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, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- NCT05849662RecruitingPhase 1/Phase 2
Trametinib plus azacitidine for newly diagnosed JMML
This trial studies whether trametinib and azacitidine can control juvenile myelomonocytic leukemia (JMML) soon after diagnosis. It’s designed to test safety and how well the treatment works in children and young adults who meet specific leukemia and genetic criteria.
Philadelphia, PennsylvaniaAges 1 month–21 years - NCT06154252RecruitingPhase 2/Phase 3
Study of CABA-201 for active myositis in adults and children
This trial tests a new treatment called CABA-201, which uses your own immune cells modified to attack the overactive cells causing myositis. It is for people with active disease that hasn't improved enough with standard therapies.
Philadelphia, PennsylvaniaAges 6–75 - NCT07125079Recruiting
Studying immune cells and a new treatment for ARDS
This study looks at how the immune system responds in people with ARDS (a severe lung injury) and tests a new treatment called PIP-2 in the lab to see if it might help. It aims to better understand lung injury and find new ways to treat it.
Philadelphia, PennsylvaniaAges 21–90 - 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 - NCT00315380Recruiting
Study following people with eosinophilic granulomatosis with polyangiitis
This study looks at the health course of people who have eosinophilic granulomatosis with polyangiitis, a rare inflammatory condition. Your participation may help researchers better understand how the illness changes over time.
Philadelphia, PennsylvaniaAges Any age - NCT02967068Recruiting
Share your study tissue for future research
This study is a “tissue repository” that collects and stores leftover tissue from other approved VCRC studies. If you already took part in one of those studies, you may be able to let researchers use your tissue to help answer future questions.
Philadelphia, PennsylvaniaAges Any age - NCT03568669Recruiting
Testing thinking and learning in congenital central hypoventilation syndrome
This study looks at thinking and memory in people with congenital central hypoventilation syndrome (CCHS) to better understand how the condition affects the brain. It may help researchers measure changes over time and guide future care.
Philadelphia, PennsylvaniaAges 3–85 - NCT05320198RecruitingPhase 1/Phase 2
Testing DISC-0974 for anemia in myelofibrosis or MDS
This trial tests an investigational drug called DISC-0974 for people with myelofibrosis or certain types of MDS who have low red blood cell counts (anemia). The goal is to see if this treatment can help improve anemia and reduce the need for blood transfusions.
Philadelphia, PennsylvaniaAges 18 years+ - NCT03949855RecruitingPhase 2
Belimumab plus rituximab for primary membranous nephropathy
This study tests whether adding belimumab to rituximab helps people with primary membranous nephropathy (a kidney disease) that has the right blood test marker and specific protein levels. The goal is to improve kidney outcomes and reduce disease activity while keeping treatment safe.
Philadelphia, PennsylvaniaAges 18–75 - 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 - NCT03816345RecruitingPhase 1
Nivolumab for advanced cancer with autoimmune conditions
This Phase 1 study tests nivolumab, an immunotherapy “anti-cancer accelerator,” in people with advanced cancer who also have an autoimmune disorder. It aims to see if nivolumab can be given safely and whether it may help shrink or control cancer.
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+ - 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 - NCT05052606Recruiting
Turner syndrome patient registry study
This is a registry study that collects health information from people who have Turner syndrome. It helps researchers better understand the condition and guide future care.
Philadelphia, PennsylvaniaAges Any age - 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 - NCT01401998Recruiting
Study collecting information from people with ARPKD
This study looks at medical records, imaging, and genetic testing for people with autosomal recessive polycystic kidney disease (ARPKD). It may help researchers better understand the condition and how it affects the liver and kidneys.
Philadelphia, PennsylvaniaAges Up to 18 years - NCT00358943Recruiting
Gaucher disease registry and pregnancy tracking study
This study creates a large, long-term record of people with Gaucher disease and details about pregnancy outcomes. It helps researchers better understand Gaucher over a person’s life and during pregnancy.
Philadelphia, PennsylvaniaAges Any age - NCT02000089RecruitingPhase 3
Pancreas screening study for people at high cancer risk
This Phase 3 study tests a structured plan for checking the pancreas early in people with a higher-than-usual risk of pancreatic cancer. The goal is to find changes sooner, when treatment may be easier.
Philadelphia, PennsylvaniaAges 18 years+ - NCT02852031Recruiting
Care program for newborns needing the Norwood procedure
This trial focuses on improving care for babies born with certain complex heart conditions where only one pumping chamber works. It is meant for babies (including unborn babies) who are planned to have the Norwood procedure, which is a common early surgery.
Philadelphia, PennsylvaniaAges Up to 1.3 years - NCT02356653RecruitingEarly Phase 1
Stem cell transplant help for children lacking a matching sibling
This expanded-access study offers a specific type of stem cell transplant preparation that removes certain immune cells (CD3 and CD19) from donor stem cells. It is for children who need a transplant but do not have a fully matched sibling donor and whose team cannot use the hospital’s usual matching device protocol.
Philadelphia, PennsylvaniaAges Up to 30 years - NCT03533985Recruiting
Music therapy for newborns in the NICU with withdrawal symptoms
This study looks at whether music therapy can help infants who are born with neonatal abstinence syndrome (withdrawal symptoms after birth). It may support comfort, bonding, or overall newborn well-being while they’re in the NICU.
Philadelphia, PennsylvaniaAges 6 months+ - NCT04545424RecruitingPhase 2
Testing cooling treatment for severe lung failure in ICU patients
This trial tests whether therapeutic cooling (hypothermia) can improve outcomes in people with ARDS, a serious lung condition causing breathing failure. It may help by reducing harmful inflammation and lung injury, but eligibility is time-sensitive and requires ICU monitoring.
Philadelphia, PennsylvaniaAges 18–85 - NCT05267821RecruitingPhase 2/Phase 3
Testing anakinra to reduce inflammation in pediatric sepsis MODS
This study tests whether a medicine called anakinra can “turn down” harmful inflammation in children with severe illness from sepsis. It aims to improve organ function when multiple organs are starting to fail.
Philadelphia, PennsylvaniaAges 1 day–17 years - NCT04715399Recruiting
Research study collecting data for neurodegenerative diseases
This study collects health information from people with neurodegenerative diseases, some family members, and healthy controls to help researchers better understand these conditions. It may help future diagnosis and research, and participation may include noninvasive checks and other procedures depending on your situation.
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.
- University of Pennsylvania · 12 studies
- Children's Hospital of Philadelphia · 11 studies
- Temple University Hospital · 3 studies
- Cooper University Health Care · 2 studies
- Thomas Jefferson University Hospital · 2 studies
- Alfred I. duPont Hospital for Children
- Drexel University
- University of Pennsylavia
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.