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This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety issue.
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| Name | Class |
|---|---|
| Genetix Biotherapeutics Inc. | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: Single Day Dosing/Apheresis | Experimental | Single dose of MGTA-145 in combination with plerixafor followed by apheresis |
|
| Part B: 2-Day Dosing/Apheresis | Experimental | MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MGTA-145 | Biological | MGTA-145 will be administered as an IV infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | 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 | 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. | Up to 11 days |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Up to 2 days |
| Investigate Plasma Concentrations of MGTA-145 Per Timepoint of Collection (Pharmacokinetics) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ji Hyun Lee, MD, MPH | Magenta Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Institutes of Health | Bethesda | Maryland | 20892 | United States | ||
| Boston Children's Hospital |
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Study terminated early by original sponsor after first patient dosed. Only 1 participant was enrolled in Part A and no participants were enrolled in Part B.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part A: Single Day Dosing/Apheresis | Single dose of MGTA-145 in combination with plerixafor followed by apheresis |
| FG001 | Part B: 2-Day Dosing/Apheresis | MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Study terminated early by original sponsor after first patient dosed
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| ID | Title | Description |
|---|---|---|
| BG000 | Part A: Single Day Dosing/Apheresis | Single dose of MGTA-145 in combination with plerixafor followed by apheresis |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Study terminated early by original sponsor after first patient dosed | Posted | Up to 2 days |
|
|
30 days
Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: Single Day Dosing/Apheresis | Single dose of MGTA-145 in combination with plerixafor followed by apheresis |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
Study terminated early by original sponsor after first patient dosed. Only 1 participant was enrolled in Part A and no participants were enrolled in Part B.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ensoma head of regulatory or head of clinical research & development | Ensoma | 6127191200 | ddietz@ensoma.com |
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 5, 2021 | Jun 19, 2025 | Prot_000.pdf |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C088327 | plerixafor |
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| Plerixafor | Drug | 240 µg/kg administered subcutaneously |
|
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]) | 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]) | Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA]) | Up to 11 days |
| Boston |
| Massachusetts |
| 02115 |
| United States |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | United States |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Primary | 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. | Study terminated early by original sponsor after first patient dosed | Posted | Number | participants | Up to 30 days |
|
|
|
| Primary | 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. | Study terminated early by original sponsor after first patient dosed | Posted | Number | participants | Up to 11 days |
|
|
|
| Primary | 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 | Study terminated early by original sponsor after first patient dosed | Posted | Count of Participants | Participants | Up to 11 days |
|
|
|
| Primary | 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. | Study terminated early by original sponsor after first patient dosed | Posted | Count of Participants | Participants | Up to 11 days |
|
|
|
| Secondary | 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 | Study terminated early by original sponsor after first patient dosed | Posted | Up to 2 days |
|
|
| Secondary | Investigate Plasma Concentrations of MGTA-145 Per Timepoint of Collection (Pharmacokinetics) | Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics) | Study terminated early by original sponsor after first patient dosed | Posted | Up to 2 days |
|
|
| Secondary | 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]) | Study terminated early by original sponsor after first patient dosed | Posted | Up to 11 days |
|
|
| Secondary | 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]) | Study terminated early by original sponsor after first patient dosed | Posted | Up to 11 days |
|
|
| 0 |
| 1 |
| 0 |
| 1 |
| 1 |
| 1 |
| Headache | Nervous system disorders | Systematic Assessment |
|
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |