Children's Hospital of Philadelphia leads or participates in hundreds of clinical research studies and clinical trials. Many of the studies that are currently enrolling patients can be found in this database. Use this finder to learn more about the purpose of these studies and clinical trials, find out who can participate, and tell us you're interested in enrolling. You can also look for studies that offer remote participation from home!
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. The purpose of CureGN is to create a patient research network to learn about four major causes of glomerular disease : 1. Minimal Change Disease (MCD) 2. Focal Segmental Glomerulosclerosis (FSGS) 3. Membranous Nephropathy (MN) 4. IgA Nephropathy (IgAN)
The purpose of this research study is to 1) validate the use of surveys such as fatigue
scales, Mitochondrial Disease scales, and quality of life scales, 2) validate clinical
exams, such as muscle strength tests, and the 6 minute walk test, and 3) to define the
natural history of the disease in the Mitochondrial Disease patient population.
Our team is working on a study to better understand neurological problems, including seizures and developmental delays, that occur in children with hyperinsulinism, including HI/HA syndrome, and type 1 diabetes mellitus. The study involves a one day visit to The Children’s Hospital of Philadelphia and The University of Pennsylvania for a specialized magnetic resonance imaging scan (MRI) of the brain, an electroencephalogram (EEG), blood draw, and completion of questionnaires to evaluate development and behavior. The entire study visit is expected to take about five hours. If you are interested in learning more, please contact us at HIResearch@email.chop.edu.
This study involves taking a study drug called CUDC-907. CUDC-907 is an oral drug that blocks certain
proteins in tumor cells; these proteins may be important in the growth of some
cancers. The overall goals of this study are to evaluate the side effects and
benefits of CUDC-907 in patients with relapsed or treatment refractory solid
tumor, brain tumor, or lymphoma, and to determine the best dose of CUDC-907 for
children and adolescents. This study
will also look at the levels of CUDC-907 in the blood over time after a dose.
The purpose of this study is to create a biorepository, which is a collection of biological samples and the data associated with them, to support our ongoing and future studies on ultra-rare pediatric neurodegenerative disorders. These include disorders associated with mitochondrial and autophagic dysfunction such as TBCK, congenital disorders of autophagy, mitochondrial disorders, and cases of neurodegeneration without known genetic diagnoses. Ultra-rare disorders affect 1 in every 50,000 kids.
Using the biological samples we collect, we intend to derive cellular disease models to study these disorders at a molecular level and screen for therapeutic targets. We also intend to look for biomarkers that may inform outcome measures in future interventions and clinical trials. By looking at the cells in these biological samples, we hope to understand how they work, what is wrong with them, and how we could fix them. The overarching aim is to yield generalizable knowledge about these disorders and translate it into better diagnostic awareness and clinical care.
Are you interested in participating in a research study about bone health in children and adolescents at high risk of suffering a broken bone? Dr. Weber at CHOP is looking for people between 5-19 years old with a genetic or a long-term medical condition affecting bone strength to participate in this study. We are also recruiting children between 5-19 years old with no medical conditions as healthy participants in the study. Participation will require a single visit at The Children’s Hospital of Philadelphia, which will include bone scans, a muscle strength evaluation by a physical therapist, blood and urine tests, and questionnaires. Eligible participants who complete the study will be compensated for their time.
Researchers at CHOP are trying to improve outcomes in
the treatment of adolescents with anorexia nervosa. Eligible families will
receive Family Based Treatment for anorexia nervosa. Some adolescents with
anorexia nervosa will also receive Cognitive Remediation Therapy (a treatment
that increases flexible thinking). Both parents are required to participate and
any siblings living at home are asked to come to the first four treatment
sessions. Families will receive treatment for approximately 6 months and will
be asked to complete number of assessments during treatment and at two time
points after treatment ends.
The purpose of this research study is to look at whether or not
a sleep questionnaire can be used on infants 3 to 24 months of age, with or at
high-risk of having Cerebral Palsy. This questionnaire is currently being used
on older children, so we will be looking at a younger age group.
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. All participants enrolled in the study will receive the investigational medication.
We are currently enrolling individuals who meet the following criteria:*
Thalassemia and not receiving regular RBC transfusions (Cohort C)
There
are additional eligibility requirements, which the investigator will explain to
you.
Are you interested learning about opportunities to participate in research? Sign up to receive information about new research studies at CHOP. It’s a great way to support the work of the Research Institute.