MGTA-145 + Plerixafor in the Mobilization of HSCs for Allogeneic Transplant in Hematologic Malignancies

NCT04762875 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 145-ADS-202
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 8

Brief Summary

This research study tests a new medicine for mobilizing stem cells so they can be collected and used for allogeneic stem cell transplant for treatment of hematological malignancies. MGTA-145, the new medicine, will be given with plerixafor.

Conditions

Related Donors Donating PBSC to a Family Member; Healthy Donors; Acute Myelogenous Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (1)

• HSC Yield in Apheresis Product

  Number of subjects with adequate number of hematopoietic stem cells (≥ 2.0 x 10\^6 CD34+ cells/kg) in one apheresis setting.

  Up to 2 days

Secondary Outcomes (6)

• HSC Yield in Apheresis Product

  Up to 2 days

• Adverse Events Experienced by Donors

  Baseline though day 180

• Graft Durability

  Day 28

• Graft-versus Host Disease (GVHD)

  Day 100

• Treatment-related Mortality

  Day 100

Study Arms (1)

Single dose MGTA-145 plus plerixafor followed by apheresis

EXPERIMENTAL

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

Biological: MGTA-145; Biological: Plerixafor

Interventions

MGTA-145 BIOLOGICAL

MGTA-145 will be be administered as an IV infusion

Single dose MGTA-145 plus plerixafor followed by apheresis

Plerixafor BIOLOGICAL

240 µg/kg subcutaneously

Also known as: Mozobil

Single dose MGTA-145 plus plerixafor followed by apheresis

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Donor medical suitability and eligibility will be determined following Institution or NMDP/Be The Match standards
• Age 18-65 years old at the time of signing informed consent
• /8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer unrelated donor
• Fulfill Institution or NMDP/Be The Match criteria to serve as a mobilized blood cell donor
• Serum creatinine \< 1.5 x institution upper limit of normal (ULN) or estimated creatinine clearance (CRCL) \> 50 mL/min using the Modification of Diet in Renal Disease Study (MDRD) equation or similar method
• At least 18 years old at the time of signing informed consent
• Has an available 8/8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer unrelated donor willing to donate peripheral blood stem cells (PBSC) for transplant
• Fulfill additional individual Transplant Center Criteria for transplant beyond NMDP/Be The Match criteria
• One of the following diagnoses:
• Acute myelogenous leukemia (AML) in 1st remission or beyond with ≤ 5% marrow blasts and no circulating blasts. Documentation of bone marrow assessment will be accepted within 45 days prior to the date of consent.
• Acute lymphoblastic leukemia (ALL) in 1st remission or beyond with ≤ 5% marrow blasts and no circulating blasts. Documentation of bone marrow assessment will be accepted within 45 days prior to the date of consent.
• Patients with myelodysplasia (MDS) with no circulating blasts and with less than 10% blasts in the bone marrow (higher blast percentage allowed in MDS due to lack of differences in outcomes with \< 5% or 5-10% blasts in MDS). Documentation of bone marrow assessment will be accepted within 45 days prior to the date of consent.
• Cardiac function: Left ventricular ejection fraction at least 45% based on most recent echocardiogram or MUGA results obtained via standard of care
• Estimated creatinine clearance acceptable per local institutional guidelines
• Pulmonary function: diffusing capacity of the lungs for carbon monoxide (DLCO) corrected for hemoglobin at least 50% and forced expiratory volume in first second (FEV1) predicted at least 50% based on most recent DLCO results obtained via standard of care

You may not qualify if:

• Donor unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
• Donor already enrolled on another investigational agent study
• Pregnant or breastfeeding females, sexually active female and male donors not willing or able to use adequate contraception, or males who do not agree to refrain from donating sperm, from the time of consent through 3 months after treatment with MGTA-145 + plerixafor
• Subject unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
• Subject whose donor does not meet the eligibility criteria and is a screen fail
• Subjects with a prior allogeneic transplant
• Subjects with active, uncontrolled infection at the time of the transplant preparative regimen
• Pregnant or breastfeeding females, sexually active female or male subjects not willing or able to use adequate contraception, or males who do not agree to refrain from donating sperm, from the time of consent through 3 months after PBSC infusion
• Subjects with clinical evidence of active Central Nervous System (CNS) tumor involvement as evidenced by documented disease on examination of spinal fluid or MRI within 45 days of start of conditioning
• A condition, which, in the opinion of the clinical investigator, would interfere with the evaluation of primary and secondary endpoints
• Planned treatment with a new investigational agent from the time of transplant through 30 days post-transplant

Sponsors & Collaborators

• Ensoma: lead
• National Marrow Donor Program: collaborator

Study Sites (8)

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

Stanford Health Care

Stanford, California, 94305, United States

Location

Emory University Hospital

Atlanta, Georgia, 30322, United States

Location

Mayo Clinic Rochester

Rochester, Minnesota, 55902, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14203, United States

Location

Ohio State Medical Center, James Cancer Center

Columbus, Ohio, 43210, United States

Location

M.D. Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Be The Match Collection Center Seattle

Seattle, Washington, 98101, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Myelodysplastic Syndromes

Interventions

plerixafor

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Bone Marrow Diseases

Results Point of Contact

• Title: Elizabeth Hook
• Organization: Ensoma

ehook@ensoma.com

617-766-3917

Study Officials

• Steven Devine, MD

  National Marrow Donor Program

  STUDY CHAIR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2021
• First Posted: February 21, 2021
• Study Start: June 16, 2021
• Primary Completion: September 14, 2021
• Study Completion: March 14, 2022
• Last Updated: November 6, 2024
• Results First Posted: August 29, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)