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

Primary Purpose

Sickle Cell Disease

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
MGTA-145
Plerixafor
Sponsored by
Magenta Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring SCD, MGTA-145, Plerixafor, Mobilization, Apheresis

Eligibility Criteria

18 Years - 35 Years (Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • National Institutes of Health
  • Boston Children's Hospital
  • St. Jude Children's Research Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Part A: Single Day Dosing/Apheresis

Part B: 2-Day Dosing/Apheresis

Arm Description

Single dose of MGTA-145 in combination with plerixafor followed by apheresis

MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
May 26, 2022
Last Updated
February 7, 2023
Sponsor
Magenta Therapeutics, Inc.
Collaborators
bluebird bio
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1. Study Identification

Unique Protocol Identification Number
NCT05445128
Brief Title
Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease
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
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Terminated
Why Stopped
This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety issue.
Study Start Date
June 24, 2022 (Actual)
Primary Completion Date
December 8, 2022 (Actual)
Study Completion Date
February 2, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Magenta Therapeutics, Inc.
Collaborators
bluebird bio

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
SCD, MGTA-145, Plerixafor, Mobilization, Apheresis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
1 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Part A: Single Day Dosing/Apheresis
Arm Type
Experimental
Arm Description
Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Arm Title
Part B: 2-Day Dosing/Apheresis
Arm Type
Experimental
Arm Description
MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
Intervention Type
Biological
Intervention Name(s)
MGTA-145
Intervention Description
MGTA-145 will be administered as an IV infusion
Intervention Type
Drug
Intervention Name(s)
Plerixafor
Intervention Description
240 µg/kg administered subcutaneously
Primary Outcome Measure Information:
Title
Apheresis Collection Yield
Description
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
Title
Assess incidence of 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
Title
Assess the incidence of 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
Title
Vital Signs - Number of participants with clinically significant changes from baseline in vital signs
Time Frame
Up to 11 days
Title
Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters.
Time Frame
Up to 11 days
Secondary Outcome Measure Information:
Title
Mobilization Effects of single-day and two-day dosing with MGTA-145 and plerixafor in peripheral blood in patients with SCD
Description
Determination of peak peripheral blood CD34+ counts
Time Frame
Up to 2 days
Title
Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics)
Time Frame
Up to 2 days
Title
Assess presence of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA])
Time Frame
Up to 11 days
Title
Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA])
Time Frame
Up to 11 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ji Hyun Lee, MD, MPH
Organizational Affiliation
Magenta Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
National Institutes of Health
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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