Trial Outcomes & Findings for Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease (NCT NCT05445128)

NCT ID: NCT05445128

Last Updated: 2025-07-25

Results Overview

Determination of the yield of CD34+ cells after either one or two consecutive days of MGTA-145 and plerixafor mobilization followed by apheresis.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

Up to 2 days

Results posted on

2025-07-25

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Part A: Single Day Dosing/Apheresis Single dose of MGTA-145 in combination with plerixafor followed by apheresis	Part B: 2-Day Dosing/Apheresis MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
Overall Study STARTED	1	0
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part A: Single Day Dosing/Apheresis n=1 Participants Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 2 days

Population: Study terminated early by original sponsor after first patient dosed

Determination of the yield of CD34+ cells after either one or two consecutive days of MGTA-145 and plerixafor mobilization followed by apheresis.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Up to 30 days

Population: Study terminated early by original sponsor after first patient dosed

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.

Outcome measures

Outcome measures
Measure	Part A: Single Day Dosing/Apheresis n=1 Participants Single dose of MGTA-145 in combination with plerixafor 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.	0 participants

PRIMARY outcome

Timeframe: Up to 11 days

Population: Study terminated early by original sponsor after first patient dosed

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.

Outcome measures

Outcome measures
Measure	Part A: Single Day Dosing/Apheresis n=1 Participants Single dose of MGTA-145 in combination with plerixafor followed by 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 outcome

Timeframe: Up to 11 days

Population: Study terminated early by original sponsor after first patient dosed

Vital Signs - Number of participants with clinically significant changes from baseline in vital signs

Outcome measures

Outcome measures
Measure	Part A: Single Day Dosing/Apheresis n=1 Participants Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs	0 Participants

PRIMARY outcome

Timeframe: Up to 11 days

Population: Study terminated early by original sponsor after first patient dosed

Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters.

Outcome measures

Outcome measures
Measure	Part A: Single Day Dosing/Apheresis n=1 Participants Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters.	1 Participants

SECONDARY outcome

Timeframe: Up to 2 days

Population: Study terminated early by original sponsor after first patient dosed

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 days

Population: Study terminated early by original sponsor after first patient dosed

Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 11 days

Population: Study terminated early by original sponsor after first patient dosed

Assess presence of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay \[ECLIA\])

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 11 days

Population: Study terminated early by original sponsor after first patient dosed

Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay \[ECLIA\])

Outcome measures

Outcome data not reported

Adverse Events

Part A: Single Day Dosing/Apheresis

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Part A: Single Day Dosing/Apheresis n=1 participants at risk Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Musculoskeletal and connective tissue disorders Back pain	100.0% 1/1 • 30 days Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Nervous system disorders Headache	100.0% 1/1 • 30 days Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

Additional Information

Ensoma head of regulatory or head of clinical research & development

Ensoma

Phone: 6127191200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place