Clinical trials · Pennsylvania
Pediatrics clinical trials in Pennsylvania
We’re tracking 135 recruiting pediatrics studies with a site in Pennsylvania — including Phase 1/Phase 2, Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT07506863RecruitingPhase 3
Mitapivat for children with transfusion-dependent thalassemia
This study tests a study pill called mitapivat for children aged 1 to 17 with transfusion-dependent thalassemia. The goal is to see if mitapivat can reduce the need for blood transfusions and improve blood counts.
Philadelphia, PennsylvaniaAges 1–17 - NCT07494045RecruitingPhase 2
Study of CTAP101 for kids with chronic kidney disease
This study tests a new extended-release capsule (CTAP101) for children aged 8 to under 18 with moderate to severe chronic kidney disease (CKD). The goal is to see how the drug works in the body, if it's safe, and if it can help control mineral and hormone levels that are often out of balance in CKD.
Philadelphia, PennsylvaniaAges 1 month–17 years - NCT07432880Recruiting
Study of children with severe MMA linked to the MMUT gene
This study is for children up to 16 years old with a severe form of methylmalonic acidemia (MMA) caused by a specific gene change (MMUT). It aims to better understand the disease and may lead to future treatments. Both children who have had a liver transplant and those who have not can join, as long as certain criteria are met.
Philadelphia, PennsylvaniaAges Up to 16 years - NCT04457154Recruiting
Registry for teens and young adults with OSA who can’t use CPAP
This study collects information in a registry to help doctors understand outcomes for young people with moderate-to-severe obstructive sleep apnea who cannot use CPAP and may use an implantable breathing stimulation device. You might qualify if you’ve tried and couldn’t tolerate CPAP and you’re able/willing to get and use the device and return for follow-ups and sleep tests.
Sewickley, PennsylvaniaAges 18–21 - NCT02249923Recruiting
Children with pulmonary hypertension registry in Spanish or English
This registry study collects information from children and families affected by pulmonary hypertension to better understand the condition. It may help researchers track outcomes over time and improve future care.
Philadelphia, PennsylvaniaAges 1 day–21 years - NCT07287982RecruitingPhase 2
ARGX-119 for children with SMA who can walk
This study tests a new medicine called ARGX-119 in children with SMA. It is given through an IV and aims to help kids who can already walk get stronger and stay healthy.
Philadelphia, PennsylvaniaAges 5–17 - NCT07279350Recruiting
Health advocate support for children after liver transplant
This study tests whether having a health advocate helps families manage care for their child after a liver transplant. The advocate will provide support and resources to reduce stress and improve the child's recovery.
Pittsburgh, PennsylvaniaAges birth–18 years - NCT07227857RecruitingPhase 1/Phase 2
Study of a new medicine for children with KCNT1-related epilepsy
This study tests a new medicine (S230815) given by lumbar puncture (spinal tap) to see if it helps reduce seizures in children with a specific genetic cause of epilepsy. It is for children aged 2 to 12 who have not responded well to other treatments.
Philadelphia, PennsylvaniaAges 2–12 - NCT04775485RecruitingPhase 2
Targeted medicine for children with certain brain or solid tumors
This Phase 2 study tests tovorafenib, a targeted cancer medicine, in children and young adults whose tumors have specific BRAF or RAF changes. It is for cancers that have come back or grown despite previous treatment, with measurable signs on scans.
Philadelphia, PennsylvaniaAges 6 months–25 years - NCT07211997Recruiting
FebriDx test for fever and cold symptoms in children
This study tests a finger-prick blood test (FebriDx) to see if it can quickly tell whether a child's fever and cold symptoms are caused by a bacterial or viral infection. If it works, it could help doctors decide whether antibiotics are needed.
Pittsburgh, PennsylvaniaAges 2–11 - NCT06929455Recruiting
Making children’s medicines taste less bitter
This study tests whether a special ingredient can block the bitter taste of children's medicines, making them easier to swallow. Healthy adults will taste different medicine ingredients to see if the blocker helps.
Philadelphia, PennsylvaniaAges 18–60 - NCT07124078RecruitingPhase 2
Study of axatilimab for young people with chronic graft-versus-host disease
This study tests a new drug called axatilimab for children and teens with chronic graft-versus-host disease (cGVHD) that hasn't improved with standard treatments. The goal is to see if axatilimab works better than the usual care options. Participants will be randomly assigned to receive either axatilimab or one of several standard treatments.
Pittsburgh, PennsylvaniaAges 2–17 - NCT07108374Enrolling by invitation
Improving blood transfusion practices in children's ICUs
This study tests whether a new set of guidelines can help doctors and nurses give red blood cell transfusions more consistently in pediatric intensive care. It aims to make transfusion decisions safer and more uniform for children in the ICU.
Philadelphia, PennsylvaniaAges Any age - NCT07095166Recruiting
Studying eating habits in healthy kids ages 6-9
This study looks at how and when children eat. It uses a special scan and asks parents about their child's mealtimes to learn about healthy eating patterns.
State College, PennsylvaniaAges 6–9 - NCT07080385RecruitingPhase 2/Phase 3
Study of encaleret in children with a rare calcium disorder
This trial tests a medicine called encaleret in children with a rare genetic condition that causes low calcium levels. The goal is to see if it safely helps control calcium and reduce symptoms.
Philadelphia, PennsylvaniaAges birth–17 years - NCT07071298Recruiting
AERA pediatrics registry study
This registry tracks children who have a procedure called endoscopic transforaminal balloon dilation (ETBD) using the AERA device. The goal is to learn more about how this treatment works for young patients.
Philadelphia, PennsylvaniaAges Up to 18 years - NCT07019922RecruitingPhase 3
A study of elsunersen for children with SCN2A-related epilepsy
This trial tests an experimental drug called elsunersen in children with a specific type of epilepsy caused by changes in the SCN2A gene. It aims to see if the drug can reduce seizures and be safe for children.
Philadelphia, PennsylvaniaAges 1 day–18 years - NCT06997588RecruitingPhase 2
Testing alpelisib for overgrowth syndrome in children and adults
This trial is testing a drug called alpelisib for people with PROS (a rare condition that causes parts of the body to grow too much). The drug targets a specific gene change (PIK3CA mutation) that is often the cause. If you have this condition and it's causing serious symptoms, this study might help shrink or slow the overgrowth, improving your quality of life.
Pittsburgh, PennsylvaniaAges 2–100 - NCT06994845RecruitingPhase 3
Testing iptacopan for children with IgA nephropathy
This study tests a new medicine called iptacopan to see if it helps children with IgA nephropathy (a kidney disease). Kids who join will receive the study treatment and be monitored for safety and effectiveness.
Philadelphia, PennsylvaniaAges 2–18 - NCT06994442RecruitingPhase 3
Best pain relief for children on a breathing tube
This study tests adding extra pain medicines (acetaminophen or an NSAID) to usual opioid pain treatment for children on a breathing machine. The goal is to find the safest and most effective way to manage pain.
Hershey, PennsylvaniaAges 2 months–17 years - NCT06934967RecruitingPhase 3
Iptacopan study for children with PNH
This trial tests an oral medication, iptacopan, for children with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. It aims to see how well the drug works in kids who have not tried anti-C5 therapy or who are switching from it.
Philadelphia, PennsylvaniaAges 2–18 - NCT06941272RecruitingPhase 1/Phase 2
Study of patritumab deruxtecan for children with hard-to-treat solid tumors
This trial tests a new drug, patritumab deruxtecan, in children and adolescents whose rhabdomyosarcoma or hepatoblastoma has returned or not responded to at least one prior treatment. The goal is to see if the drug can shrink tumors or stop them from growing.
Philadelphia, PennsylvaniaAges 1 month–17 years - NCT06941389Recruiting
Comparing stem cell transplant vs standard therapy for sickle cell disease
This study compares two treatment approaches for children with sickle cell anemia: a stem cell transplant from a matched family donor versus continuing regular disease-modifying medicines. It aims to see which treatment works better over time.
Philadelphia, PennsylvaniaAges 3–20 - NCT06910813RecruitingPhase 1/Phase 2
Gene therapy trial for children with cystinosis
This trial tests a new gene therapy called DFT383 for children aged 2 to 5 with nephropathic cystinosis. It aims to see if the treatment is safe and can help improve kidney function.
Philadelphia, PennsylvaniaAges 2–5
Pediatrics trials by city in Pennsylvania
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting pediatrics studies listed above.
- Children's Hospital of Philadelphia · 13 studies
- Children's Hospital of Pittsburgh · 2 studies
- Children's Hospital of Philadelphia (MRD-HCT)
- Children's Hospital of Philadelphia (Recruiting Cohort 0)
- CHOP (Children's hospital of Philadelphia)
- Monell Chemical Senses Center
- OPKO Site
- PAS Research
What taking part in a pediatrics 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.
Pediatrics trials in other states
Common questions
- Are there pediatrics clinical trials in Pennsylvania?
- Yes. We're currently tracking 135 recruiting pediatrics 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 pediatrics 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 pediatrics 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 pediatrics 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.