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


Phase 1/Phase 2
18 Years
65 Years
Open (Enrolling)
Both
Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)

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Trial Information

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


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 18-65 years of age, up to a maximum
of 15 treated patients.


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

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Engraftment

Outcome Description:

Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.

Outcome Time Frame:

42 days

Safety Issue:

No

Principal Investigator

Mitchell Horwitz, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Duke University

Authority:

United States: Food and Drug Administration

Study ID:

GC P#03.01.020

NCT ID:

NCT01816230

Start Date:

April 2013

Completion Date:

Related Keywords:

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

Name

Location

Duke University Medical CenterDurham, North Carolina  27710