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 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT04715399Recruiting
Research study collecting data for neurodegenerative diseases
This study collects health information from people with neurodegenerative diseases, some family members, and healthy controls to help researchers better understand these conditions. It may help future diagnosis and research, and participation may include noninvasive checks and other procedures depending on your situation.
Philadelphia, 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 - 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.
Hershey, PennsylvaniaAges 1–30 - NCT04064060RecruitingPhase 3
Long-term safety follow-up for luspatercept study participants
This trial studies the long-term safety of luspatercept (ACE-536) in people who already took part in an earlier luspatercept study. It mainly helps doctors better understand ongoing side effects and overall safety over time.
Philadelphia, PennsylvaniaAges 18 years+ - NCT04068597RecruitingPhase 1/Phase 2
Testing inobrodib for blood cancers that came back
This is an early-stage study of inobrodib (CCS1477) for blood cancers like lymphoma, multiple myeloma, or acute myeloid leukemia that have returned or did not respond to standard treatment. It aims to see whether the drug is safe and can help control the cancer.
Philadelphia, PennsylvaniaAges 18 years+ - NCT03949855RecruitingPhase 2
Belimumab plus rituximab for primary membranous nephropathy
This study tests whether adding belimumab to rituximab helps people with primary membranous nephropathy (a kidney disease) that has the right blood test marker and specific protein levels. The goal is to improve kidney outcomes and reduce disease activity while keeping treatment safe.
Philadelphia, PennsylvaniaAges 18–75 - NCT02967068Recruiting
Share your study tissue for future research
This study is a “tissue repository” that collects and stores leftover tissue from other approved VCRC studies. If you already took part in one of those studies, you may be able to let researchers use your tissue to help answer future questions.
Philadelphia, PennsylvaniaAges Any age - 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.
Philadelphia, PennsylvaniaAges 18 years+ - NCT03810196Recruiting
Stem cell addback after transplant to treat viral or fungal infection
This study tests a stem cell “addback” treatment after a specific type of transplant (with certain immune cells removed). It may help the body recover faster and fight serious viral or fungal infections in children and teens.
Philadelphia, PennsylvaniaAges Up to 25 years - NCT03533985Recruiting
Music therapy for newborns in the NICU with withdrawal symptoms
This study looks at whether music therapy can help infants who are born with neonatal abstinence syndrome (withdrawal symptoms after birth). It may support comfort, bonding, or overall newborn well-being while they’re in the NICU.
Philadelphia, PennsylvaniaAges 6 months+ - 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 - NCT02780297Recruiting
Study of rare stone-causing kidney conditions
This study is for people with certain rare kidney conditions that can cause kidney stones. It aims to learn how these conditions behave over time and may help improve future care.
Philadelphia, PennsylvaniaAges Any age - NCT03011814RecruitingPhase 1/Phase 2
Durvalumab plus lenalidomide for relapsed T-cell lymphoma
This study tests a combination of two drugs—durvalumab (an immunotherapy) and lenalidomide (a targeted therapy)—to see if they are safe and effective for people with T-cell lymphoma that has come back or not responded to treatment. It aims to harness your immune system to fight the cancer while also attacking the cancer cells directly.
Philadelphia, PennsylvaniaAges 18 years+ - NCT02646839Enrolling by invitationPhase 2
Testing a gene-matched transplant approach for childhood leukemia
This trial studies a specific type of partially matched (haploidentical) stem cell transplant in children with ALL, AML, or MDS. It looks at whether using certain immune system gene patterns (KIR) and measuring leftover disease after treatment can improve outcomes and cost-effectiveness.
Philadelphia, PennsylvaniaAges Up to 21 years - NCT00358943Recruiting
Gaucher disease registry and pregnancy tracking study
This study creates a large, long-term record of people with Gaucher disease and details about pregnancy outcomes. It helps researchers better understand Gaucher over a person’s life and during pregnancy.
Hershey, PennsylvaniaAges Any age - NCT01209000Recruiting
Study for children with new kidney protein loss
This study is for children (and some newly diagnosed patients) who have nephrotic syndrome—kidneys leaking a lot of protein. It aims to better understand the kidney condition (sometimes using biopsy) and support future care decisions.
Philadelphia, PennsylvaniaAges Up to 80 years - NCT00315380Recruiting
Study following people with eosinophilic granulomatosis with polyangiitis
This study looks at the health course of people who have eosinophilic granulomatosis with polyangiitis, a rare inflammatory condition. Your participation may help researchers better understand how the illness changes over time.
Philadelphia, PennsylvaniaAges Any age - NCT01276470Recruiting
Study of environmental triggers in anti-synthetase syndrome
This study looks for environmental risk factors that may be linked to anti-synthetase syndrome, a type of inflammatory muscle disease. It compares people with the condition to carefully matched people without it, using questionnaires, blood samples, and lung imaging when needed.
Pittsburgh, PennsylvaniaAges 2–100 - 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
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 · 7 studies
- University of Pennsylvania · 4 studies
- UPMC Children's Hospital of Pittsburgh · 4 studies
- University of Pittsburgh · 3 studies
- Thomas Jefferson University Hospital · 2 studies
- Children's Hospital of Pittsburgh
- Drexel University/Hahnemann Hospital-College of Nursing and Health Professions
- Geisinger Medical 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.