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A Phase II Study of Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors for Treatment of Hematologic Malignancies and Hematopoietic Failure States


Phase 2
18 Years
75 Years
Open (Enrolling)
Both
Hematologic Neoplasms, Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Multiple Myeloma

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Trial Information

A Phase II Study of Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors for Treatment of Hematologic Malignancies and Hematopoietic Failure States


Fewer than 35% of patients who might benefit from allogeneic HCT have an HLA-identical sib.
Transplantation of peripheral blood stem cells (PBSCs) or bone marrow (BM)from HLA-matched
or one-locus mismatch unrelated volunteer donors may be an alternative in some patients who
lack HLA-matched sib donors. Despite increasing numbers of volunteer unrelated donors in
national and international registries, identification of suitable unrelated donors who are
matched with the recipient at all HLA-A, -B, -C and -DRB1 loci (8/8 HLA match) or mismatched
at one of those loci (7/8 HLA match) is still challenging, especially for patients who are
African-American or multiracial. Additionally, the 3- to 4-month delay between initiation
of unrelated donor search to HCT is unacceptably long in patients with aggressive
hematologic malignancies that are likely to relapse or progress during that interval.
Transplantation of single or dual unrelated umbilical cord blood cells (UCB) units is
another alternative, although problems with inadequate cell doses, delayed engraftment,
graft rejection and infection persist in adult recipients of unrelated UCB transplants.

This is a phase II single-arm open-label study to evaluate the efficacy and safety of
haploidentical related allogeneic PBSCT using a nonmyeloablative preparative regimen of
intravenous busulfan (Busulfex®), intravenous melphalan (Alkeran®) and intravenous
alemtuzumab (Campath®) in subjects who are candidates for related or unrelated allogeneic
hematopoietic cell transplantation (HCT; transplantation of bone marrow or PBSCs) but who
lack histocompatible related or unrelated donors. This study will also evaluate
immunological reconstitution following haploidentical PBSCT by measurement of circulating T
cell receptor excision circle (TREC)-positive cells, an indicator of thymic output.
Systematic analyses of TREC-positive cells have not been performed in recipients of
haploidentical PBSCT after the preparative regimen described in this protocol.


Inclusion Criteria:



- Age between 18 years and 75 years.

- one of these diagnoses: acute myeloid leukemia in remission or relapse, acute
lymphocytic leukemia in remission or relapse, chronic myeloid leukemia, chronic
lymphocytic leukemia, Hodgkin's disease or non-Hodgkin's lymphoma, multiple myeloma,
myelodysplastic syndrome, severe aplastic anemia, or paroxysmal nocturnal
hemoglobinuria.

- Subjects with hematologic malignancies must have received at least one previous
course of chemotherapy or biological therapy (i.e., a subject cannot be enrolled on
this study for initial treatment of the malignancy).

- Absence of a healthy related or unrelated volunteer allogeneic donor with whom the
subject is either completely HLA matched at HLA-A, -B, -C and -DRB1 (8/8 HLA match)
or mismatched at no more than one HLA locus (7/8 HLA match).

- Availability of a healthy haploidentical relative (parent, sib or child) who is able
to donate peripheral blood stem cells by apheresis.

Exclusion Criteria:

- Eligibility for another clinical therapeutic protocol or standard-of-care treatment
that offers higher probability of cure or long-term control of subject's malignancy.

- Availability of a related or unrelated 7/8 or 8/8 HLA-matched allogeneic donor.

- Severe organ dysfunction

- Untreated or progressive central nervous system involvement by malignancy.

- Subject is pregnant or breast feeding.

- Karnofsky score below 50.

- Seropositivity for human immunodeficiency virus (HIV).

- Life expectancy less than 12 weeks with conventional treatments.

- For subjects who are fertile, refusal to practice contraception upon entering this
study and for at least 12 months after PBSCT or after cessation of post-transplant
immunosuppressive treatments, whichever occurs later.

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood)in peripheral blood by day +100.

Outcome Time Frame:

by day +100 (i.e., 100 days after haploidentical PBSCT).

Safety Issue:

Yes

Principal Investigator

Andrew M Yeager, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Arizona

Authority:

United States: Institutional Review Board

Study ID:

BIO 07-122

NCT ID:

NCT00618969

Start Date:

February 2008

Completion Date:

February 2014

Related Keywords:

  • Hematologic Neoplasms
  • Anemia, Aplastic
  • Hemoglobinuria, Paroxysmal
  • Multiple Myeloma
  • Haploidentical
  • peripheral blood stem cell transplantation
  • leukemia
  • myeloma
  • Hodgkin's disease
  • non-Hodgkin's lymphoma
  • Anemia
  • Anemia, Aplastic
  • Neoplasms
  • Hemoglobinuria
  • Hemoglobinuria, Paroxysmal
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Hematologic Neoplasms

Name

Location
Arizona Cancer Center/University Medical Center Tucson, Arizona  85719