The PUMA Trial is a Trial of a Single ProHema Modulated-Cord Blood (CB) Unit as Part of a Double CB Transplant in Patients With Hematologic Malignancies.

NCT01627314 | Terminated Phase 2 DMC FDA Drug

• 1 other identifier: FT1050-03
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 11

Brief Summary

This study is an open-label randomized, prospectively and historically controlled trial of the safety and efficacy of a single ProHema-CB unit used as part of a double CB transplant following myeloablative or reduced intensity conditioning for subjects age 15-65 years with hematologic malignancies. A maximum of 60 eligible subjects will be enrolled and treated in the trial at approximately 10 centers within the U.S.

Conditions

Hematologic Malignancies

Outcome Measures

Primary Outcomes (2)

• Rate of early neutrophil engraftment using Myeloablative Conditioning

  To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant following myeloablative conditioning for subjects with hematologic malignancies.

  Neutrophil engraftment < 26 days

• Rate of early neutrophil engraftment using Reduced Intensity Conditioning

  To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant following reduced intensity conditioning for subjects with hematologic malignancies.

  Neutrophil engraftment < 21 days

Study Arms (4)

ProHema-CB with MAC Preparative Regimen

EXPERIMENTAL

ProHema-CB and Wash-Only CB Unit with myeloablative conditioning regimen (MAC)

Biological: ProHema-CB

ProHema-CB with RIC Preparative Regimen

EXPERIMENTAL

ProHema-CB and Wash-Only CB Unit with reduced intensity conditioning regimen (RIC)

Biological: ProHema-CB

Control Arm with MAC Preparative Regimen

PLACEBO COMPARATOR

Two Wash-Only CB Units (Untreated CB) with myeloablative conditioning regimen

Biological: Untreated CB

Control Arm with RIC Preparative Regimen

PLACEBO COMPARATOR

Two Wash-Only CB Units (Untreated CB) with reduced intensity conditioning regimen

Biological: Untreated CB

Interventions

ProHema-CB BIOLOGICAL

Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

ProHema-CB with MAC Preparative Regimen; ProHema-CB with RIC Preparative Regimen

Untreated CB BIOLOGICAL

Cord Blood

Control Arm with MAC Preparative Regimen; Control Arm with RIC Preparative Regimen

Eligibility Criteria

• Age: 15 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate. Eligible diseases and stages include:
• Acute lymphoblastic leukemia (ALL) (including T lymphoblastic lymphoma) in complete remission (CR).
• Remission is defined as \< 5% blasts with no morphological characteristics of acute leukemia in a bone marrow with \> 5% cellularity.
• Myelodysplastic disease, International Prognostic Scoring System (IPSS) Intermediate-2 or High risk (e.g., refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Patients have to have received leukemia type induction chemotherapy within ≤ 3 months and with ≤ 10% blasts by a bone marrow aspirate; a single hypomethylating agent is not considered adequate cytotoxic chemotherapy; all subtypes except chronic myelomonocytic leukemia (CMML).
• Acute myelogenous leukemia (AML) in high risk first CR or second or subsequent CR.
• High risk first CR is defined by but is not limited to at least one of the following factors: greater than one cycle of induction chemotherapy to achieve CR, prior myelodysplastic syndrome (MDS), presence of fms-like tyrosine kinase 3 (FLT3) abnormalities, French-American-British (FAB) M6 or M7 subtypes of leukemia, or adverse cytogenetics.
• Remission is defined as \< 5% blasts with no morphological characteristics of acute leukemia (e.g. Auer Rods) in a bone marrow with \> 5% cellularity.
• AML arising from myelofibrosis is not permitted.
• Biphenotypic/undifferentiated leukemia in first or subsequent CR (same definition of CR as for ALL/AML).
• Chronic myelogenous leukemia (CML) with prior exposure to cytotoxic chemotherapy for the treatment of blast phase or with demonstrated intolerance to at least 2 tyrosine kinase inhibitors.
• Non Hodgkin's lymphoma (T cell, large cell or mantle cell) or Hodgkin's lymphoma in second or subsequent CR or in partial remission (PR) with documented chemosensitivity. In addition, marginal zone lymphoma or follicular lymphoma that has progressed after ≥ 2 therapies (excluding single agent rituximab).
• If the myeloablative conditioning regimen is selected, a history of prior myeloablative procedure is not allowed.
• If the reduced intensity conditioning regimen is selected, and the subject has had a prior autologous transplant, it must have taken place \> 3 months from anticipated Day 0 visit.
• Lack of suitable 5 6/6 HLA matched related or (if institutional guidelines dictate) suitable 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe.
• Both cord blood units (CBUs) are qualified by Fate Therapeutics

You may not qualify if:

• History of prior allogeneic transplantation
• Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction (Ejection fraction \< 50%) as measured by gated radionuclide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension; history of myocardial infarction with depressed ejection fraction.
• Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected diffusing capacity for carbon monoxide (DLCO) of \< 50% of predicted, corrected for hemoglobin.
• Renal disease: serum creatinine \> 2.0 mg/dl and calculated creatinine clearance \< 40 mL/min.
• Hepatic disease: serum bilirubin \> 2.0 mg/dl (except in the case of Gilbert's syndrome or ongoing hemolytic anemia), aspartate aminotransferase (SGOT) or alanine aminotransferase (SGPT) \> 5 × upper limit of normal.
• Neurologic disease: symptomatic leukoencephalopathy, active central nervous system (CNS) malignancy or other neuropsychiatric abnormalities believed to preclude transplantation.
• HIV antibody.
• Uncontrolled infection.
• Pregnancy or breast feeding mother.
• Inability to comply with the requirements for care after allogeneic stem cell transplantation.

Sponsors & Collaborators

• Fate Therapeutics: lead

Study Sites (11)

City of Hope

Duarte, California, 91010, United States

Location

Emory University-Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

Location

Dana-Farber Cancer Institute- Hematopoietic Stem Cell Transplant Program

Boston, Massachusetts, 02215, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Mount Sinai Hospital

New York, New York, 10029, United States

Location

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

Location

Oregon Health Sciences

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Chris Storgard, M.D.

  Fate Therapeutics

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: June 21, 2012
• First Posted: June 25, 2012
• Study Start: July 1, 2012
• Primary Completion: March 1, 2016
• Study Completion: May 1, 2017
• Last Updated: November 30, 2021

Record last verified: 2021-11

Locations

US (11)