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 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
- 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 - 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 - 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+ - 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 - 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 - NCT03816345RecruitingPhase 1
Nivolumab for advanced cancer with autoimmune disease
This trial tests whether the immunotherapy drug nivolumab can safely treat advanced cancer in people who also have an autoimmune disease like rheumatoid arthritis, lupus, or multiple sclerosis. It aims to find out if the drug may help control cancer while managing their autoimmune condition.
Philadelphia, PennsylvaniaAges 18 years+ - NCT04068597RecruitingPhase 1/Phase 2
Testing inobrodib for blood cancers that came back
This is an early-stage study of inobrodib (CCS1477) for blood cancers like lymphoma, multiple myeloma, or acute myeloid leukemia that have returned or did not respond to standard treatment. It aims to see whether the drug is safe and can help control the cancer.
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 - 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+ - 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 - 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+ - NCT05432349Recruiting
Rett syndrome genetic registry for MECP2 changes
This trial is a registry that collects health and genetic information from people with Rett syndrome. It may help researchers better understand how MECP2 loss-of-function changes lead to symptoms and how to support patients.
Philadelphia, PennsylvaniaAges birth–99 years - 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 - 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+ - 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+ - 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 - 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.
Philadelphia, PennsylvaniaAges 45 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+ - 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+ - 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 - 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 - 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+ - NCT01429389Recruiting
Testing genetic samples in pregnancy to check chromosome changes
This study collects genetic samples from pregnant people who have a higher chance of chromosome changes in the baby. It tests whether taking samples through CVS and/or amniocentesis can help understand the baby’s genetic makeup.
Camden, New JerseyAges 18 years+ - NCT05795465RecruitingPhase 2
Study drug for people with acute respiratory distress syndrome
This Phase 2 trial tests GEn-1124 to see if it can improve outcomes in people with acute respiratory distress syndrome (ARDS). You may be eligible if the study drug can be started very soon after ARDS is diagnosed and if you meet the safety and health requirements.
Camden, New JerseyAges 18–85
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 · 8 studies
- Thomas Jefferson University Hospital · 6 studies
- Alfred I duPont Hospital for Children · 2 studies
- Cooper University Hospital · 2 studies
- Lehigh Valley Hospital-Cedar Crest · 2 studies
- University of Pennsylvania · 2 studies
- Jefferson Health - Abington Hospital
- Nemours at Thomas Jefferson University
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.