Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM

NCT ID: NCT01816230

Last Updated: 2020-10-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-04-30
• Study Completion Date: 2018-06-30

Brief Summary

A Study Evaluating the Safety and Efficacy of Transplantation of a single cord blood unit (CBU) of NiCord®, umbilical cord blood-derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hematological Malignancies.

Detailed Description

Umbilical cord blood (UCB) is an alternative stem cell source for hematopoietic stem cell transplantations (HSCT) and can be used for the treatment of various life-threatening diseases, such as hematological malignancies or genetic blood disorders, in such cases where a matched related stem cell donor is not available. However, the major drawback of using this valuable stem cells source is the limited cell dose in a single cord blood unit (CBU), which was shown to be associated with inadequate hematopoietic reconstitution and high risk of transplant-related mortality. To improve outcomes and extend applicability of UCB transplantation, one potential solution is ex vivo expansion of UCB-derived stem and progenitor cells. NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord® is based on a novel technology for the ex vivo cell expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of the short and long-term reconstitution progenitor cells transplanted, NiCord® has the potential to enable the broader application of UCB transplantation, and improve the clinical outcomes of UCB transplantation.

The study is designed as a multi center, single arm study, evaluating the safety and efficacy of the transplantation of NiCord® to patients with hematological malignancies following myeloablative therapy.

Total study duration is approximately 400 days from the signing of informed consent to the last visit one year following transplantation

The overall study objective is to evaluate the safety and efficacy of NiCord®: single ex-vivo expanded cord blood unit transplantation in patients with hematological malignancies following myeloablative therapy as follows:

The main study objectives are to assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation and to assess the incidence of secondary graft failure at 180 days following transplantation of NiCord® Ten evaluable patients recruited for the study should be 12-65 years of age, up to a maximum of 15 treated patients.

Conditions

Hematological Malignancies; Acute Lymphoblastic Leukemia (ALL); Acute Myeloid Leukemia (AML); Myelodysplastic Syndrome (MDS)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

NiCord®

NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells.

Group Type: EXPERIMENTAL

NiCord®

Intervention Type: DRUG

Interventions

NiCord®

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• HLA-matched donor able to donate
• Prior allogeneic HSCT
• Other active malignancy
• Active or uncontrolled infection
• Active/symptoms of central nervous system (CNS) disease
• Pregnancy or lactation

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gamida Cell ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mitchell Horwitz, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

Cedars-Sinai Medical Center

Los Angeles, California, United States

Loyola University, Cardinal Bernardin Cancer Center

Maywood, Illinois, United States

University of Minnesota

Minneapolis, Minnesota, United States

Steven and Alexandra Cohen Children's Medical Center

New York, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Cleveland Clinic

Cleveland, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

Vanderbilt

Nashville, Tennessee, United States

Children Hospital Gaslini Scientific Institute

Genoa, , Italy

University of Turin

Turin, , Italy

University Medical Center Utrecht

Utrecht, , Netherlands

National University Cancer Institute

Singapore, , Singapore

Singapore General Hospital

Singapore, , Singapore

University Hospital Vall d´Hebron

Barcelona, , Spain

Hospital Universitario La Fe

Valencia, , Spain

Countries

United States; Italy; Netherlands; Singapore; Spain

References

Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, Nierkens S, Jagasia M, Wagner JE, Kuball J, Koh LP, Majhail NS, Stiff PJ, Hanna R, Hwang WYK, Kurtzberg J, Cilloni D, Freedman LS, Montesinos P, Sanz G. Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374. doi: 10.1200/JCO.18.00053. Epub 2018 Dec 4.

Reference Type: DERIVED

PMID: 30523748 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

GC P#03.01.020

Identifier Type: -

Identifier Source: org_study_id