Clinical trials · Pennsylvania
Down Syndrome clinical trials in Pennsylvania
We’re tracking 141 recruiting down syndrome studies with a site in Pennsylvania — including Early Phase 1, Phase 1/Phase 2, Phase 2, Phase 2/Phase 3, Phase 3 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT06430385RecruitingPhase 1/Phase 2
Study of ION440 for MECP2 Duplication Syndrome
This trial tests an experimental medicine called ION440 for people with MECP2 Duplication Syndrome. The medicine is given as an injection into the spinal fluid, and the study aims to check its safety and how well it works.
Philadelphia, PennsylvaniaAges 2–65 - 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 - NCT06690801Recruiting
Nitric oxide breathing gas for children with lung failure
This trial tests whether adding a breathing gas called nitric oxide to standard care helps children with severe lung failure (ARDS) recover faster. It is for children on a breathing machine who still have low oxygen levels.
Philadelphia, PennsylvaniaAges birth–21 years - NCT06730126RecruitingPhase 2
Soquelitinib for ALPS-FAS-related swelling and low blood counts
This trial tests an investigational drug called soquelitinib to see if it can shrink enlarged lymph nodes and improve low blood cell counts in people with ALPS-FAS. It may offer a new treatment option for those who have active symptoms.
Philadelphia, PennsylvaniaAges 16–120 - NCT06825390Recruiting
Ear stimulation and brain imaging for chronic low back pain
This study tests if a non-invasive ear stimulation technique can change brain activity linked to chronic low back pain. You would have a brain scan before and after the treatment to see how your brain responds.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06839456RecruitingPhase 1/Phase 2
Stem cell addback to prevent infections after transplant
This trial tests whether adding back certain immune cells after a stem cell transplant can help prevent serious viral or fungal infections. It's for children and young adults up to age 25 who need a transplant for a blood cancer or other curable disease.
Philadelphia, PennsylvaniaAges 1 month–25 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 - 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 - NCT07209462RecruitingPhase 2
MRM-3379 for Fragile X Syndrome in males
This trial tests an investigational medicine called MRM-3379 in males with Fragile X syndrome. It looks at how the medicine affects thinking and behavior over time.
Media, PennsylvaniaAges 13–45 - 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 - 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+ - 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.
Pittsburgh, PennsylvaniaAges 18 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 - NCT01852370Enrolling by invitationPhase 1/Phase 2
Lung and bone marrow transplant plan for immune system disorders
This early-phase study tests a step-by-step transplant approach: first replacing the lungs, then doing a bone marrow (stem cell) transplant to treat certain immune deficiency diseases. It may help people whose immune problem has led to severe, end-stage lung disease.
Pittsburgh, PennsylvaniaAges 5–45 - 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+ - 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 - NCT07576036Enrolling by invitation
Study of severe low sodium and brain safety during treatment
This is a retrospective study (meaning researchers look back at medical records) of patients who had very low sodium levels in their blood when admitted to the hospital. Researchers want to understand the link between how quickly doctors correct low sodium and a rare brain condition called osmotic demyelination syndrome.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06843811Enrolling by invitationPhase 2
Sirolimus for Leigh Syndrome
This trial tests if sirolimus can help people with Leigh syndrome. It aims to see if the drug can improve neurological symptoms and is looking for participants aged 6 months to 55 years.
Philadelphia, PennsylvaniaAges 6 months–55 years - NCT04880876Enrolling by invitationPhase 3
Oral IBS-D medicine trial for kids and teens
This trial tests whether an oral medicine (eluxadoline) can help children and teens with IBS-D, a condition that causes stomach pain and frequent loose stools. It’s a Phase 3 study, meaning it’s designed to confirm benefits and safety.
Pittsburgh, PennsylvaniaAges 6–17 - 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.
Allentown, PennsylvaniaAges 18 years+ - NCT06827262Recruiting
A procedure for vertebrogenic back pain relief
This trial tests a minimally invasive procedure that uses a special device to target and treat nerve-related back pain coming from the vertebrae (the bones in your spine). It may offer lasting relief without more major surgery.
Pittsburgh, PennsylvaniaAges 18 years+ - 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 - NCT06979531RecruitingPhase 2
A study of IMVT-1402 for Sjogren's disease with moderate to severe symptoms
This trial tests an experimental drug, IMVT-1402, in adults with primary Sjogren's disease who have moderate to severe symptoms. The goal is to see if it can reduce disease activity safely.
Philadelphia, PennsylvaniaAges 18–74 - 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.
Pittsburgh, PennsylvaniaAges 1 day–17 years
Down Syndrome trials by city in Pennsylvania
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting down syndrome studies listed above.
- Children's Hospital of Philadelphia · 12 studies
- University of Pittsburgh · 4 studies
- University of Pennsylvania · 3 studies
- Thomas Jefferson University Hospital · 2 studies
- UPMC Children's Hospital of Pittsburgh · 2 studies
- Allegheny General Hospital
- Geisinger Medical Center
- Geisinger Wyoming Valley/Henry 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.
Down Syndrome trials in other states
Common questions
- Are there down syndrome clinical trials in Pennsylvania?
- Yes. We're currently tracking 141 recruiting down syndrome studies with a site in Pennsylvania, 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 in Pennsylvania?
- 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 Pennsylvania to take part?
- Not necessarily. These studies have a site in Pennsylvania, 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 in Pennsylvania regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.