Clinical trial NCT02743351: Study of ProTmune for Allogeneic HCT in Adult Patients With Hematologic Malignancies
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Hematologic Malignancies
Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Myelodysplastic Syndromes
Chronic Myelogenous Leukemia
Acute Graft-versus-host Disease
Keywords: Cell Transplants
Hematopoietic Cell Transplant
HCT
Hematologic Malignancies
Hematologic Malignancy
Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
ALL
AML
Myelodysplastic Syndrome
MDS
Allogeneic
CML
Stem Cell Transplant
Transplant
aGvHD
Acute graft-versus-host Disease
cGvHD
Chronic graft-versus-host Disease
Lead sponsor: Fate Therapeutics
Sponsor class: INDUSTRY
First posted: 2016-04-19
Enrollment: 96
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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.


Eligibility criteria: Key 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.

Key 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.

Primary outcomes:
measure: Percentage of Participants With Cumulative Incidence of Grade II to IV aGvHD Through Day +100 Based on Investigator Assessment
timeFrame: 100 days post-HCT
Secondary outcomes:
measure: 1-year GvHD-free, Relapse-free Survival (GRFS)
timeFrame: 365 days post-HCT
measure: Percentage of Subjects Alive Without Relapse and Without Moderate or Severe cGvHD at Day +365 - mITT Population
timeFrame: 365 days post-HCT
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Duarte
country: United States
facility: City of Hope
state: California
city: San Diego
country: United States
facility: University of California, San Diego (UCSD) Moores Cancer Center
state: California
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Blood and Marrow Transplant
state: Indiana
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Detroit
country: United States
facility: Barbara Ann Karmanos Cancer Institute
state: Michigan
city: New York
country: United States
facility: Weill Cornell Medicine
state: New York
city: Cincinnati
country: United States
facility: Jewish Hospital
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Nashville
country: United States
facility: Sarah Cannon Research Institute
state: Tennessee
city: San Antonio
country: United States
facility: Texas Transplant Institute
state: Texas
city: Salt Lake City
country: United States
facility: Huntsman Cancer Institute (University of Utah)
state: Utah
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
Study officials:
affiliation: Fate Therapeutics
name: Sarah Cooley, MD
role: STUDY_DIRECTOR
References: