Children at a higher risk for Cerebral Palsy or children diagnosed with Cerebral Palsy within the age range of 4-13 months (corrected age) are being recruited for an Early Detection study.

For 3-months, two programs will be offered that may help babies with cerebral palsy move and use their arms more like other children at an early age. If you decide to participate, your participation may mean:

• 6-week telehealth therapy program (with one in-person visit) with our occupational/physical therapist to learn simple exercises to help your baby use their weaker arm. Your baby would also wear a soft mitt on their stronger arm to help them use their weaker arm.

• 6-week telehealth parent support program, (with one in-person visit) with our counselor to learn more about movement, how babies learn, and parenting tips specifically designed for your family.

• Two to three total in-person visits to our lab space to complete assessments and answer questions about how your baby is growing

  
  

The study will enroll male and female patients between 2 years to 25 years of age with sickle cell disease or beta thalassemia major, who do not have a matched related donor, and their doctor feels their condition should be treated with hematopoietic stem cell transplantation (HSCT).        

·       -For Sickle Cell Disease, individuals must have significant disease. We would consider you for transplant if you have had severe neurologic symptoms including stroke, frequent vaso-occlusive events (examples include pain episodes requiring pain management or acute chest syndrome), or administration of regular and frequent red blood cell transfusions to prevent disease complications. 

·       -For Beta-Thalassemia Major, you would be considered a candidate for transplant if you have genetic test confirming Beta Thalassemia and have required 8 or more transfusions per year in the past two years.

The purpose of this research study is to test a new method of cell processing for stem cell transplants with a closely matched unrelated donor. The new method is called alpha/beta T cell and B cell depletion using a device called CliniMACs. We want to see if this method can reduce some of the complications of the transplant and decrease the time it takes for the new stem cells to establish a new immune system. The use of the CliniMACs device for alpha/beta T and B cell depletion is experimental and is considered research.

The goal of this study is to evaluate the safety and efficacy of an investigational drug called "Elamipretide" in adult patients with mitochondrial disease. "Investigational" means the study drug has not been approved by the U.S. Food and Drug Administration (FDA) and its use in this study is experimental.

The study will last for approximately 13 months and will include at least 7 outpatient visits at CHOP, where participants will be provided the study drug (randomized to active drug or placebo) and monitored for safety.  The clinical trial also involves blood and urine tests, cardiac tests, physical exercise tests, and other study procedures.  For more information: https://clinicaltrials.gov/ct2/show/NCT05162768

You will also receive travel support and reimbursement for related expenses.

The purpose of the study is to evaluate blood sugar levels and beta-cell function in adolescents and adult with K_ATP hyperinsulinism whom did not undergo pancreatectomy. We will perform oral glucose tolerance tests, glucose potentiated arginine tests, and evaluate blood sugars in home environment by using continuous glucose monitoring for 10 days.

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 goal of this study is to develop ultrasound techniques that assess blood supply to the intestines using an investigational contrast agent. Our hope is to contribute to early diagnosis and improved outcomes.

The goal of this HIE (Hypoxic Ischemic Encephalopathy) Study is to develop ultrasound techniques for detecting impaired blood supply to the brain using an investigational contrast agent.  Our hope is to contribute to early diagnosis and improved outcomes. 

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.

The Child and Adolescent Thyroid Consortium (CATC) Biorepository is a collaborative research program dedicated to studying thyroid cancers and other thyroid-related conditions. The goal of the CATC Biorepository is to address the unmet need of creating a well-annotated registry and bio specimen repository for patients with varying thyroid-related diagnoses. Collaborating clinicians and scientists are encouraged to use the specimens and infrastructure of the CATC Biorepository to make meaningful discoveries to help patients most at-risk.

The ENERGY study is for adults (ages 18 and up) with warm Autoimmune Hemolytic Anemia (wAIHA) who are currently receiving treatment or have previously received treatment. The goal of the study is to see if the study drug, Nipocalimab is safe and effective in the treatment of wAIHA. Nipocalimab is an investigational medication designed to stop your immune system from destroying your red blood cells.

You may be able to join the study if you are:

•          18 years of age or older.
•          Diagnosed with wAIHA for at least 3 months.
•          Currently receiving treatment or have previously received treatment for wAIHA
•          Have platelet counts of more than 30,000/ L and Hemoglobin less than 10 g/dL

 Other study requirements will apply.