Study of ProTmune for Allogeneic HCT in Adult Patients With Hematologic Malignancies
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 in Adult Subjects With Hematologic Malignancies
1 other identifier
interventional
96
1 country
15
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Dec 2016
Longer than P75 for phase_1
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 9, 2016
CompletedFirst Posted
Study publicly available on registry
April 19, 2016
CompletedStudy Start
First participant enrolled
December 20, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 4, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
November 5, 2021
CompletedResults Posted
Study results publicly available
February 8, 2023
CompletedFebruary 8, 2023
December 1, 2022
4 years
February 9, 2016
October 14, 2022
January 12, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Participants With Cumulative Incidence of Grade II to IV aGvHD Through Day +100 Based on Investigator Assessment
Acute GvHD (aGvHD) is assessed by assigning the clinical stage for the target organs (skin, liver, and gut) along with assigning an overall grade based on the minimum degree of organ involvement required to confer that grade. Grade II is defined as Stage 1 skin, Stage 1 liver, or Stage 1 GI; Grade III is defined as any Stage 1-3 skin, and Stage 2-3 liver, or Stage 2-4 GI; Grade IV is defined as Stage 4 skin, or Stage 4 liver and any Stage GI. A higher overall grade indicates a more severe outcome. The cumulative incidence of CIBMTR Grade II to IV aGVHD through approximately 100 days following HCT is measured by the percentage of participants who experienced grade II to IV aGVHD. The cumulative incidence and the associated 95% confidence interval are estimated using a competing risk analysis with death and relapse without grade II-IV aGvHD as a competing risk.
100 days post-HCT
Secondary Outcomes (2)
1-year GvHD-free, Relapse-free Survival (GRFS)
365 days post-HCT
Percentage of Subjects Alive Without Relapse and Without Moderate or Severe cGvHD at Day +365 - mITT Population
365 days post-HCT
Study Arms (2)
ProTmune
EXPERIMENTALConditioning 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.
Control Arm
ACTIVE COMPARATORConditioning 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.
Interventions
Eligibility Criteria
You may qualify if:
- Male and female patients aged 18 years and older, inclusive;
- 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:
- Acute myeloid leukemia
- Acute lymphoblastic leukemia, including T lymphoblastic lymphoma with a history of marrow involvement
- Myelodysplastic Syndrome
- Chronic myelogenous leukemia
- Availability of a suitable 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated mPB donor;
- mBP donor collection that meets protocol specifications;
- Adequate performance status, defined as Karnofsky score greater than or equal to 70%;
- 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.
- +2 more criteria
You may not qualify if:
- Phase 1 only: Known bone marrow fibrosis; Phase 2 only: Bone marrow fibrosis grade 3 (severe) or greater;
- Positive serology for human immunodeficiency virus (HIV) or human T-cell lymphotropic virus (HTLV) at any time prior to enrollment;
- Currently uncontrolled bacterial, viral, or fungal infection (progression of clinical symptoms despite therapy);
- Prior autologous or allogeneic HCT;
- 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;
- Pulmonary disease, renal dysfunction, hepatic disease, cardiac disease, neurologic disease;
- Participation in another clinical trial involving an investigational product within 30 days prior to screening; or
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
University of Alabama
Birmingham, Alabama, United States
City of Hope
Duarte, California, 91010, 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, 46237, United States
Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
Weill Cornell Medicine
New York, New York, United States
Jewish Hospital
Cincinnati, Ohio, United States
The Ohio State University
Columbus, Ohio, 43210, United States
Oregon Health & Science University
Portland, Oregon, United States
Sarah Cannon Research Institute
Nashville, Tennessee, 37203, United States
Texas Transplant Institute
San Antonio, Texas, United States
Huntsman Cancer Institute (University of Utah)
Salt Lake City, Utah, 84103, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Executive Director, Clinical Operations
- Organization
- Fate Therapeutics
Study Officials
- STUDY DIRECTOR
Sarah Cooley, MD
Fate Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 9, 2016
First Posted
April 19, 2016
Study Start
December 20, 2016
Primary Completion
December 4, 2020
Study Completion
November 5, 2021
Last Updated
February 8, 2023
Results First Posted
February 8, 2023
Record last verified: 2022-12
Data Sharing
- IPD Sharing
- Will not share