Single Treatment With FT1050 of an Ex-vivo Modulated Umbilical Cord Blood Unit

NCT01527838 | Completed Phase 1 DMC

• 1 other identifier: FT1050-02
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 3

Brief Summary

This trial is a prospective, open-label, single-arm trial of the safety of a single FT1050-treated CB unit for hematopoietic reconstitution after a reduced-intensity conditioning regimen for hematologic malignancies. A maximum of 40 eligible adult subjects will be enrolled and treated in the trial at approximately 2-4 centers within the U.S.

Conditions

Non-Hodgkin's Lymphoma (NHL); Hodgkin's Disease; Chronic Lymphocytic Leukemia (CLL); Acute Myelogenous Leukemia (AML); Acute Lymphoblastic Leukemia (ALL)

Keywords

Hematologic malignancies

Outcome Measures

Primary Outcomes (1)

• Neutrophil engraftment/chimerism

  To determine the minimally effective TNC dose for a single FT1050-treated CB unit based on neutrophil engraftment/chimerism when used for hematopoietic reconstitution following a reduced-intensity conditioning regimen for hematologic malignancies.

  Day 42

Secondary Outcomes (3)

• Safety

  Day 100

• Immune reconstitution

  2 years

• Donor search

  Day 0

Study Arms (1)

Single FT1050 treated UCB Unit

EXPERIMENTAL

Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

Biological: Single FT1050 treated UCB unit

Interventions

Single FT1050 treated UCB unit BIOLOGICAL

Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

Single FT1050 treated UCB Unit

Eligibility Criteria

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

You may qualify if:

• Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate. Eligible diseases and stages include:
• Non-Hodgkin's lymphoma or Hodgkin's lymphoma
• Chronic lymphocytic leukemia (CLL)
• Acute myelogenous leukemia (AML)
• Chronic myelogenous leukemia (CML)
• Lack of 5-6/6 HLA-matched related or 8/8 HLA-A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe.
• Identification of suitable backup CB unit(s) (single unit with pre-cryopreservation cell dose ≥ 2.5 x 10\^7 TNC/kg or two units with pre-cryopreservation cell dose ≥ 1.5 x 10\^7 TNC/kg each) and meeting minimum HLA match criteria.
• An acceptable alternative to one or two backup CB unit(s) is the identification of an eligible related haploidentical donor that meets minimum HLA match criteria.
• Age 18-65 years.
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
• Signed IRB approved Informed Consent Form (ICF).

You may not qualify if:

• The following hematologic malignancies are excluded:
• Myelofibrosis (Agnogenic Myeloid Metaplasia)
• Aplastic anemia.
• Previous treatment that included an allogeneic transplant
• Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular
• dysfunction (Ejection fraction \< 40%) as measured by gated radionucleotide 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 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), SGOT or SGPT \> 3 x upper limit of normal.
• Neurologic disease: symptomatic leukoencephalopathy, active 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.
• Participation in a concurrent clinical trial with a novel, unapproved investigational agent \< 30 days prior to Day 0.

Sponsors & Collaborators

• Fate Therapeutics: lead

Study Sites (3)

Massachusetts General Hospital

Boston, Massachusetts, 02214, United States

Location

Dana Farber Cancer Institute-Hematopoietic Stem Cell Transplant Program

Boston, Massachusetts, 02215, United States

Location

Ohio State Univeristy Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

Location

MeSH Terms

Conditions

Lymphoma, Non-Hodgkin; Hodgkin Disease; Leukemia, Lymphocytic, Chronic, B-Cell; Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, B-Cell; Leukemia, Lymphoid; Leukemia; Hematologic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Leukemia, Myeloid; Neoplasms by Site

Study Officials

• Samuel Dychter, MD

  Fate Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2012
• First Posted: February 7, 2012
• Study Start: January 1, 2012
• Primary Completion: October 1, 2013
• Study Completion: November 1, 2013
• Last Updated: September 12, 2016

Record last verified: 2015-01

Locations

US (3)