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Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies


Phase 1/Phase 2
8 Years
65 Years
Open (Enrolling)
Both
Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Non-Hodgkin's Lymphoma, Hodgkin's Disease

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Trial Information

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies


Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative
procedure for various hematological malignancies, bone marrow failure syndromes and
inherited metabolic disorders. The application of allogeneic HSCT is limited by donor
availability such that only approximately one-third of the otherwise appropriate candidates
have suitably matched family donors. Alternative donors include mismatched family members or
matched unrelated donors, but these approaches are often complicated by an increased risk of
graft-versus-host disease (GvHD) and a prolonged and cumbersome search and procurement
process. In addition, far fewer subjects of racial minorities find suitable human leukocyte
antigen (HLA)-matched donors.

Umbilical cord blood has been increasingly used as an alternative source of stem cells and
has extended the availability of allogeneic HSCT to patients who would otherwise not be
eligible for this curative approach. In the last decade the number of cord blood
transplantations from related and unrelated donors has increased dramatically. It is
estimated that more than 20,000 patients have undergone cord blood transplantation from
unrelated donors to date for a variety of genetic, hematological, immunological, metabolic
and oncologic disorders. The major advantages of cord blood transplantation include easy
procurement, no risk to donors, reduced incidence of transmitting infections, immediate
availability, and reduced risk of acute GvHD in the setting of donor-recipient HLA mismatch.
Nevertheless, the low cell dose remains a main limitation of this cell source leading to
delayed hematopoietic reconstitution, higher risk of graft failure and relatively high
treatment related mortality rates as compared to other hematopoeitic cell sources. To
improve outcomes and extend applicability of cord blood transplantation, one potential
solution is ex vivo expansion of cord blood-derived stem and progenitor cells.

The Sponsor has undertaken to develop NiCord®, which is based on a novel technology for 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 broader application of umbilical cord blood transplantation and
improve clinical outcomes in subjects with high-risk hematological malignancies.

The main objective of the current study is to evaluate the safety of co-transplantation of
NiCord® and an unmanipulated cord blood unit in patients with hematological malignancies
following myeloablative therapy.


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 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

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety and Tolerability: will be measured by acute NiCord® infusional toxicity, and assessment of the proportion of patients with neutrophil engraftment

Outcome Time Frame:

180 days post-transplant

Safety Issue:

Yes

Principal Investigator

David Snyder, PhD

Investigator Role:

Study Director

Investigator Affiliation:

Gamida Cell ltd

Authority:

United States: Food and Drug Administration

Study ID:

GC P#01.01.020

NCT ID:

NCT01221857

Start Date:

November 2010

Completion Date:

March 2013

Related Keywords:

  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myelogenous Leukemia (AML)
  • Myelodysplastic Syndrome (MDS)
  • Non-Hodgkin's Lymphoma
  • Hodgkin's Disease
  • Double Umbilical Cord Blood Stem Cell Transplantation
  • Hematological Malignancies
  • HLA Mismatched Donors
  • Cord Blood Transplantation
  • Neoplasms
  • Hodgkin Disease
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Lymphoma
  • Lymphoma, Non-Hodgkin
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms

Name

Location

Duke University Medical CenterDurham, North Carolina  27710
Loyola University, Cardinal Bernardin Cancer CenterMaywood, Illinois  60153