Study of ProTmune for Allogeneic HCT in Adult Patients With Hematologic Malignancies

NCT ID: NCT02743351

Last Updated: 2023-02-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 96 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-12-20
• Study Completion Date: 2021-11-05

Brief Summary

This study is a Phase 1, non-randomized, open-label/Phase 2 randomized, blinded study of ProTmune (ex vivo programmed mobilized peripheral blood cells) versus non-programmed mobilized peripheral blood cells for allogeneic hematopoietic cell transplantation (HCT) in adult subjects aged 18 years and older with hematologic malignancies. A total of 88 study subjects were treated in the trial at approximately 15 centers in the US.

Conditions

Hematologic Malignancies; Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndromes; Chronic Myelogenous Leukemia; Acute Graft-versus-host Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: PREVENTION
• Blinding Strategy: QUADRUPLE

Study Groups

ProTmune

Conditioning regimen consisting of one of the following five preparative regimens: fludarabine and busulfan (FluBu4); busulfan (Bu) and cyclophosphamide (Cy); Cy and >or=12 Gy total body irradiation (TBI); TBI and etoposide; or fludarabine and melphalan (FluMel 140).

Subjects will receive mPB cells from an available 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated peripheral blood cell donor that were programmed ex vivo with ProTmune.

Group Type: EXPERIMENTAL

ProTmune

Intervention Type: BIOLOGICAL

Ex-vivo, programmed mobilized peripheral blood (mPB) cells

Control Arm

Conditioning regimen consisting of one of the following five preparative regimens: fludarabine and busulfan (FluBu4); busulfan (Bu) and cyclophosphamide (Cy); Cy and >or=12 Gy total body irradiation (TBI); TBI and etoposide; or fludarabine and melphalan (FluMel 140).

Subjects will receive unmanipulated mPB cells from an available 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated peripheral blood cell donor.

Group Type: ACTIVE_COMPARATOR

Control Arm

Intervention Type: BIOLOGICAL

Untreated mobilized peripheral blood (mPB) cells

Interventions

ProTmune

Ex-vivo, programmed mobilized peripheral blood (mPB) cells

Intervention Type: BIOLOGICAL

Control Arm

Untreated mobilized peripheral blood (mPB) cells

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Male and female patients aged 18 years and older, inclusive;

2. Patients must have a hematologic malignancy for which allogeneic hematopoietic peripheral blood cell transplantation is deemed clinically appropriate.

Eligible diseases and stages include the following:

1. Acute myeloid leukemia

2. Acute lymphoblastic leukemia, including T lymphoblastic lymphoma with a history of marrow involvement

3. Myelodysplastic Syndrome

4. Chronic myelogenous leukemia

3. Availability of a suitable 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated mPB donor;

4. mBP donor collection that meets protocol specifications;

5. Adequate performance status, defined as Karnofsky score greater than or equal to 70%;

6. For female patients of childbearing potential, all of the following criteria must be met:

• They are not pregnant (i.e., female patients must have a negative serum pregnancy test at screening);
• They are not breastfeeding;
• They do not plan to become pregnant during the study; and
• They are using an effective method of contraception from screening to the end of the study, unless their sexual partner is surgically sterile.

7. For male patients, agreement to use condoms with spermicide during sexual intercourse from screening to the end of study; and

8. Willingness and ability to sign an IRB/IEC-approved ICF before performance of any study-specific procedures or tests and to comply with protocol visits, and study procedures.

Exclusion Criteria

1. Phase 1 only: Known bone marrow fibrosis; Phase 2 only: Bone marrow fibrosis grade 3 (severe) or greater;

2. Positive serology for human immunodeficiency virus (HIV) or human T-cell lymphotropic virus (HTLV) at any time prior to enrollment;

3. Currently uncontrolled bacterial, viral, or fungal infection (progression of clinical symptoms despite therapy);

4. Prior autologous or allogeneic HCT;

5. Active malignancy, other than the one for which the allogeneic mPB transplant is being performed, within 12 months of enrollment, excluding superficial basal cell and carcinoma in situ cervical cancer;

6. Pulmonary disease, renal dysfunction, hepatic disease, cardiac disease, neurologic disease;

7. Participation in another clinical trial involving an investigational product within 30 days prior to screening; or

8. Any condition or therapy, which, in the opinion of the Investigator, might pose a risk to the patient or make participation in the study not in the best interest of the patient.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fate Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sarah Cooley, MD

Role: STUDY_DIRECTOR

Fate Therapeutics

Locations

University of Alabama

Birmingham, Alabama, United States

City of Hope

Duarte, California, United States

University of California, San Diego (UCSD) Moores Cancer Center

San Diego, California, United States

University of Chicago

Chicago, Illinois, United States

Indiana Blood and Marrow Transplant

Indianapolis, Indiana, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, United States

Weill Cornell Medicine

New York, New York, United States

Jewish Hospital

Cincinnati, Ohio, United States

The Ohio State University

Columbus, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

Sarah Cannon Research Institute

Nashville, Tennessee, United States

Texas Transplant Institute

San Antonio, Texas, United States

Huntsman Cancer Institute (University of Utah)

Salt Lake City, Utah, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

PT-001

Identifier Type: -

Identifier Source: org_study_id