Clinical trials · Philadelphia, PA
Down Syndrome clinical trials in Philadelphia
We’re tracking 111 recruiting down syndrome studies with a site in or near Philadelphia — including Early Phase 1, Phase 1, Phase 1/Phase 2, Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- 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 - NCT06852365RecruitingPhase 2
Birth control pill and resistant starch for PCOS
This trial tests whether adding a type of fiber (resistant starch) to a common birth control pill can improve health in women with PCOS. It aims to see if this combination helps with weight, hormones, and metabolism.
Philadelphia, PennsylvaniaAges 18–40 - 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.
Philadelphia, PennsylvaniaAges Any age - 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.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06247787RecruitingPhase 1
Imetelstat plus chemo for relapsed leukemia in kids
This trial tests a new drug called imetelstat combined with two chemotherapy drugs (fludarabine and cytarabine) for children and teens with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) that has come back or not responded to treatment. The goal is to find the safest dose of imetelstat and see if it helps control the cancer.
Philadelphia, PennsylvaniaAges 1–18 - 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 - NCT05306080RecruitingEarly Phase 1
Rescue treatment for CAR T side effects in adults
This trial tests a rescue medicine given to treat severe immune over-reactions that can happen after CAR T cell therapy, such as cytokine release syndrome and an HLH-like syndrome. It may help calm the immune response if you are already enrolled in a specific CAR T study.
Philadelphia, PennsylvaniaAges 18 years+ - 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+ - 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+ - NCT06043440RecruitingPhase 2
Oxygen therapy for sleep apnea in Down syndrome
This study tests whether oxygen therapy can help children with Down syndrome who have sleep apnea. It compares oxygen treatment to no treatment to see if it improves breathing during sleep and thinking skills.
Philadelphia, PennsylvaniaAges 5–18 - 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+ - NCT06914609RecruitingPhase 3
Study of ION582 for people with Angelman syndrome
This trial tests a new medicine called ION582 for people with Angelman syndrome. It aims to see if it can help with symptoms and is given as a lumbar puncture (spinal tap). You may be able to join if you have a genetic diagnosis of Angelman syndrome and are between 2 and 50 years old.
Philadelphia, PennsylvaniaAges 2–50 - NCT06930417Recruiting
Williams syndrome and related 7q11.23 changes study
This study follows people with Williams syndrome, 7q11.23 duplication, or related genetic changes over time to understand how these conditions affect health and development. It also includes close family members to learn about the genetics.
Philadelphia, PennsylvaniaAges Any age - 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+ - 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 - 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 - 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 - NCT06730126RecruitingPhase 2
Soquelitinib for ALPS-FAS-related swelling and low blood counts
This trial tests an investigational drug called soquelitinib to see if it can shrink enlarged lymph nodes and improve low blood cell counts in people with ALPS-FAS. It may offer a new treatment option for those who have active symptoms.
Philadelphia, PennsylvaniaAges 16–120 - 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+ - NCT07003100Recruiting
Studying mogamulizumab rash in mycosis fungoides or sezary syndrome
This study looks at a common skin rash caused by the drug mogamulizumab in patients with certain types of lymphoma. It aims to learn more about the rash and how to manage it, so your experience can help future patients.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06371417RecruitingPhase 1
Testing RAY121 for several immune diseases (RAINBOW trial)
This trial tests a new medicine called RAY121 (an antibody) for people with certain autoimmune diseases. It aims to see if RAY121 is safe and how the body handles it. If you have one of these conditions and meet the basic health requirements, you might be able to join.
Philadelphia, PennsylvaniaAges 18–85 - NCT06843811Enrolling by invitationPhase 2
Sirolimus for Leigh Syndrome
This trial tests if sirolimus can help people with Leigh syndrome. It aims to see if the drug can improve neurological symptoms and is looking for participants aged 6 months to 55 years.
Philadelphia, PennsylvaniaAges 6 months–55 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 - 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+
Hear when a new Down Syndrome trial opens near Philadelphia, PA
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Down Syndrome trials in other cities
Where these studies are running near Philadelphia
Institutions with a site for the recruiting down syndrome studies listed above.
- Children's Hospital of Philadelphia · 11 studies
- University of Pennsylvania · 7 studies
- Thomas Jefferson University Hospital · 4 studies
- Alfred I. duPont Hospital for Children
- Cooper University Health Care
- Perelman Center for Advanced Medicine
- Temple University Hospital
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.
Looking wider? See all recruiting down syndrome studies in Pennsylvania.
Common questions
- Are there down syndrome clinical trials near Philadelphia?
- Yes. We're currently tracking 111 recruiting down syndrome studies with a site near Philadelphia, 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 near Philadelphia?
- 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 Philadelphia to take part?
- Not necessarily. These studies have a site near Philadelphia, 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 near Philadelphia regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.