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 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
- 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 - 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+ - NCT05819697Recruiting
Helping burning mouth symptoms with improved memory recall
This trial looks at ways to reduce how much people with burning mouth syndrome struggle to recall information during the study. If you qualify, you may be asked to complete study tasks and share saliva to support the research.
Philadelphia, PennsylvaniaAges 18 years+ - 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 - 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 - 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 - NCT07220187RecruitingPhase 3
Adding pirtobrutinib to usual treatment for newly diagnosed Richter transformation
This study tests whether adding a targeted drug called pirtobrutinib to the usual chemotherapy for Richter transformation can improve outcomes. It is a phase 3 trial, meaning it compares the new combination to the standard approach, and may help patients live longer or delay progression.
Philadelphia, PennsylvaniaAges 18 years+ - NCT07278375Recruiting
Comparing treatments for babies with opioid withdrawal
This study looks at different medicine approaches to treat newborns with opioid withdrawal (NOWS). It compares how well babies do with standard care and may help find the best way to manage symptoms and support recovery.
Philadelphia, PennsylvaniaAges birth+ - 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 - 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 - 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 - 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+ - NCT06043440RecruitingPhase 2
Oxygen therapy for sleep apnea in Down syndrome
This study tests whether oxygen therapy can help children with Down syndrome who have sleep apnea. It compares oxygen treatment to no treatment to see if it improves breathing during sleep and thinking skills.
Philadelphia, PennsylvaniaAges 5–18 - NCT07523581RecruitingPhase 2
Study of an experimental drug for Alport syndrome
This study tests an experimental medicine for people with Alport syndrome caused by a specific type of gene change (nonsense mutation). The drug may help reduce protein in the urine and slow kidney decline. It is a blinded study, so you won't know if you get the real drug or a placebo.
Philadelphia, PennsylvaniaAges 12 years+ - 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 - 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 - 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+ - NCT07503444RecruitingPhase 3
Fenfluramine for Rett syndrome symptoms
This study tests whether fenfluramine, a medication already used for seizures, can help people with Rett syndrome. It aims to see if it improves daily life and reduces symptoms.
Philadelphia, PennsylvaniaAges 5–35 - 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 - 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+ - 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 - 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 - 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 - 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
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 · 11 studies
- University of Pennsylvania · 5 studies
- Thomas Jefferson University Hospital · 2 studies
- Children's Hospital of Philadelphia - Accepting Young Adult and Juvenile Patients
- Children's Regional Hospital, Cooper University Health Care
- ChristianaCare
- Ep0247 21003
- Massive Bio SYNERGY-AI site
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.