A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of MGTA-145 in Combination With Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients With Sickle Cell Disease
Overview
- Phase
- Phase 2
- Intervention
- MGTA-145
- Conditions
- Sickle Cell Disease
- Sponsor
- Ensoma
- Enrollment
- 1
- Locations
- 3
- Primary Endpoint
- Apheresis Collection Yield
- Status
- Terminated
- Last Updated
- 9 months ago
Overview
Brief Summary
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.
Detailed Description
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subject must be ≥18 to ≤35 years of age.
- •Subject must weigh ≥30 kg.
- •Subject must have a diagnosis of Sickle Cell Disease.
Exclusion Criteria
- •Subject must not have had a vaso-occlusive event (VOE) requiring a visit to a healthcare facility within 30 days of screening.
- •Subject must not have undergone or attempted and failed previous hematopoietic stem cell (HSC) collection.
- •Subject must not have had a prior autologous or allogeneic transplantation, inclusive of gene therapy.
- •Male subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.
- •Female subject must not be pregnant or breastfeeding. If sexually active, female subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.
Arms & Interventions
Part A: Single Day Dosing/Apheresis
Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Intervention: MGTA-145
Part A: Single Day Dosing/Apheresis
Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Intervention: Plerixafor
Part B: 2-Day Dosing/Apheresis
MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
Intervention: MGTA-145
Part B: 2-Day Dosing/Apheresis
MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
Intervention: Plerixafor
Outcomes
Primary Outcomes
Apheresis Collection Yield
Time Frame: Up to 2 days
Determination of the yield of CD34+ cells after either one or two consecutive days of MGTA-145 and plerixafor mobilization followed by apheresis.
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 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 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
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.
Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Time Frame: Up to 11 days
Vital Signs - Number of participants with clinically significant changes from baseline in vital signs
Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters.
Time Frame: Up to 11 days
Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters.
Secondary Outcomes
- Mobilization Effects of Single-day and Two-day Dosing With MGTA-145 and Plerixafor in Peripheral Blood in Patients With SCD(Up to 2 days)
- Investigate Plasma Concentrations of MGTA-145 Per Timepoint of Collection (Pharmacokinetics)(Up to 2 days)
- Assess Presence of MGTA-145 Anti-Drug Antibodies (ADA) in Plasma Samples (Using Electrochemiluminescent Immunoassay [ECLIA])(Up to 11 days)
- Assess Titers of MGTA-145 Anti-Drug Antibodies (ADA) in Plasma Samples (Using Electrochemiluminescent Immunoassay [ECLIA])(Up to 11 days)