131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)
brief summary
The purpose of this study is to find the smallest amount of the 131 I-apamistamab needed for preparing patients with severe sickle cell disease (SCD) for a bone marrow transplant. This is the first time 131 I-apamistamab is being used for advanced Sickle Cell Disease (SCD) in the setting of allogeneic stem cell transplant. 131 I-apamistamab is an investigational product. This means that 131 I-apamistamab has not been approved by the Food and Drug Administration (FDA) for medical use in patients. The study treatment that is given before the transplant is called the conditioning regimen. In this study, the investigators are adding a drug called 131 I-apamistamab instead of the conditioning regimen typically given before a stem cell transplant.
detailed description
The purpose of this study is to research the minimum effective dose (MED) of 131 I-apamistamab conditioning for hematopoietic stem cell transplantation for patients with advanced SCD. 131 Iapamistamab is an investigational product. This means that 131 I-apamistamab has not been approved by the Food and Drug Administration (FDA) for medical use in patients.
The study treatment that is given before the transplant is called the conditioning regimen. In this study, investigators are adding 131 I-apamistamab instead of the conditioning regimen typically given before a stem cell transplant.
The current standard of care conditioning for allogeneic stem cell transplant in SCD is a combination of chemotherapy, total body irradiation and an antibody called Campath. This study is being done to see if the stem cell transplant for SCD can still be effective by eliminating total body irradiation from the conditioning as it has potential long term side effects such as secondary cancers, infertility, early cataracts and lung toxicity.
This is a single center, Phase I, dose finding study to estimate the MED of hematopoietic stem cell transplantation for patients with advanced sickle cell disease using 131I-apamistmab-based nonmyeloablative conditioning. The study will enroll 24 patients who are 12-50 years of age with sickle cell anemia (Hb SS, SC, or Sβ0 thalassemia) and have an available HLA-matched sibling donor.
official title
Open-label, Single-Center, Phase 1 Study to Estimate the Minimum Effective Dose (MED) of 131I-apamistamab for Non-myeloablative Conditioning in Patients With Severe Sickle Cell Disease