Phase 2
Testing blood and bone marrow samples in young leukemia patients
This trial looks at blood and bone marrow samples from children and teens whose leukemia has come back or is hard to treat. The goal is to better understand the disease and guide future options for these patients.
Pittsburgh, PennsylvaniaAges Up to 22 years - NCT03047369Recruiting
Myelin brain disorder research biobank for samples and data
This study collects brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
Pittsburgh, PennsylvaniaAges Any age - NCT03970096RecruitingPhase 2
Reduce graft-versus-host disease after stem cell transplant
This Phase 2 study tests ways to lower the risk of graft-versus-host disease (GVHD), a serious complication after an allogeneic stem cell transplant from a matched donor. It is for people with acute leukemia or MDS who are going to receive intensive conditioning therapy, aiming to improve safety while keeping the transplant effective.
Pittsburgh, PennsylvaniaAges 1–60 - NCT04880876Enrolling by invitationPhase 3
Oral IBS-D medicine trial for kids and teens
This trial tests whether an oral medicine (eluxadoline) can help children and teens with IBS-D, a condition that causes stomach pain and frequent loose stools. It’s a Phase 3 study, meaning it’s designed to confirm benefits and safety.
Pittsburgh, PennsylvaniaAges 6–17 - NCT00177268Recruiting
Collecting blood and tissue samples in skin diseases research
This study collects blood, urine, and possibly small tissue samples to learn more about cutaneous T-cell lymphoma (CTCL) and either eczema or atopic dermatitis. It may help researchers better understand these skin conditions and improve future treatments.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT07519395RecruitingPhase 2
Camlipixant for IBS with diarrhea or mixed symptoms
This study tests an investigational drug, camlipixant, to see if it can reduce belly pain and diarrhea or mixed bowel symptoms in adults with irritable bowel syndrome (IBS). If you've tried other IBS treatments without enough relief, this might be an option.
Pittsburgh, PennsylvaniaAges 18–80 - 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+ - 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 - NCT06339177Recruiting
HLH study looking at immune system and genes
This study is for people with hemophagocytic lymphohistiocytosis (HLH), a condition where the immune system becomes overactive. Researchers want to collect medical data and blood samples to better understand HLH and find better ways to diagnose and treat it.
Pittsburgh, PennsylvaniaAges 18–120 - 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 - 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 - 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.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT04528355Recruiting
Study of hospital data after stem cell transplant for non-cancer illness
This study collects medical information from people with certain non-cancer conditions who receive a stem cell transplant. It may help doctors better understand transplant outcomes and care, since the study focuses on data collection rather than adding new treatment.
Pittsburgh, PennsylvaniaAges 2 months–60 years - 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).
Pittsburgh, PennsylvaniaAges 12 years+ - NCT04546399RecruitingPhase 2
Blinatumomab with or without nivolumab for first relapse ALL
This study compares blinatumomab alone versus blinatumomab plus nivolumab in young people whose B-cell ALL has returned for the first time. It aims to see whether adding nivolumab improves control of the leukemia after relapse.
Pittsburgh, PennsylvaniaAges 1–30 - 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 - NCT05564390RecruitingPhase 2
MyeloMATCH screening to match you to AML or MDS treatment
This study screens people with newly suspected acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) to see which treatment approach fits best—either a specific study treatment or standard care. It may help by directing you to the right option and by sharing your blood/bone marrow samples for research.
Pittsburgh, PennsylvaniaAges 18 years+ - 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+
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.
- UPMC Children's Hospital of Pittsburgh · 6 studies
- University of Pittsburgh Medical Center · 4 studies
- UPMC Hillman Cancer Center · 3 studies
- University of Pittsburgh · 2 studies
- Allegheny Health Network
- Children Hospital of Pittsburgh
- Children's Hospital of Pittsburgh
- Everett and Hurite Ophthalmic Association
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 55 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.