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 2, Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- 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+ - NCT05979441Enrolling by invitationPhase 3
Long-term safety of efgartigimod injection for myositis
This study tests the long-term safety and effectiveness of a subcutaneous (under-the-skin) version of efgartigimod for adults with active idiopathic inflammatory myopathy. It is for people who have already completed an earlier study of this medicine.
Pittsburgh, PennsylvaniaAges 18 years+ - 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 - NCT05995535RecruitingPhase 2
Pregabalin and Lofexidine for Opioid Withdrawal
This study tests whether combining two medications, pregabalin and lofexidine, can help people stop using opioids by easing withdrawal symptoms. It's for adults who use opioids heavily and want to switch to a non-addictive blocker medication.
Philadelphia, PennsylvaniaAges 18 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+ - NCT05905328RecruitingPhase 1/Phase 2
New drug to prevent and treat side effects of CAR T-cell therapy
This study tests an experimental drug, CTO1681, that may help prevent or treat cytokine release syndrome (CRS) — a common side effect of CAR T-cell therapy. It could make the recovery period safer and more comfortable.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT05905926Enrolling by invitationPhase 3
Tenapanor safety study for teens with constipation-predominant IBS
This trial checks the safety of tenapanor in children and teens (ages 6 to under 18) with IBS-C. It is only for people who finished a previous related study and agreed to continue follow-up.
Scottdale, PennsylvaniaAges 6–18 - 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 - 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+ - 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 - NCT05701917Recruiting
Clot-busting device versus blood thinners for leg clot
This study compares a clot-removal device (ClotTriever) plus standard care versus standard blood thinners alone for a deep vein clot in the leg. It aims to see which approach better improves leg symptoms and outcomes.
Erie, PennsylvaniaAges 18 years+ - NCT05654922RecruitingPhase 3
Prevent lung transplant airways scarring with ARINA-1
This Phase 3 trial tests ARINA-1 to slow or prevent worsening lung transplant scarring in the breathing tubes (bronchiolitis obliterans). It may help people who are showing early decline in lung function but are not yet at advanced disease.
Philadelphia, PennsylvaniaAges 18–75 - 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 - NCT05564390RecruitingPhase 2
MyeloMATCH screening to match you to AML or MDS treatment
This study screens people with newly suspected acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) to see which treatment approach fits best—either a specific study treatment or standard care. It may help by directing you to the right option and by sharing your blood/bone marrow samples for research.
Allentown, PennsylvaniaAges 18 years+ - 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 - 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 - 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 - 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 - 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+ - NCT05267821RecruitingPhase 2/Phase 3
Testing anakinra to reduce inflammation in pediatric sepsis MODS
This study tests whether a medicine called anakinra can “turn down” harmful inflammation in children with severe illness from sepsis. It aims to improve organ function when multiple organs are starting to fail.
Hershey, PennsylvaniaAges 1 day–17 years - 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+ - 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 - 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+ - NCT04771130RecruitingPhase 1/Phase 2
Study of BGB-11417 for blood cancers
This early-phase study tests BGB-11417 in people with certain bone-marrow (blood) cancers such as AML, MDS, or MDS/MPN. The goal is to find out how the medicine works and whether it can control the cancer.
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 · 8 studies
- University of Pittsburgh Medical Center · 3 studies
- Allegheny General Health Research Network
- Allegheny Health Network
- Allegheny St. Vincent Hospital
- Children's Hospital of Pittsburgh
- Frontier Clinical Research
- Frontier Clinical Research, LLC
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.