Sickle Cell DiseasePhase 2NCT05445128

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

A Phase 2 study testing MGTA-145 and Plerixafor in people with Sickle Cell Disease, run by Ensoma at 3 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 to 35 years
Conditions the study is for: Sickle Cell Disease
What is being tested?: MGTA-145 and Plerixafor
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 1 participants
Where: 3 sitesUnited States
Study dates: 24 Jun 2022to 2 Feb 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 35 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Subject must be ≥18 to ≤35 years of age.
Subject must weigh ≥30 kg.
Subject must have a diagnosis of Sickle Cell Disease.

Who cannot join

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.

Trial locations and contacts

3 sites in 1 country.

United States3 sites

National Institutes of Health
Bethesda, Maryland, 20892
Boston Children's Hospital
Boston, Massachusetts, 02115
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105

Other trials in United States

About this study

Official title: 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

Read the 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.

Conditions: Sickle Cell Disease
Being tested: MGTA-145, Plerixafor
Keywords: SCD, MGTA-145, Plerixafor, Mobilization, Apheresis
Medical subject headings: Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MGTA-145Biological
MGTA-145 will be administered as an IV infusion
PlerixaforDrug
240 µg/kg administered subcutaneously

Study groups in NCT05445128
Study group	Role	What they receive
Part A: Single Day Dosing/Apheresis	Experimental	Single dose of MGTA-145 in combination with plerixafor followed by apheresis Receives: Biological: MGTA-145; Drug: Plerixafor
Part B: 2-Day Dosing/Apheresis	Experimental	MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days Receives: Biological: MGTA-145; Drug: Plerixafor

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: Up to 11 days

Secondary outcomes (4)

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
Measured: 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)
Measured: 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])
Measured: 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])
Measured: Up to 11 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Jun 20222 Feb 2023

First posted6 Jul 2022
Study start24 Jun 2022
Primary completion8 Dec 2022
Study completion2 Feb 2023

Why it stopped: This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety issue.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Enrolment: 1 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05445128
Sponsor's protocol ID: 145-SCD-204
Status last verified: Jan 2024
First submitted: 26 May 2022
Last updated: 25 Jul 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

All Sickle Cell Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 25 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT05445128
Acronym / sponsor ID: 145-SCD-204
Conditions: Sickle Cell Disease
Keywords: SCD, MGTA-145, Plerixafor, Mobilization, Apheresis
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety issue.
Enrolment: 1 participants (actual)
Lead sponsor: EnsomaIndustry (pharma, biotech, devices)
Collaborators: Genetix Biotherapeutics Inc.
Study start: 24 Jun 2022 (actual)
Primary completion: 8 Dec 2022 (actual)
Study completion: 2 Feb 2023 (actual)
First posted: 6 Jul 2022 (actual)
Results: Posted 25 Jul 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Part A: Single Day Dosing/Apheresis	Experimental	Biological: MGTA-145, Drug: PlerixaforSingle dose of MGTA-145 in combination with plerixafor followed by apheresis
Part B: 2-Day Dosing/Apheresis	Experimental	Biological: MGTA-145, Drug: PlerixaforMGTA-145 in combination with plerixafor followed by apheresis on two consecutive days

Who may qualify

Ages: 18 to 35 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (3 listed)

Subject must be ≥18 to ≤35 years of age.
Subject must weigh ≥30 kg.
Subject must have a diagnosis of Sickle Cell Disease.

Key exclusion criteria (5 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
MGTA-145MGTA-145 will be administered as an IV infusion	Biological	Part A: Single Day Dosing/Apheresis, Part B: 2-Day Dosing/Apheresis
Plerixafor240 µg/kg administered subcutaneously	Drug	Part A: Single Day Dosing/Apheresis, Part B: 2-Day Dosing/Apheresis

Primary outcome

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.

4 secondary outcomes

Mobilization Effects of 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)
Time frame: Up to 2 days
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])
Time frame: Up to 11 days

Results and safety

Results posted to the registry on 25 Jul 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Part A: Single Day Dosing/Apheresis	1	1
Part B: 2-Day Dosing/Apheresis	0	0

Primary outcome: Apheresis Collection Yield

Time frame: Up to 2 days

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

Primary outcome: 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. Number. Unit: participants

Measure	Part A: Single Day Dosing/Apheresis n = 1
Value	0

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

Primary outcome: 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. Number. Unit: participants

Measure	Part A: Single Day Dosing/Apheresis n = 1
Value	0

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

Primary outcome: Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs

Time frame: Up to 11 days. Count of participants. Unit: Participants

Measure	Part A: Single Day Dosing/Apheresis n = 1
Value	0

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

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: 30 days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part A: Single Day Dosing/Apheresis	0 of 1 (0%)	1 of 1 (100%)	0 of 1

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Sickle Cell Disease

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
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RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
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RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
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Not yet recruitingPhase 1
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Ji Hyun Lee, MD, MPH study director

Magenta Therapeutics

3 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing MGTA-145 and Plerixafor in people with Sickle Cell Disease, run by Ensoma at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 25 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 25 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Apheresis Collection Yield (time frame: Up to 2 days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05445128.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jul 2025.