Clinical trials · Pennsylvania
Down Syndrome clinical trials in Pennsylvania
We’re tracking 139 recruiting down syndrome studies with a site in Pennsylvania — including Early 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
- 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+ - 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+ - 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 - 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+ - NCT05320198RecruitingPhase 1/Phase 2
Testing DISC-0974 for anemia in myelofibrosis or MDS
This trial tests an investigational drug called DISC-0974 for people with myelofibrosis or certain types of MDS who have low red blood cell counts (anemia). The goal is to see if this treatment can help improve anemia and reduce the need for blood transfusions.
Gettysburg, PennsylvaniaAges 18 years+ - 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 - 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 - NCT05066217RecruitingPhase 3
Study tests clemizole for Lennox-Gastaut seizures
This Phase 3 trial studies whether clemizole helps reduce seizures in people with Lennox-Gastaut syndrome (LGS) and checks safety. If you have LGS and meet the seizure and heart-safety requirements, this could be an option to explore.
Philadelphia, PennsylvaniaAges 2–55 - 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 - 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+ - NCT04462770RecruitingPhase 3
Clemizole trial for children and adults with Dravet syndrome
This Phase 3 study tests EPX-100 (clemizole) to help reduce seizures in people with Dravet syndrome whose seizures are not fully controlled by current anti-seizure medicines. You may consider this study if your Dravet diagnosis is confirmed and your medical situation fits the specific rules about medicines and seizure causes.
Philadelphia, PennsylvaniaAges 2 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 - 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 - 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 - 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 - 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 - NCT02356653RecruitingEarly Phase 1
Stem cell transplant help for children lacking a matching sibling
This expanded-access study offers a specific type of stem cell transplant preparation that removes certain immune cells (CD3 and CD19) from donor stem cells. It is for children who need a transplant but do not have a fully matched sibling donor and whose team cannot use the hospital’s usual matching device protocol.
Philadelphia, PennsylvaniaAges Up to 30 years - NCT01852370Enrolling by invitationPhase 1/Phase 2
Lung and bone marrow transplant plan for immune system disorders
This early-phase study tests a step-by-step transplant approach: first replacing the lungs, then doing a bone marrow (stem cell) transplant to treat certain immune deficiency diseases. It may help people whose immune problem has led to severe, end-stage lung disease.
Pittsburgh, PennsylvaniaAges 5–45 - 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+ - NCT07421167RecruitingPhase 2
Ianalumab with TPO-RA for immune thrombocytopenia
This trial tests a new drug called ianalumab combined with a standard platelet-boosting medicine (TPO-RA) for adults with immune thrombocytopenia (ITP) or Evans syndrome. The goal is to see if the combination is well-tolerated and helps raise platelet counts.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06154252RecruitingPhase 2/Phase 3
Study of CABA-201 for active myositis in adults and children
This trial tests a new treatment called CABA-201, which uses your own immune cells modified to attack the overactive cells causing myositis. It is for people with active disease that hasn't improved enough with standard therapies.
Philadelphia, PennsylvaniaAges 6–75 - NCT03418857RecruitingPhase 1/Phase 2
Probiotics for weight- and blood-sugar–related gut health
This trial tests whether taking probiotics can improve gut health in people with early metabolic problems related to extra body fat, cholesterol, blood pressure, or blood sugar. It’s a Phase 1/2 study, meaning safety and early effectiveness are being tested.
University Park, PennsylvaniaAges 55–75 - NCT07798700Recruiting
Biofluid research for brain and movement disorders
This study collects blood, spinal fluid, and other samples from people with brain or movement disorders, their family members, and healthy volunteers. The goal is to find better ways to diagnose and understand these conditions.
Philadelphia, PennsylvaniaAges 18 years+ - 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+
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 · 6 studies
- University of Pennsylvania · 4 studies
- University of Pittsburgh Medical Center · 4 studies
- UPMC Children's Hospital of Pittsburgh · 2 studies
- Children's Hospital of Philadelphia - Accepting Young Adult and Juvenile Patients
- Everett and Hurite Ophthalmic Association
- Jefferson Health - Abington Hospital
- Penn Presbyterian 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.