Clinical trials · Pennsylvania
Down Syndrome clinical trials in Pennsylvania
We’re tracking 139 recruiting down syndrome studies with a site in Pennsylvania — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 3 and Phase 4 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 - 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+ - NCT07280468RecruitingPhase 4
Personalized sleep apnea treatment for Down syndrome
This study tests if a treatment chosen based on your specific type of sleep apnea works better for people with Down syndrome. It looks for participants who have sleep apnea but are not already using a CPAP machine regularly.
Philadelphia, PennsylvaniaAges 6 years+ - 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+ - 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.
Media, PennsylvaniaAges 13–45 - NCT06846606RecruitingPhase 1
Study of AUTX-703 for advanced blood cancers
This trial tests an experimental drug, AUTX-703, for people with certain blood cancers (AML or MDS) that have returned or not improved after other treatments. The goal is to see if the drug is safe and helps control the disease.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06852365RecruitingPhase 2
Birth control pill and resistant starch for PCOS
This trial tests whether adding a type of fiber (resistant starch) to a common birth control pill can improve health in women with PCOS. It aims to see if this combination helps with weight, hormones, and metabolism.
Philadelphia, PennsylvaniaAges 18–40 - 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.
Philadelphia, PennsylvaniaAges 5–20 - NCT06843811Enrolling by invitationPhase 2
Sirolimus for Leigh Syndrome
This trial tests if sirolimus can help people with Leigh syndrome. It aims to see if the drug can improve neurological symptoms and is looking for participants aged 6 months to 55 years.
Philadelphia, PennsylvaniaAges 6 months–55 years - NCT06839456RecruitingPhase 1/Phase 2
Stem cell addback to prevent infections after transplant
This trial tests whether adding back certain immune cells after a stem cell transplant can help prevent serious viral or fungal infections. It's for children and young adults up to age 25 who need a transplant for a blood cancer or other curable disease.
Philadelphia, PennsylvaniaAges 1 month–25 years - 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+ - NCT06730126RecruitingPhase 2
Soquelitinib for ALPS-FAS-related swelling and low blood counts
This trial tests an investigational drug called soquelitinib to see if it can shrink enlarged lymph nodes and improve low blood cell counts in people with ALPS-FAS. It may offer a new treatment option for those who have active symptoms.
Philadelphia, PennsylvaniaAges 16–120 - 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 - NCT06641414RecruitingPhase 3
Lisaftoclax with azacytidine for higher-risk myelodysplastic syndrome
This trial tests a new drug called lisaftoclax combined with a standard treatment, azacytidine, for people newly diagnosed with higher-risk myelodysplastic syndrome (MDS). The goal is to see if the combination helps control the disease better than what's currently available.
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.
Danville, 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+ - 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.
Philadelphia, PennsylvaniaAges 2–65 - 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 - 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 - 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 - NCT02000089RecruitingPhase 3
Pancreas screening study for people at high cancer risk
This Phase 3 study tests a structured plan for checking the pancreas early in people with a higher-than-usual risk of pancreatic cancer. The goal is to find changes sooner, when treatment may be easier.
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+ - NCT06066502RecruitingPhase 3
Precision ventilation for ARDS
This study tests whether a customized breathing machine approach ("precision ventilation") is better than usual care for patients with ARDS. The goal is to improve oxygen levels and recovery while avoiding lung damage.
Philadelphia, 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
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 · 3 studies
- Thomas Jefferson University · 2 studies
- UPMC Hillman Cancer Center · 2 studies
- Ascentage Investigative Site
- Biomed Testing Facility #BIO-17-19104
- Fox Chase 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 139 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.