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

Sponsor

Magenta Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05445128

Collaborator

bluebird bio (Industry)

Enrollment

Locations

Arms

17.2

Anticipated Duration (Months)

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease	Biological: MGTA-145 Drug: Plerixafor	Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

14 participants

Allocation:

Non-Randomized

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Jun 24, 2022

Anticipated Primary Completion Date :

Dec 1, 2023

Anticipated Study Completion Date :

Dec 1, 2023

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Apheresis Collection Yield [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 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

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]
Determination of peak peripheral blood CD34+ counts
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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 35 Years

Sexes Eligible for Study:

All

Accepts 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.