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

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

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.

Study Overview

• Status: Terminated
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Biological: MGTA-145; Drug: 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.

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Magenta Clinical Trials; Phone Number: 857-242-0170; Email: clinicaltrials@magentatx.com

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 35 years (Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part A: Single Day Dosing/Apheresis; Single dose of MGTA-145 in combination with plerixafor followed by apheresis	Biological: MGTA-145; MGTA-145 will be administered as an IV infusion; Drug: Plerixafor; 240 µg/kg administered subcutaneously
Experimental: Part B: 2-Day Dosing/Apheresis; MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days	Biological: MGTA-145; MGTA-145 will be administered as an IV infusion; Drug: Plerixafor; 240 µg/kg administered subcutaneously

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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 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.		Up to 30 days
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.		Up to 11 days
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.		Up to 11 days

Secondary Outcome Measures

Outcome Measure	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	Up to 2 days
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])		Up to 11 days
Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA])		Up to 11 days

Collaborators and Investigators

• Sponsor: Ensoma
• Collaborators: bluebird bio
• Investigators: Study Director: Ji Hyun Lee, MD, MPH, Magenta Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): June 24, 2022
• Primary Completion (Actual): December 8, 2022
• Study Completion (Actual): February 2, 2023

Study Registration Dates

• First Submitted: May 26, 2022
• First Submitted That Met QC Criteria: July 5, 2022
• First Posted (Actual): July 6, 2022

Study Record Updates

• Last Update Posted (Actual): January 11, 2024
• Last Update Submitted That Met QC Criteria: January 9, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: Apheresis; SCD; Mobilization; Plerixafor; MGTA-145
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell; Anti-Infective Agents; Antiviral Agents; Anti-HIV Agents; Anti-Retroviral Agents; Plerixafor
• Other Study ID Numbers: 145-SCD-204

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No