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 Phase 1/Phase 2, Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- 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+ - 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 - 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 - 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+ - NCT07083713Enrolling by invitation
Group coaching for life goals with chronic conditions
This study tests if group coaching helps people with certain chronic conditions set and achieve life goals. You would join phone or video sessions with a coach and others like you.
Philadelphia, PennsylvaniaAges 18 years+ - NCT07087054RecruitingPhase 3
A daily pill for carcinoid syndrome symptoms
This trial tests an oral daily medication, paltusotine, to reduce flushing and other symptoms of carcinoid syndrome in people with neuroendocrine tumors. It may be an option for those who need treatment and have stable disease.
Philadelphia, PennsylvaniaAges 18 years+ - 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 - NCT06668805RecruitingPhase 2
Testing vosoritide for children with Noonan syndrome and slow growth
This study tests a medication called vosoritide for children with Noonan syndrome who are still growing slowly despite using human growth hormone. The goal is to see if vosoritide can help them grow taller.
Philadelphia, PennsylvaniaAges 3–11 - 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+ - 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 - 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+ - 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 - 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 - NCT03396562Recruiting
Study of early development in babies with extra sex chromosomes
This study follows infants and young children with conditions caused by having an extra sex chromosome (like XXY, XYY, or XXX) to learn how health and development unfold over time. It may help families and doctors understand what to expect and what support could be helpful.
Philadelphia, PennsylvaniaAges 1 month–1.1 years - 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.
Philadelphia, PennsylvaniaAges 18 years+ - NCT07121140Recruiting
Home blood transfusions for cancer patients
This trial tests whether it is safe to give blood transfusions at home instead of in the hospital. It is for people who need regular blood transfusions for their cancer care.
Philadelphia, PennsylvaniaAges 18 years+ - NCT03452774Recruiting
AI matches your cancer tests to possible treatment trials
This trial uses an AI system to help match people with certain cancer types and specific tumor test results (like gene or protein markers) to relevant clinical trials. It may help you and your doctor find studies that fit your cancer biology faster.
Philadelphia, PennsylvaniaAges Any age - NCT03339128RecruitingPhase 2
Eluxadoline for diarrhea-predominant IBS in children
This trial tests whether eluxadoline can reduce symptoms of IBS with diarrhea (IBS-D) in children ages 6 to 17. It also asks families to track symptoms with a daily diary to measure whether treatment helps over the study period.
Philadelphia, PennsylvaniaAges 6–17 - NCT04546399RecruitingPhase 2
Blinatumomab with or without nivolumab for first relapse ALL
This study compares blinatumomab alone versus blinatumomab plus nivolumab in young people whose B-cell ALL has returned for the first time. It aims to see whether adding nivolumab improves control of the leukemia after relapse.
Philadelphia, PennsylvaniaAges 1–30
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
- Thomas Jefferson University Hospital · 5 studies
- University of Pennsylvania · 5 studies
- Alfred I. duPont Hospital for Children · 4 studies
- Lehigh Valley Hospital-Cedar Crest · 2 studies
- Celen Medical Group Corp
- Cooper University Health Care
- Fox Chase Cancer Center
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.