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

NCT05445128 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 145-SCD-204
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 3

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.

Conditions

Sickle Cell Disease

Keywords

SCD; MGTA-145; Plerixafor; Mobilization; Apheresis

Outcome Measures

Primary Outcomes (5)

• 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 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.

  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.

  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

  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.

  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

  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

Study Arms (2)

Part A: Single Day Dosing/Apheresis

EXPERIMENTAL

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

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

Biological: MGTA-145; Drug: Plerixafor

Interventions

MGTA-145 BIOLOGICAL

MGTA-145 will be administered as an IV infusion

Part A: Single Day Dosing/Apheresis; Part B: 2-Day Dosing/Apheresis

Plerixafor DRUG

240 µg/kg administered subcutaneously

Part A: Single Day Dosing/Apheresis; Part B: 2-Day Dosing/Apheresis

Eligibility Criteria

• Age: 18 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Ensoma: lead
• Genetix Biotherapeutics Inc.: collaborator

Study Sites (3)

National Institutes of Health

Bethesda, Maryland, 20892, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

plerixafor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

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.

Results Point of Contact

• Title: Ensoma head of regulatory or head of clinical research & development
• Organization: Ensoma

ddietz@ensoma.com

6127191200

Study Officials

• Ji Hyun Lee, MD, MPH

  Magenta Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2022
• First Posted: July 6, 2022
• Study Start: June 24, 2022
• Primary Completion: December 8, 2022
• Study Completion: February 2, 2023
• Last Updated: July 25, 2025
• Results First Posted: July 25, 2025

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)