Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide
St. Jude Children's Research Hospital
Summary
This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use. PRIMARY OBJECTIVE * To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs). SECONDARY OBJECTIVES * To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg). * To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts * To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects
Description
This study is divided into 2 parts. Participants will be assigned to a part based on when they enroll. Early in the study, participants will be assigned to part A. Later in the study, participants will be assigned to part B. In both parts, participants will receive the study drug, motixafortide, by an injection under the skin. About 8 hours after the injection, stem cells will be collected. Participants will follow-up within 7-10 days after the study drug and stem cell collection. Study staff will contact participants about 30 (±3) days after the last drug dose administration. The follow up ma…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Participants with severe sickle cell disease (SCD) who are ≥18 years of age and willing to donate autologous hematopoietic stem cells (HSCs) for advancing future gene therapy for SCD after collection of back-up product. Severe SCD, for the purpose of this study, will be defined as participants who are receiving chronic transfusion therapy due to SCD related complications or are eligible for or currently enrolled on an allogeneic transplant protocol. * Participant must have a documented diagnosis of SCD with documentation of SCD genotype by medical history * Participants…
Interventions
- DrugMotixafortide
Given Subcutaneously (under the skin).
Locations (2)
- University of Alabama at BirminghamBirmingham, Alabama
- St. Jude Children's Research HospitalMemphis, Tennessee