This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.
This Phase 2, multicenter, open-label study will be conducted in 2 parts (Parts A and B). Part A is intended to characterize the efficacy, safety, PK and PD of a single dose of MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD. Part B is designed to characterize the efficacy, safety, PK and PD of 2 consecutive days of dosing with MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
1
MGTA-145 will be administered as an IV infusion
240 µg/kg administered subcutaneously
National Institutes of Health
Bethesda, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Apheresis Collection Yield
Determination of the yield of CD34+ cells after either one or two consecutive days of MGTA-145 and plerixafor mobilization followed by apheresis.
Time frame: Up to 2 days
Assess Number of Participants With Treatment Emergent Adverse Events Leading to Study Drug Discontinuation Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Assess number of participants with treatment emergent adverse events leading to study drug discontinuation based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame: Up to 30 days
Assess the Number of Participants With Treatment Emergent >/= Grade 3 Clinical Laboratory Abnormalities Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Assess the number of participants with treatment emergent \>/= Grade 3 clinical laboratory abnormalities based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame: Up to 11 days
Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Vital Signs - Number of participants with clinically significant changes from baseline in vital signs
Time frame: Up to 11 days
Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters.
Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters.
Time frame: Up to 11 days
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Mobilization Effects of Single-day and Two-day Dosing With MGTA-145 and Plerixafor in Peripheral Blood in Patients With SCD
Determination of peak peripheral blood CD34+ counts single-day and two-day dosing with MGTA-145 and plerixafor in peripheral blood in patients with SCD
Time frame: Up to 2 days
Investigate Plasma Concentrations of MGTA-145 Per Timepoint of Collection (Pharmacokinetics)
Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics)
Time frame: Up to 2 days
Assess Presence of MGTA-145 Anti-Drug Antibodies (ADA) in Plasma Samples (Using Electrochemiluminescent Immunoassay [ECLIA])
Assess presence of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay \[ECLIA\])
Time frame: Up to 11 days
Assess Titers of MGTA-145 Anti-Drug Antibodies (ADA) in Plasma Samples (Using Electrochemiluminescent Immunoassay [ECLIA])
Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay \[ECLIA\])
Time frame: Up to 11 days