Motixafortide for Sickle Cell Disease
Trial Summary
Will I have to stop taking my current medications?
The trial requires participants to stop taking hydroxyurea for 30 days before the first dose of the study drug. For other medications, the protocol does not specify, so it's best to discuss with the trial team.
Is the drug Motixafortide a promising treatment for Sickle Cell Disease?
What is the purpose of this trial?
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
Research Team
Akshay Sharma, MBBS, MSc
Principal Investigator
St. Jude Children's Research Hospital
Eligibility Criteria
This trial is for individuals with sickle cell disease (SCD) or sickle cell anemia. It's designed to test if a drug called motixafortide can safely increase the number of stem cells in their blood, potentially helping future transplant treatments.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part A
Participants receive a single dose of motixafortide followed by stem cell collection 8 hours later
Treatment Part B
Participants receive two daily doses of motixafortide with stem cell collection after each dose
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extended Follow-up
Study staff will contact participants about 30 days after the last drug dose administration
Treatment Details
Interventions
- Motixafortide
Motixafortide is already approved in United States for the following indications:
- Multiple myeloma
Find a Clinic Near You
Who Is Running the Clinical Trial?
St. Jude Children's Research Hospital
Lead Sponsor
BioLineRx, Ltd.
Industry Sponsor