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
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 may be conducted in person if the participant is in clinic during that follow up period.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
20
Given Subcutaneously (under the skin).
University of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITINGNational Heart, Blood and Lung Institute-Cellular and Molecular Therapy Branch
Bethesda, Maryland, United States
RECRUITINGSt. Jude Children's Research Hospital
Memphis, Tennessee, United States
RECRUITINGTo assess the safety and tolerability of motixafortide in participants with sickle cell disease (SCD) as determined by the incidence of adverse events.
Time frame: 0 - 30 days
To determine the yield of CD34+ cells/kg after a single or daily dose of motixafortide in participants with SCD.
Time frame: 1 - 2 days
To determine the mobilization effects as determined by peripheral blood CD34+ counts/uL after a single or daily dose of motixafortide in participants with SCD.
Time frame: 1 - 2 days
To recommend a phase 2 dosing strategy of one or two doses motixafortide in participants with SCD based on the incidence of adverse events
Time frame: through study completion, an average of 1 year
To recommend a phase 2 dosing strategy for timing of apheresis based on peripheral blood CD34+ counts/uL after motixafortide administration in participants with SCD
Time frame: through study completion, an average of 1 year
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