Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

April 27, 2022 updated by: Gamida Cell ltd

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

In Part 1 of this study, NiCord® will be administered to the patient in conjunction with a second, unmanipulated CBU. In Part 2 of this study, NiCord® will be administered to the patient without a second, unmanipulated CBU. The study duration per patient is approximately 270 days from signing of informed consent to last visit on day 180 post-transplant.

The overall study objectives of part 1 of this study are to evaluate the safety and efficacy of co-transplantation of NiCord® and an unmanipulated CBU in patients with Hemoglobinopathies (Sickle Cell Disease (SCD), or thalassemia major) following myeloablative therapy. The overall study objectives of part 2 of this study are to evaluate the safety and efficacy of transplantation of NiCord® in patients with Hemoglobinopathies (Sickle Cell Disease (SCD), or thalassemia major) following myeloablative therapy.

The study hypothesis for part 1 of this study is that the co-transplantation of NiCord® and an unmanipulated unrelated cord blood graft in patients with hemoglobinopathies (SCD, or thalassemia major) following myeloablative preparative therapy will be safe and will enable cord blood engraftment. The study hypothesis for part 2 of this study is that transplantation of NiCord® in patients with hemoglobinopathies (SCD, or thalassemia major) following myeloablative preparative therapy will be safe and will enable cord blood engraftment.

Up to fifteen (15) evaluable patients recruited for part 1 of the study and up to five (5) patients for part 2 of the study should be 2-45 years of age, at least 10 kg in weight, have symptomatic SCD or thalassemia major and should be considered as candidates for allogeneic myeloablative HSCT for the treatment of SCD.

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 11040
        • Steven & Alexandra Cohen Children's Medical Center, New York
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
    • Texas
      • Houston, Texas, United States, 77030-4009
        • The University of Texas M. D. Anderson Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 45 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Must be 2 - 45 years of age and at least 10 kg
  • Must have clinically severe SCD (SS, SC or SBeta0 Thal) or thalassemia major and be eligible for myeloablative SCT
  • Must have two partially HLA-matched CBUs for part 1; and one partially HLA-matched CBU for part 2
  • Back-up autologous stem cells harvested from bone marrow
  • Adequate Karnofsky Performance score or Lansky Play-Performance scale
  • Sufficient physiological reserves
  • Signed written informed consent

Exclusion Criteria:

  • HLA-matched related donor able to donate
  • Severe alloimmunization with inability to guarantee a supply of adequate PRBC donors
  • Prior allogeneic hematopoietic SCT within the last 12 months or reduced-intensity transplant within the past 6 months
  • Human immunodeficiency virus (HIV) infection
  • Active or uncontrolled infection
  • Pregnancy or lactation

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NiCord
NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Drug: NiCord
NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity.
Time Frame: 24 hours post-infusion
Assessment of acute toxicity associated with the infusion of NiCord within 24 hours post-infusion.
24 hours post-infusion
Assessment of Cumulative Incidence of Donor-derived Neutrophil Engraftment.
Time Frame: By Day 42
Neutrophil engraftment is defined as achieving an absolute neutrophil count (ANC) of ≥500/ μL for 3 consecutive measurements on different days by Day 42 inclusive (the day of engraftment was defined as the first of these 3 days).
By Day 42

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of Transplant-related Mortality.
Time Frame: at 100 days
Transplant-related mortality is defined as death not preceded by autologous recovery.
at 100 days
Event-free Survival
Time Frame: 100 days post-transplant
Patients with event-free survival at 100 days post-transplant that did not have one of the following events: death, autologous recovery, primary or secondary graft failure.
100 days post-transplant
Overall Survival
Time Frame: 180 days
Overall survival at 180 days
180 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gamida Cell ltd

Investigators

  • Principal Investigator: Joanne Kurtzberg, MD, Duke University Medical Center, NC, USA
  • Principal Investigator: Joel Brochstein, MD, Steven & Alexandra Cohen Children's Medical Center, New York

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2012

Primary Completion (Actual)

September 1, 2018

Study Completion (Actual)

October 1, 2019

Study Registration Dates

First Submitted

April 15, 2012

First Submitted That Met QC Criteria

May 2, 2012

First Posted (Estimate)

May 3, 2012

Study Record Updates

Last Update Posted (Actual)

May 20, 2022

Last Update Submitted That Met QC Criteria

April 27, 2022

Last Verified

April 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GC P#02.01.020

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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