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

Acute Myeloid Leukemia (AML) Clinical Trial

Official title:

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adolescent and Adult Patients With Hematological Malignancies

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01816230
• Other study ID #: GC P#03.01.020
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: April 2013
• Est. completion date: June 2018

Study information

• Verified date: October 2020
• Source: Gamida Cell ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 36
• Est. completion date: June 2018
• Est. primary completion date: June 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Acute Lymphoblastic Leukemia (ALL)
• Acute Myeloid Leukemia (AML)
• Hematologic Neoplasms
• Hematological Malignancies
• Leukemia
• Leukemia, Myeloid, Acute
• Myelodysplastic Syndrome (MDS)
• Myelodysplastic Syndromes
• Neoplasms
• Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Preleukemia

Intervention

Drug:
• NiCord®

Locations

Country	Name	City	State
Italy	Children Hospital Gaslini Scientific Institute	Genoa
Italy	University of Turin	Turin
Netherlands	University Medical Center Utrecht	Utrecht
Singapore	National University Cancer Institute	Singapore
Singapore	Singapore General Hospital	Singapore
Spain	University Hospital Vall d´Hebron	Barcelona
Spain	Hospital Universitario La Fe	Valencia
United States	Cleveland Clinic	Cleveland	Ohio
United States	Duke University Medical Center	Durham	North Carolina
United States	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	Vanderbilt	Nashville	Tennessee
United States	Steven and Alexandra Cohen Children's Medical Center	New York	New York
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Gamida Cell ltd

Countries where clinical trial is conducted

United States,  Italy,  Netherlands,  Singapore,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Engraftment	Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.	42 days