Department of Pediatrics Research Studies

The purpose of this research study is to test a new method of cell processing for stem cell transplants with an unrelated donor or partially matched related donor for patients with BMF who do not have a well matched related donor.

The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 12 to 35 years who have been diagnosed with severe sickle cell disease.

The purpose of this study is to compare the genes of African American children and adults who have inflammatory bowel disease (IBD) with the genes of those who do not have IBD.

The study compares 2 different experimental chemotherapy combinations with the usual chemotherapy combination given during post-Induction therapy.

The purpose of this registry is to study the long-term effects and safety of Abatacept(Orencia) as used for the treatment of Juvenile Idiopathic Arthritis(JIA). JIA patients that are currently taking Abatacept may be eligible to participate in this study.

The purpose of this study is to determine the highest safe dose of abemaciclib in patients with relapsed or treatment refractory solid tumors given in combination with the chemotherapy drugs, irinotecan and temozolomide (Part A) or in combination with just temozolomide (Part B).

The Alterra study is for patients in need of pulmonary valve replacement.

A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease.

The goal of the study extension is to see whether the incretin hormone GIP increases insulin secretion in individuals with CF and normal glucose tolerance compared to individuals without CF and normal glucose tolerance.

The purpose of this research study is to collect different types of samples (such as stool, swabs, urine) to form a biorepository.

The purpose of this research study is to compare transplant (HCT/BMT) to standard of care treatment in young adults with SCD who have experienced complications.

This study will test whether supplemental oxygen therapy, a common post-discharge treatment for BPD, improves outcomes.

The purpose of this research study is to examine and understand the various mechanisms that contribute to CFRD and gain a better understanding of potential means to treat CFRD.

The purpose of this research study is to examine and understand the various mechanisms that contribute to Cystic Fibrosis Related Diabetes and gain a better understanding of potential means to treat Cystic Fibrosis Related Diabetes.

Dr. William Gaetz. has partnered with pediatric neurologist, Dr. Eric Marsh and neuropsychologist, Dr. Lisa Blaskey to better understand Epilepsy in children who have an Autism Spectrum Disorder (ASD).

The purpose of this study is to assess deficits in movement and coordination in children with cerebral palsy (CP) and without.

Cure Glomerulonephropathy Network (CureGN) is a multi-year study of 2,400 children and adults with four rare glomerular (kidney) diseases at over 50 centers in the US, Canada, and Italy.

This study is a 2 period, open label study that will evaluate the safety and efficacy of dasiglucagon in children between the ages of 3 months and 12 years.

The purpose of this study is to find out how well surveys can measure disease activity in kids with lower back arthritis. The study will last for either 3 or 6 months and take 2 or 3 visits.

This research is studying a vaccine against Mpox (formerly known as monkeypox) that is FDA approved for adults but not yet approved for children. 
 

The Dova study is for children below 18 years with primary Immune Thrombocytopenia (ITP) who have had an insufficient response to previous treatment.

The purpose of this research study is to determine if certain markers in your blood are increased over the duration of your chemotherapy, especially as you get sicker.

This study is interested in identifying how the brain reacts to pain both before and after treatment in the intensive RND program.

The primary objective is to confirm the association of the Filament-Aggregating Protein loss-of-function mutations with respect to the severity of disease and its association with components of the atopic triad within our Atopic Dermatitis cohort.

The goal of this study is to identify and measure anxiety symptoms in children and adolescents with food allergy. We are looking for families of children with IgE-mediated food allergies between 8 and 18 years of age to participate.

This is a multicenter natural history and clinical measure study.

This Phase 2 clinical research study is evaluating the safety and effectiveness of a once-daily oral investigational medicine that may improve anemia and reduce the need for red blood cell (RBC) transfusions among some patients who are chronically transfused.

The Harmony Valve study is for patients in need of pulmonary valve replacement.

Children (less than 10 years of age) are eligible if they have been diagnosed with either a medulloblastoma tumor or CNS embryonal tumors of the brain or spinal cord.

The purpose of this research study is to learn more about the normal function of blood vessels in healthy children and adolescents.

This purpose of this study is to determine the efficacy and safety of tisagenlecleucel (also referred to as CTL019, or Kymriah™) in children, adolescents and young adults with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL).

A new, safe, state-of-the-art technique called HR-pQCT (high resolution peripheral quantitative computed tomography) can be used to measure bone health in children and adults.

In this study, we are developing new magnetic resonance imaging (MRI) and ultrasound methods to determine if it is effective in diagnosing complication in kidney transplant patients.

The purpose of this research study is to collect information over time in juvenile SpA and back pain patients.

This study will take the patients white blood cells - T cells - and change them to turn against the cancer.

The Children's Hospital of Philadelphia is conducting a Natural History Study in Leigh Syndrome.

The LUNA 3 study is for teenagers and adults (ages 12 and up) with primary immune thrombocytopenia (ITP) for which standard medications are no longer working.

Collect data and blood samples to make observations and determinations about rate and severity of graft-versus-host disease in HCT patients.

The purpose of this research study is to test and adapt new techniques for performing magnetic resonance imaging (MRI). MRI provides pictures of the inside of the body and information about chemicals the body makes.

The purpose of this research study is to validate the use of surveys such as fatigue scales, Mitochondrial Disease scales, and quality of life scales.

The purpose of this study is to find out if myofascial release therapy (MFR), (a massage method that will be used in the belly area), will improve the symptoms in CHOP patients with Irritable Bowel Syndrome (IBS).

The purpose of this research study is to learn more about Graves' disease and PTC/Hypothyroidism, how it effects brain function and brain function changes after starting medical treatment.

Vancomycin is a commonly administered antibiotic in critically ill children, but it can have unwanted side effects on the kidney (acute kidney injury).

The purpose of this study is to characterize the microbiome in children with Crohn's disease who have perianal fistula and to compare it to other children with Crohn's disease who do not have perianal disease.

The purpose of this research study is to evaluate the side effects of using proton radiation therapy for kidney tumors.

This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19.

The purpose of this research study is to find out if stem cells from umbilical cord blood can strengthen the muscle of the right side of the heart for patients diagnosed with Hypolplastic Left Heart Syndrome who are scheduled to undergo the Stage II Glenn surgical procedure.

The purpose of this study is to determine how what you eat and drink (your diet), antibiotics you take, and the bacteria that live in your gut (microbiome) contribute to developing kidney stones.

This is a randomized controlled trial that tests whether a program designed to maintain a high fluid intake decreases kidney stone recurrence.

The purpose of this study is to collect data from routine standard of care visits of SMA patients at the Children's Hospital of Philadelphia Neuromuscular Clinic.

PRONTO-PEDS will test if a ultra-fast insulin product given before or immediately after meals improves glycemic control in children and teens with type 1 diabetes.

The purpose of this study is to identify and engage young black or hispanic/latino men in Philadelphia, PA, Baltimore, MD and Washington, DC in HIV and substance use prevention using a new Mobile-enhanced Engagement intervention.

Individuals with beta thalassemia that is treated with regular transfusions, and who are between the ages of 18 and 40 years old, may be able to participate.

This study is aiming to compare at home sleep studies and in lab sleep studies.

CHOP and the Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) consortium is conducting a study for children and young adults 21 years old or younger who have relapsed or refractory acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma.

This is an observational registry of children and young adults with pediatric-onset rheumatic conditions such as juvenile idiopathic arthritis (JIA) and Systemic Lupus Erythematosus (SLE) and SLE-like conditions.

The SLEEP study will test different sleep promoting strategies for children.

This is a clinical trial to study the use of Tadekinig alfa or placebo (medication) in the treatment of NLRC4 MAS mutation or XIAP deficiency.

The purpose of this study is to examine CHOP's experience with patients ages 0 to 19 years old who have already undergone or will be undergoing total thyroidectomy (TTx) and/or completion thyroidectomy performed at the Thyroid Center at CHOP.

The purpose of this research study is to help us understand how well tofacitinib works as a treatment for ulcerative colitis or indeterminate colitis in pediatric patients.

The purpose of this research study is to identify genes that are involved in the development of Inflammatory Bowel Disease (IBD) at a young age (less than 8 years of age).

The goal of this study is to identify unique characteristics in the way kids and teens with Autism Spectrum Disorder (ASD) speak in comparison to typically developing peers.