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A Prospective Study of Optimal Cord Selection for Haplo-Cord Transplantation: Targeting the Inherited Paternal Antigen (IPA) and Matching for the Non-Inherited Maternal Antigen (NIMA)

18 Years
Open (Enrolling)
Haplo-Cord Transplantation

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Trial Information

A Prospective Study of Optimal Cord Selection for Haplo-Cord Transplantation: Targeting the Inherited Paternal Antigen (IPA) and Matching for the Non-Inherited Maternal Antigen (NIMA)

This is a clinical trial for subjects with hematologic malignancies ( acute leukemia,
myeloproliferative disorders, lymphoma, myeloma) who are in need of a donor stem cell
transplant, and for whom an umbilical cord blood transplant is thought to be the best
option. As donors for allogeneic transplant, we typically try to use related family members,
such as brothers or sisters, or volunteer donors who are 'HLA matched', i.e. share similar
proteins on their cells. This study is for subjects for whom such a matched sibling donor or
a matched unrelated donor is not available.

For such subjects a commonly used transplant procedure is to use stem cells from one or two
umbilical cords (UCB) from a newborn. These umbilical cord blood grafts, despite not
completely matching the recipient, cause few problems with graft vs host disease (a common
complication of transplant). But they tend to grow very slowly and subjects often have very
prolonged hospital stays and are at high risk for complications due to low blood counts.
Umbilical cord blood transplant will be the standard arm for this protocol.

This study uses a new method of bone marrow transplantation called combined haplo-identical
cord (haplo-cord) transplantation. In this procedure, cells from a related donor who shares
half of the HLA proteins ( haplo-identical) are collected from the blood, as well as cells
from an umbilical cord, and then both are transplanted. It is hoped that by using cells from
a haplo-identical relative, subjects will have a faster recovery and require fewer
transfusions. Over time the haplo-identical cells from the relative are replaced by the
cells from the cord blood. The combined transplantation of haplo-identical stem cells and
cord blood has previously been used in approximately 60 subjects with very encouraging

Traditionally it has been felt that the most important determinant of outcome of an UCB stem
cell transplant is the cord blood cell dose. The second determinant is the degree of
matching between donor and recipient. Many times, we have difficulty identifying UCB units
of sufficient cell dose that are well matched. Of interest,in our prior study of
haplo-cord SCT indicated outcomes seemed independent of the UCB cell dose. If this
preliminary observation is correct, we may be able to improve the outcomes of haplo cord
transplant further by accepting lower threshold UCB doses and rather focusing on optimal
matching (including matching for HLA and another characteristic called IPA). This is the
primary objective of this study.

Inclusion Criteria:

- Subject must have a confirmed diagnosis of:

1. Previously Relapsed or refractory acute leukemia (myeloid or lymphoid)

2. Acute leukemia in first remission at high-risk for recurrence

3. Chronic myelogenous leukemia in chronic, accelerated phase or blast-crisis

4. Recurrent or refractory malignant lymphoma or Hodgkin lymphoma

5. Chronic lymphocytic leukemia, relapsed or with poor prognostic features

6. Multiple myeloma

7. Myelodysplastic syndrome

8. Chronic myeloproliferative disease

9. Hemoglobinopathies

10. Aplastic anemia

- Age ≥ 18 years

- Likely to benefit from allogeneic transplant in the opinion of the transplant

- An HLA-identical related or unrelated donor cannot be identified within an
appropriate time frame.

- Karnofsky (KPS) Performance status of > 80

- Acceptable organ function as defined below: Serum bilirubin: < 2.0mg/dL ALT(SGPT): <
3 X upper limit of normal Creatinine Clearance: > 50 mL/min/1.73m2 (as estimated by
the modified MDRD equation)

- Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

- Life expectancy is severely limited by concomitant illness or uncontrolled infection

- Severely decreased Left Ventricular Ejection Fraction (LVEF) or impaired pulmonary
function tests (PFT's)

- Evidence of chronic active hepatitis or cirrhosis

- HIV-positive

- Pregnant or lactating

Type of Study:


Study Design:

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

Outcome Measure:

Lowest threshold of umbilical cord nucleated cell dose for haplo-cord transplants.

Outcome Description:

We aim to identify the lowest threshold of umbilical cord nucleated cell dose that can be utilized assure durable umbilical cord blood engraftment in the haplo-cord transplants. The threshold will be defined as the lowest dose which assures cord blood engraftment occurs in at least 80% of subjects.

Outcome Time Frame:

100 days

Safety Issue:


Principal Investigator

Koen van Besien, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Weill Medical College of Cornell University


United States: Food and Drug Administration

Study ID:




Start Date:

August 2012

Completion Date:

October 2016

Related Keywords:

  • Haplo-Cord Transplantation
  • Allogeneic transplant
  • Hematologic Malignancies
  • Acute Leukemia
  • Myeloproliferative disorders
  • Lymphoma
  • Myeloma



Weill Cornell Medical College New York, New York  10021