Clinical trials · Pennsylvania
Down Syndrome clinical trials in Pennsylvania
We’re tracking 141 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
- NCT04988555RecruitingPhase 1/Phase 2
Study of an investigational drug for high-risk acute leukemia
This Phase 1/2 study tests enzomenib (DSP-5336) in people with relapsed or hard-to-treat acute leukemia (and, in some places, certain other blood cancers). The goal is to see how the drug works and how safe it is, especially for leukemia cases with specific genetic changes (KMT2A/MLL or NPM1).
Philadelphia, PennsylvaniaAges 12 years+ - NCT05003986RecruitingPhase 2
Sparsentan for children with protein leaking kidney diseases
This Phase 2 study tests sparsentan in children with certain kidney diseases that cause high amounts of protein in the urine. It aims to see if the medicine can reduce kidney damage signals and protein leakage compared with standard care.
Philadelphia, PennsylvaniaAges 1–17 - NCT05031897RecruitingPhase 2
Haplo transplant with reduced donor mismatch and radiation
This trial tests a special bone marrow/stem cell transplant approach using a partially matched family donor, along with conditioning treatment that involves radiation. It aims to help treat certain blood cancers by replacing damaged blood-forming cells with new ones from the donor.
Philadephia, PennsylvaniaAges 18 years+ - NCT05194397RecruitingPhase 2
Exercise plus NR supplement to boost fitness after stem-cell transplant
This trial tests whether a supervised exercise program, plus an NR supplement, can improve fitness in young people who have had an allogeneic (donor) stem-cell transplant for certain blood cancers or bone marrow conditions. It’s meant to help with energy and physical ability while monitoring safety.
Philadelphia, PennsylvaniaAges 10–30 - NCT05219617RecruitingPhase 3
Carisbamate for frequent drop seizures in Lennox-Gastaut
This Phase 3 study tests whether carisbamate can reduce drop seizures in people with Lennox-Gastaut syndrome (LGS). It also checks whether the treatment is safe when added to existing seizure medicines.
Philadelphia, PennsylvaniaAges 4–55 - NCT05348564Recruiting
Family genetic screening methods for heart rhythm risk
This study compares two ways to find relatives who may carry a specific inherited genetic change. It may help your family learn faster who should get follow-up testing and care.
Lancaster, PennsylvaniaAges 18 years+ - NCT05382754Recruiting
Home sleep testing for children being checked for sleep apnea
This trial tests whether home sleep testing can help diagnose sleep apnea in children. It is for kids who are already being referred for an in-lab sleep study at CHOP.
Philadelphia, PennsylvaniaAges 5–12 - NCT05583942Recruiting
Nerve stimulation trial for children with steroid-resistant kidney syndrome
This pilot study tests a device that stimulates nerves in the ear to help children with steroid-resistant nephrotic syndrome. It may improve urine protein and kidney-related outcomes for kids whose standard steroid treatment does not work.
Philadelphia, PennsylvaniaAges 3–17 - NCT05588063Recruiting
Device-based nerve stimulation for relapse-preventing kidney disease in kids
This trial tests whether a small non-surgical device can help keep children with steroid-sensitive nephrotic syndrome in remission. If it works, it could lower the chance of relapse (kidney flare-ups) and help maintain kidney health.
Philadelphia, PennsylvaniaAges 3–17 - NCT05819697Recruiting
Helping burning mouth symptoms with improved memory recall
This trial looks at ways to reduce how much people with burning mouth syndrome struggle to recall information during the study. If you qualify, you may be asked to complete study tasks and share saliva to support the research.
Philadelphia, PennsylvaniaAges 18 years+ - NCT05849662RecruitingPhase 1/Phase 2
Trametinib plus azacitidine for newly diagnosed JMML
This trial studies whether trametinib and azacitidine can control juvenile myelomonocytic leukemia (JMML) soon after diagnosis. It’s designed to test safety and how well the treatment works in children and young adults who meet specific leukemia and genetic criteria.
Philadelphia, PennsylvaniaAges 1 month–21 years - NCT05918861RecruitingPhase 3
Tested heart-risk drug for people with recent heart-attack genetics
This trial studies whether dalcetrapib can lower future heart and blood-vessel risk in people who recently had an ACS (acute coronary syndrome, often a heart attack or unstable chest pain) and have a specific genetic marker. It may help people like you by targeting a risk pathway related to that genetic background.
Allentown, PennsylvaniaAges 45 years+ - NCT05975671Recruiting
Survey in pediatric ICU to reduce upfront vancomycin use
This study is a survey and quality-improvement effort in participating children’s intensive care units. It aims to understand and change how doctors decide to start vancomycin (an antibiotic) when sepsis is suspected, to improve care and avoid unnecessary use.
Philadelphia, PennsylvaniaAges Any age - NCT05978141Recruiting
T-cell lymphoma registry study
This study creates a registry for people with certain T-cell or NK-cell lymphomas. It collects health information and tumor samples to learn more about these diseases and improve future treatments.
Philadelphia, PennsylvaniaAges Any age - 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 - NCT06193928Recruiting
Long-term safety study of Livmarli in ALGS or PFIC
This study is following patients who are already taking Livmarli (maralixibat) for Alagille syndrome or PFIC to see how safe and effective it is over the long term.
Pittsburgh, PennsylvaniaAges Any age - NCT06288672Recruiting
Skin patch test for food allergies in IBS
This study tests whether a skin patch can help identify food allergies in people with irritable bowel syndrome (IBS) whose symptoms are not well controlled. It involves avoiding certain foods and using patches on your back to see if you react.
North Wales, PennsylvaniaAges 18 years+ - NCT06112418Recruiting
Personalized vs standard care to prevent heart attacks
This trial compares two ways to prevent heart attacks and strokes: one uses a special heart CT scan to guide care, the other uses standard risk factors like blood pressure and cholesterol. You may be eligible if you are older (men over 55, women over 65) and have diabetes, prediabetes, or metabolic syndrome.
Camp Hill, PennsylvaniaAges 55 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 - NCT06576323Recruiting
Support for mothers with opioid use disorder and babies in the NICU
This study aims to improve care for mothers with opioid use disorder and their babies with neonatal abstinence syndrome. Researchers will interview healthcare providers and mothers to develop better support programs.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06789445RecruitingPhase 1/Phase 2
Cell therapy for inherited retinal disease (CLARICO)
This study tests a new cell therapy (OpCT-001) for adults with inherited retinal diseases that affect the light-sensing cells. It aims to see if the treatment is safe and possibly helps improve vision.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06760546RecruitingPhase 3
Testing setmelanotide for weight gain after brain injury
This trial tests a daily injection called setmelanotide to see if it can help people who have gained weight due to a brain injury or condition affecting the hypothalamus. It may help reduce hunger and support weight loss.
Philadelphia, PennsylvaniaAges 4 years+ - NCT06859918Recruiting
Social skills program for adults with autism
This study tests a social and emotional program called TUNE In 3.0 for adults with autism. It is designed to help improve social connections and emotional well-being.
Philadelphia, PennsylvaniaAges 18 years+ - 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+
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 · 12 studies
- University of Pennsylvania · 4 studies
- Thomas Jefferson University Hospital · 2 studies
- UPMC Children's Hospital of Pittsburgh · 2 studies
- Allegheny Health Network
- Capital Area Research, LLC
- Clinical Research Site
- North Wales Dermatology, PLLC
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 141 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.