Clinical trials · Pennsylvania
Down Syndrome clinical trials in Pennsylvania
We’re tracking 142 recruiting down syndrome studies with a site in Pennsylvania — including Phase 1, Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- 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.
King of Prussia, PennsylvaniaAges 22–80 - NCT06679855RecruitingPhase 3
Milrinone to prevent heart problems after PDA closure
This trial tests if giving milrinone before surgery to close a common heart vessel (PDA) can prevent heart issues in premature babies. It may help very premature infants who need surgery for a large PDA.
Philadelphia, PennsylvaniaAges Up to 3 months - NCT07278375Recruiting
Comparing treatments for babies with opioid withdrawal
This study looks at different medicine approaches to treat newborns with opioid withdrawal (NOWS). It compares how well babies do with standard care and may help find the best way to manage symptoms and support recovery.
Philadelphia, PennsylvaniaAges birth+ - NCT07742735RecruitingPhase 3
Study of apraglutide for short bowel syndrome with intestinal failure
This study tests a new medicine called apraglutide, given as a shot, to see if it can help adults with short bowel syndrome and intestinal failure reduce their need for intravenous nutrition (parenteral support). The study compares apraglutide to a placebo (a shot with no medicine) to check if it is safe and effective.
Philadelphia, 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.
Danville, PennsylvaniaAges 18 years+ - NCT05677100Recruiting
Aortix device for acute heart failure
This trial is testing a small pump placed in the aorta to help the kidneys remove extra fluid when standard IV diuretics aren't working well enough for severe heart failure. It may help you if you're hospitalized and still very congested (swollen, fluid buildup) despite strong water pills.
Abington, PennsylvaniaAges 21 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+ - NCT06371417RecruitingPhase 1
Testing RAY121 for several immune diseases (RAINBOW trial)
This trial tests a new medicine called RAY121 (an antibody) for people with certain autoimmune diseases. It aims to see if RAY121 is safe and how the body handles it. If you have one of these conditions and meet the basic health requirements, you might be able to join.
Philadelphia, PennsylvaniaAges 18–85 - NCT07422480RecruitingPhase 3
Comparing elritercept to epoetin alfa for anemia in MDS needing transfusions
This trial tests a new drug called elritercept against a standard treatment, epoetin alfa, for people with a low-risk form of MDS who need regular blood transfusions for anemia. The goal is to see if elritercept works better to reduce the need for transfusions.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06078553Recruiting
Natural history study of congenital myasthenic syndromes (CMS)
This study follows people with congenital myasthenic syndrome (CMS) caused by changes in certain genes (DOK7, MUSK, AGRN, or LRP4) over time. It does not test any new drug, but helps researchers understand how the condition changes and how to plan future treatments.
Philadelphia, PennsylvaniaAges 2 years+ - NCT03339128RecruitingPhase 2
Eluxadoline for diarrhea-predominant IBS in children
This trial tests whether eluxadoline can reduce symptoms of IBS with diarrhea (IBS-D) in children ages 6 to 17. It also asks families to track symptoms with a daily diary to measure whether treatment helps over the study period.
Pittsburgh, PennsylvaniaAges 6–17 - NCT03568669Recruiting
Testing thinking and learning in congenital central hypoventilation syndrome
This study looks at thinking and memory in people with congenital central hypoventilation syndrome (CCHS) to better understand how the condition affects the brain. It may help researchers measure changes over time and guide future care.
Philadelphia, PennsylvaniaAges 3–85 - 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 - NCT04165109Recruiting
Down syndrome study to test memory and thinking changes
This study enrolls adults with Down syndrome to better understand how thinking and memory develop and change over time. It may help by building a “trial-ready” group and by measuring thinking in a careful, consistent way.
Pittsburgh, PennsylvaniaAges 25–55 - NCT04278404Recruiting
Drug safety study for children already on certain medicines
This study measures how certain medicines move through the body and checks their safety in children. It may help doctors better understand dosing and side effects in kids who are already receiving these treatments, or in some kids who recently tested positive for COVID-19.
Hershey, PennsylvaniaAges birth–20 years - NCT04367246Recruiting
TP53 (Li-Fraumeni) family biobank and sample study
This study collects and stores health information and biological samples from people who have a known TP53 change (Li-Fraumeni syndrome) and from close family members. The goal is to help researchers learn more about risks and future treatments for this genetic condition.
Phildelphia, PennsylvaniaAges Any age - 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+ - NCT05052606Recruiting
Turner syndrome patient registry study
This is a registry study that collects health information from people who have Turner syndrome. It helps researchers better understand the condition and guide future care.
Philadelphia, 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 - 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.
Philadelphia, 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.
Hershey, PennsylvaniaAges 18 years+ - NCT06043440RecruitingPhase 2
Oxygen therapy for sleep apnea in Down syndrome
This study tests whether oxygen therapy can help children with Down syndrome who have sleep apnea. It compares oxygen treatment to no treatment to see if it improves breathing during sleep and thinking skills.
Philadelphia, PennsylvaniaAges 5–18 - 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.
Philadelphia, PennsylvaniaAges 1–18 - NCT05432648Recruiting
Fiber food introduction for children with short bowel syndrome
This trial tests if slowly adding fiber to the diet helps children with short bowel syndrome. It looks at how fiber affects digestion and overall health.
Philadelphia, PennsylvaniaAges 4 months–18 years
Down Syndrome trials by city in Pennsylvania
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting down syndrome studies listed above.
- Children's Hospital of Philadelphia · 9 studies
- University of Pennsylvania · 4 studies
- University of Pittsburgh Medical Center · 3 studies
- Thomas Jefferson University Hospital · 2 studies
- UPMC Hillman Cancer Center · 2 studies
- Fox Chase Cancer Center
- Geisinger Medical Center
- Geisinger Wyoming Valley/Henry Cancer Center
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 Pennsylvania?
- Yes. We're currently tracking 142 recruiting down syndrome studies with a site in Pennsylvania, 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 Pennsylvania?
- 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 Pennsylvania to take part?
- Not necessarily. These studies have a site in Pennsylvania, 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 Pennsylvania 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.