Sickle Cell Disease
Conditions
Keywords
SCD, MGTA-145, Plerixafor, Mobilization, Apheresis
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.
Interventions
BIOLOGICALMGTA-145
MGTA-145 will be administered as an IV infusion
DRUGPlerixafor
240 µg/kg administered subcutaneously
Sponsors
Genetix Biotherapeutics Inc.
Ensoma
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 35 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters. | Up to 11 days | Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters. |
| Apheresis Collection Yield | 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. | 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. | 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 | Up to 11 days | Vital Signs - Number of participants with clinically significant changes from baseline in vital signs |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Investigate Plasma Concentrations of MGTA-145 Per Timepoint of Collection (Pharmacokinetics) | Up to 2 days | Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics) |
| Assess Presence of MGTA-145 Anti-Drug Antibodies (ADA) in Plasma Samples (Using Electrochemiluminescent Immunoassay [ECLIA]) | Up to 11 days | Assess presence 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 | Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay \[ECLIA\]) |
| 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 | 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 |
Countries
United States
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Part A: Single Day Dosing/Apheresis Single dose of MGTA-145 in combination with plerixafor followed by apheresis | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Part A: Single Day Dosing/Apheresis |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 1 |
| other Total, other adverse events | 1 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
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.
Time frame: Up to 2 days
Population: Study terminated early by original sponsor after first patient dosed
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.
Time frame: Up to 30 days
Population: Study terminated early by original sponsor after first patient dosed
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Part A: Single Day Dosing/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. | 0 participants |
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.
Time frame: Up to 11 days
Population: Study terminated early by original sponsor after first patient dosed
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Part A: Single Day Dosing/Apheresis | 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. | 0 participants |
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.
Time frame: Up to 11 days
Population: Study terminated early by original sponsor after first patient dosed
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part A: Single Day Dosing/Apheresis | Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters. | 1 Participants |
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
Time frame: Up to 11 days
Population: Study terminated early by original sponsor after first patient dosed
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part A: Single Day Dosing/Apheresis | Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs | 0 Participants |
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 \[ECLIA\])
Time frame: Up to 11 days
Population: Study terminated early by original sponsor after first patient dosed
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 \[ECLIA\])
Time frame: Up to 11 days
Population: Study terminated early by original sponsor after first patient dosed
Secondary
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
Population: Study terminated early by original sponsor after first patient dosed
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
Time frame: Up to 2 days
Population: Study terminated early by original sponsor after first patient dosed