Clinical trials · Philadelphia, PA
Down Syndrome clinical trials in Philadelphia
We’re tracking 112 recruiting down syndrome studies with a site in or near Philadelphia — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 3 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- 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 - 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 - 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+ - NCT05677100Recruiting
Aortix device for acute heart failure
This trial is testing a small pump placed in the aorta to help the kidneys remove extra fluid when standard IV diuretics aren't working well enough for severe heart failure. It may help you if you're hospitalized and still very congested (swollen, fluid buildup) despite strong water pills.
Philadelphia, PennsylvaniaAges 21 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 - 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+ - 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+ - 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+ - 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.
Philadelphia, PennsylvaniaAges Any age - 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 - NCT07280468RecruitingPhase 4
Personalized sleep apnea treatment for Down syndrome
This study tests if a treatment chosen based on your specific type of sleep apnea works better for people with Down syndrome. It looks for participants who have sleep apnea but are not already using a CPAP machine regularly.
Philadelphia, PennsylvaniaAges 6 years+ - 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 - 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 - 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+ - NCT06066502RecruitingPhase 3
Precision ventilation for ARDS
This study tests whether a customized breathing machine approach ("precision ventilation") is better than usual care for patients with ARDS. The goal is to improve oxygen levels and recovery while avoiding lung damage.
Philadelphia, PennsylvaniaAges 18 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 - NCT05432648Recruiting
Fiber food introduction for children with short bowel syndrome
This trial tests if slowly adding fiber to the diet helps children with short bowel syndrome. It looks at how fiber affects digestion and overall health.
Philadelphia, PennsylvaniaAges 4 months–18 years - NCT06078553Recruiting
Natural history study of congenital myasthenic syndromes (CMS)
This study follows people with congenital myasthenic syndrome (CMS) caused by changes in certain genes (DOK7, MUSK, AGRN, or LRP4) over time. It does not test any new drug, but helps researchers understand how the condition changes and how to plan future treatments.
Philadelphia, PennsylvaniaAges 2 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+ - NCT05066217RecruitingPhase 3
Study tests clemizole for Lennox-Gastaut seizures
This Phase 3 trial studies whether clemizole helps reduce seizures in people with Lennox-Gastaut syndrome (LGS) and checks safety. If you have LGS and meet the seizure and heart-safety requirements, this could be an option to explore.
Philadelphia, PennsylvaniaAges 2–55 - 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 - 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+ - NCT05702645Recruiting
Down syndrome long-term study after leukemia treatment
This study follows children and young adults with Down syndrome who have survived acute leukemia (ALL or AML) to learn about their longer-term health after treatment ends. It may help families and doctors understand what health problems to watch for and how to provide better follow-up care.
Philadelphia, PennsylvaniaAges 6–39 - NCT06789913RecruitingPhase 2
Testing RLY-2608 for growth disorders linked to PIK3CA mutations
This trial tests a new medicine, RLY-2608, that targets the specific genetic change (PIK3CA mutation) causing overgrowth conditions. It aims to see if the drug can slow or stop the growth of affected tissues.
Philadelphia, PennsylvaniaAges 2 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 · 12 studies
- Alfred I. duPont Hospital for Children · 4 studies
- University of Pennsylvania · 4 studies
- Thomas Jefferson University Hospital · 3 studies
- Fox Chase Cancer Center
- Investigative Site
- Jefferson Abington Hospital
- Local Institution
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 112 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.