Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies

NCT01221857 | Completed Phase 1 Results DMC

• 1 other identifier: GC P#01.01.020
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord®, a UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells with a Second, Unmanipulated CBU in Patients with Hematological Malignancies

Conditions

Acute Lymphoblastic Leukemia (ALL); Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Non-Hodgkin's Lymphoma; Hodgkin's Disease

Keywords

Double Umbilical Cord Blood Stem Cell Transplantation; Hematological Malignancies; HLA Mismatched Donors; Cord Blood Transplantation

Outcome Measures

Primary Outcomes (2)

• Acute Toxicity Associated With the Infusion of NiCord

  Acute toxicity associated with the infusion of NiCord will be measured by adverse events within 24 hours post-infusion, defined as the acute toxicity period. Known adverse events associated with myeloablation and cord blood transplant were specifically monitored including fever, chills, allergic reaction/hypersensitivity, anaphylaxis, sinus bradycardia, sinus tachycardia, hypertension, hypotension, nausea, vomiting, diarrhea, dyspnea, hypoxia, hemoglobinuria, infection, flank pain and any other skin, CNS, cardiac, pulmonary or other toxicity manifestations.

  180 days post-transplant

• Proportion of Patients With Neutrophil Engraftment

  Neutrophil engraftment was defined as achieving an Absolute Neutrophil Count (ANC) of ≥500 mm3 for 3 consecutive measurements on different days by day 42 inclusive (the day of engraftment was defined as the first of these 3 days). The ANC recovery must be of donor origin documented by peripheral blood chimerism assays indicating less than or equal to 10% host cells in peripheral blood.

  42 days

Secondary Outcomes (2)

• Proportion of Patients Who Developed Acute GvHD Grade II-IV and III-IV

  180 days

• Non-relapse Mortality

  100 days

Study Arms (1)

NiCord

EXPERIMENTAL

Drug: NiCord®

Interventions

NiCord® DRUG

NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.

NiCord

Eligibility Criteria

• Age: 8 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Applicable disease and eligible for myeloablative SCT
• Patients must have two partially HLA-matched CBUs
• Back-up stem cell source
• Adequate Karnofsky Performance score or Lansky Play-Performance scale
• Sufficient physiological reserves
• Signed written informed consent

You may not qualify if:

• HLA-matched related donor able to donate
• Prior allogeneic HSCT
• Lymphoma patients with progressive disease
• Other active malignancy
• Human immunodeficiency virus (HIV) infection
• Active or uncontrolled infection
• Active/symptoms of central nervous system (CNS) disease
• Pregnancy or lactation

Sponsors & Collaborators

• Gamida Cell ltd: lead

Study Sites (2)

Loyola University, Cardinal Bernardin Cancer Center

Maywood, Illinois, 60153, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Related Publications (2)

• Horwitz ME, Chao NJ, Rizzieri DA, Long GD, Sullivan KM, Gasparetto C, Chute JP, Morris A, McDonald C, Waters-Pick B, Stiff P, Wease S, Peled A, Snyder D, Cohen EG, Shoham H, Landau E, Friend E, Peleg I, Aschengrau D, Yackoubov D, Kurtzberg J, Peled T. Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment. J Clin Invest. 2014 Jul;124(7):3121-8. doi: 10.1172/JCI74556. Epub 2014 Jun 9.

  PMID: 24911148 DERIVED

• Peled T, Shoham H, Aschengrau D, Yackoubov D, Frei G, Rosenheimer G N, Lerrer B, Cohen HY, Nagler A, Fibach E, Peled A. Nicotinamide, a SIRT1 inhibitor, inhibits differentiation and facilitates expansion of hematopoietic progenitor cells with enhanced bone marrow homing and engraftment. Exp Hematol. 2012 Apr;40(4):342-55.e1. doi: 10.1016/j.exphem.2011.12.005. Epub 2011 Dec 20.

  PMID: 22198152 DERIVED

Related Links

• Gamida Cell Ltd.
• Duke University Medical Center
• Loyola University

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Lymphoma, Non-Hodgkin; Hodgkin Disease; Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Lymphoma; Neoplasms by Site

Results Point of Contact

• Title: Kelly Myers
• Organization: Gamida Cell

clinicaltrials@gamida-cell.com

+972-2-659-5631

Study Officials

• David Snyder, PhD

  Gamida Cell ltd

  STUDY DIRECTOR

• Joanne Kurtzberg, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• Mitchell Horwitz, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• Patrick Stiff, MD

  Loyola University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

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: October 14, 2010
• First Posted: October 15, 2010
• Study Start: November 1, 2010
• Primary Completion: September 1, 2012
• Study Completion: May 1, 2013
• Last Updated: August 3, 2021
• Results First Posted: February 26, 2019

Record last verified: 2021-08

Locations

US (2)