Clinical trials · Philadelphia, PA
Down Syndrome clinical trials in Philadelphia
We’re tracking 108 recruiting down syndrome studies with a site in or near Philadelphia — including Phase 1/Phase 2, Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- 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+ - NCT06840496RecruitingPhase 3
Study of oral NA-921 vs placebo for girls and women with Rett syndrome
This trial tests an oral medication called NA-921 in females with Rett syndrome to see if it works better than a placebo. It may be an option for those who have a confirmed MECP2 gene mutation and stable seizure patterns.
Philadelphia, PennsylvaniaAges 5–20 - NCT02780297Recruiting
Study of rare stone-causing kidney conditions
This study is for people with certain rare kidney conditions that can cause kidney stones. It aims to learn how these conditions behave over time and may help improve future care.
Philadelphia, PennsylvaniaAges Any age - 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 - 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+ - 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 - NCT05932589Recruiting
Study of brain signals as signs of Rett syndrome
This study measures brain and nerve activity (brain signals) to find patterns that match Rett syndrome. It may help researchers better track Rett changes over time and develop more precise ways to measure response in future treatments.
Philadelphia, PennsylvaniaAges 1–18 - NCT07625579Recruiting
Collaboration for Down Syndrome Progress Research Study
This research study aims to better understand Down syndrome by collecting health information, family history, and sometimes brain imaging or sleep data from children and adults with Down syndrome and their families. The goal is to advance knowledge that could improve health and development for people with Down syndrome.
Philadelphia, PennsylvaniaAges Any age - NCT04064060RecruitingPhase 3
Long-term safety follow-up for luspatercept study participants
This trial studies the long-term safety of luspatercept (ACE-536) in people who already took part in an earlier luspatercept study. It mainly helps doctors better understand ongoing side effects and overall safety over time.
Philadelphia, PennsylvaniaAges 18 years+ - 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 - 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 - 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.
Philadelphia, PennsylvaniaAges Any age - NCT07742735RecruitingPhase 3
Study of apraglutide for short bowel syndrome with intestinal failure
This study tests a new medicine called apraglutide, given as a shot, to see if it can help adults with short bowel syndrome and intestinal failure reduce their need for intravenous nutrition (parenteral support). The study compares apraglutide to a placebo (a shot with no medicine) to check if it is safe and effective.
Philadelphia, 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+ - NCT06679855RecruitingPhase 3
Milrinone to prevent heart problems after PDA closure
This trial tests if giving milrinone before surgery to close a common heart vessel (PDA) can prevent heart issues in premature babies. It may help very premature infants who need surgery for a large PDA.
Philadelphia, PennsylvaniaAges Up to 3 months - 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 - NCT04571658Recruiting
NEPTUNE Match study linking you to possible kidney research trials
This study is an add-on process that uses information from the NEPTUNE kidney study to see whether you might be able to join other kidney research trials. It may help connect eligible people with appropriate new treatment studies when they become available.
Philadelphia, PennsylvaniaAges 1–80 - NCT04726241RecruitingPhase 1/Phase 2
Testing blood and bone marrow samples in young leukemia patients
This trial looks at blood and bone marrow samples from children and teens whose leukemia has come back or is hard to treat. The goal is to better understand the disease and guide future options for these patients.
Philadelphia, PennsylvaniaAges Up to 22 years - NCT03047369Recruiting
Myelin brain disorder research biobank for samples and data
This study collects brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
Philadelphia, PennsylvaniaAges Any age - 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 - 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 - NCT07422480RecruitingPhase 3
Comparing elritercept to epoetin alfa for anemia in MDS needing transfusions
This trial tests a new drug called elritercept against a standard treatment, epoetin alfa, for people with a low-risk form of MDS who need regular blood transfusions for anemia. The goal is to see if elritercept works better to reduce the need for transfusions.
Philadelphia, PennsylvaniaAges 18 years+ - 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 - 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
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 · 13 studies
- University of Pennsylvania · 6 studies
- Hospital of the University of Pennsylvania · 2 studies
- Temple University Hospital · 2 studies
- Alfred I duPont Hospital for Children
- Biomed Testing Facility #BIO-17-19104
- Fox Chase Cancer Center
- Lehigh Valley Hospital-Cedar Crest
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 108 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.