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This study involves taking a study drug called SAR443579. The overall goal of this study is to find out if the study drug is safe and effective at treating adults and children with relapsed or refractory acute myeloid leukemia (R/R AML), high risk myelodysplastic syndrome (HR-MDS), or B-cell acute lymphoblastic leukemia (B-ALL). This is a first-in-human research study.
CHOP, in collaboration with Onyx Therapeutics, Inc., is conducting a study for children and young adults up to 21 years old, with relapsed or refractory acute lymphoblastic leukemia. This clinical trial is adding the study drug, Carfilzomib, to a standard relapse chemotherapy regimen. For more information about this study, including specific eligibility criteria, please visit clinicaltrials.gov and search for "NCT02303821". To hear more about this and other available trails or get any questions answered please contact our Cancer Intake Specialist by phone at 267-426-0762 or email Oncointake@email.chop.edu.
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. The study procedures involve both the caregiver and child filling out online questionnaires on two separate occasions. If you are interested in participating, you can sign up immediately by clicking the link below: https://is.gd/FoodAllergyAnxiety If you have any questions, please contact the research coordinator, Leah Wilkey, by phone at 215-590-6198 or email at wilkeyl@email.chop.edu.
In this gene therapy study, we will treat children with deafness caused by an abnormal gene called otoferlin (OTOF). We will try to transfer the normal human OTOF gene into cells of the inner ear, using a modified form of a non-disease-causing virus. This study will see if this gene therapy is safe and improves hearing. The study drug, and the device to deliver it to the ear, are investigational, meaning that they are not approved by the United States Food and Drug Administration (FDA). This study is the first time that this study drug, and the device to deliver it, will be used in humans.
The study drug is placed into the inner ear during a surgical procedure. Children are then monitored for about 2 years for any side effects, for any effects on the body, and for hearing improvement. Different doses will be tested for their safety and effectiveness. There are about 14 visits to CHOP, including one surgery visit including an overnight hospital stay. Participants undergo follow up audiology tests monthly for 3 months, then at 6 months, then at 1 and 2 years. This includes auditory brainstem response (ABR) testing, which may require sedation. There are also repeated research blood tests, urine samples, physical examinations, and interviews.
To be eligible, children must be age 2-17 and have profound deafness in both ears. Children are not eligible if they have already received cochlear implants in both ears. Once in this study, children should not undergo cochlear implantation in the treated ear, unless the treatment fails to improve hearing after a 6 month period.
The main risks of this study are from the study drug and the surgery to deliver it. These include injury to the inner ear from the drug or surgery, injury to the eardrum, risks of anesthesia for the surgery and sedation for the ABR tests, local site inflammation or infection, injury to the liver, and delay of cochlear implantation in the treated ear if the treatment does not improve hearing. Since the study drug has never been given to humans before, it is possible there are other risks we do not know about yet.
Participants might benefit if the study drug improves their hearing. However, it is possible they will not benefit directly from participating in this study.
This study enrolls patients that have been diagnosed with acute leukemia associated with a KMT2A (MLL) gene rearrangement (referred to as KMT2Arearranged, or KMT2A-R). The KMT2A-R leukemia has come back after treatment (relapsed) or is not responding to treatment (it is refractory). This study involves taking a study drug called SNDX-5613. The study is divided into two phases: one in which the study drug is given in combination with chemotherapy and a phase where the study drug is given alone. The purpose of the study is to test the safety of the study drug with chemotherapy, and how well the treatment regimen works for infants and young children with relapsed or refractory KMT2A-R leukemia.