Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Sickle Cell Disease
- Type
- Interventional
- Phase
- Phase 1
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentIntervention Model Description: Each SCD research participant will receive up to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose)Masking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 40 years
- Gender
- Both males and females
Description
Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for sickle cell disease (SCD) patients but its use is significantly limited by the lack of compatible donors. Gene therapy using autologous hematopoietic stem cell (HSC) transplantation represents an alternative approach...
Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for sickle cell disease (SCD) patients but its use is significantly limited by the lack of compatible donors. Gene therapy using autologous hematopoietic stem cell (HSC) transplantation represents an alternative approach but requires the collection of significant numbers of cells. Severe adverse events have been observed in SCD patients after mobilization using the standard agent granulocyte colony-stimulating factor (G-CSF), and bone marrow harvesting is also associated with side effects. The use of a single administration of plerixafor has been suggested as an alternative mobilization strategy in SCD but may not mobilize sufficient number of HSC. In this study, our primary objective is to assess if up to two injections of plerixafor (starting dose level: 240 µg/kg/dose) are safe in SCD patients and can recruit enough blood stem cells needed for future gene therapy.
Tracking Information
- NCT #
- NCT03664830
- Collaborators
- Not Provided
- Investigators
- Principal Investigator: Joseph Rosenthal, MD City of Hope Medical Center