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, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT06994676RecruitingPhase 1
Study of CBX-250 for advanced myeloid leukemias
This study tests an experimental drug, CBX-250, for people with certain advanced blood cancers (like AML) that have not responded to standard treatments or have returned. The drug works only for people who have a specific genetic marker (HLA-A*02:01). The goal is to see if it is safe and how well it works.
Philadelphia, PennsylvaniaAges 12 years+ - NCT07038200RecruitingPhase 3
Study of Del-brax (AOC 1020) for People with FSHD
This study tests an experimental medicine called Del-brax (AOC 1020) for people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to see if it can help improve muscle strength and slow the disease. You may be able to join if you have a confirmed FSHD diagnosis and can walk at least 10 meters on your own.
Philadelphia, PennsylvaniaAges 16–70 - NCT07121140Recruiting
Home blood transfusions for cancer patients
This trial tests whether it is safe to give blood transfusions at home instead of in the hospital. It is for people who need regular blood transfusions for their cancer care.
Philadelphia, PennsylvaniaAges 18 years+ - NCT07125079Recruiting
Studying immune cells and a new treatment for ARDS
This study looks at how the immune system responds in people with ARDS (a severe lung injury) and tests a new treatment called PIP-2 in the lab to see if it might help. It aims to better understand lung injury and find new ways to treat it.
Philadelphia, PennsylvaniaAges 21–90 - NCT07283341Recruiting
Diet and Gut Movement in Irritable Bowel Syndrome
This study looks at how diet affects gut movement in children with irritable bowel syndrome (IBS). It may help find better ways to manage IBS symptoms.
Philadelphia, PennsylvaniaAges 8–18 - NCT07565220RecruitingPhase 1
Thiotepa-based stem cell transplant for blood cancers
This trial tests a new chemotherapy preparation (using thiotepa) before a stem cell transplant from a matched donor to treat blood cancers like leukemia and myelodysplastic syndrome. The goal is to improve survival while reducing severe side effects.
Pittsburgh, PennsylvaniaAges 18 years+ - 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+ - 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.
Pittsburgh, PennsylvaniaAges Any age - 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+ - NCT01962415RecruitingPhase 2
Reduced-intensity stem cell transplant for non-cancer disorders
This trial tests whether a gentler (reduced-intensity) conditioning treatment before a stem cell transplant works for non-cancer diseases. It uses specific donor stem cell matches and checks heart, lung, liver, and kidney function to help lower transplant stress while still aiming for benefit.
Pittsburgh, PennsylvaniaAges 2 months–55 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 - 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 - 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.
Harrisburg, 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 - 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 - 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 - 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 - 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+ - NCT07083713Enrolling by invitation
Group coaching for life goals with chronic conditions
This study tests if group coaching helps people with certain chronic conditions set and achieve life goals. You would join phone or video sessions with a coach and others like you.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06472765Enrolling by invitation
Study of the Vaginal Ecosystem in Healthy Women
This study looks at the natural balance of bacteria and other factors in the vagina of healthy women. It aims to learn more about what a healthy vaginal ecosystem looks like.
Bryn Mawr, PennsylvaniaAges 18–89 - NCT05594992Enrolling by invitationPhase 3
Study extension of JR-141 for long-term Hunter syndrome safety
This is a follow-up study for people who already took JR-141 in an earlier trial for Hunter syndrome (MPS II). It checks how well the treatment works over the long term and monitors long-term safety.
Philadelphia, 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
- UPMC Children's Hospital of Pittsburgh · 5 studies
- University of Pennsylvania · 4 studies
- Thomas Jefferson University Hospital · 3 studies
- University of Pittsburgh · 3 studies
- University of Pittsburgh Medical Center · 2 studies
- Center for Pelvic Medicine
- 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.
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.