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Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil


N/A
N/A
N/A
Open (Enrolling)
Both
Immune System Disorder, Severe Combined Immunodeficiency

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Trial Information

Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil


OBJECTIVES:

I. To safely establish partial lymphoid chimerism (1-95% donor CD3+ cells) using a
non-lethal conditioning regimen in patients with Severe Combined Immunodeficiency Syndrome.

II. To define the kinetics of immune reconstitution following a non-lethal conditioning
regimen in patients with immunodeficiency diseases.

OUTLINE:

Patients receive cyclosporine orally (PO) or intravenously (IV) on days -3 to 100 followed
by a taper until day 180 and mycophenolate mofetil PO or IV on days 0-40 with a taper until
day 96 in the absence of unacceptable toxicity. Unrelated donor recipients also undergo TBI
on day 0. Patients undergo bone marrow transplant on day 0.

After completion of study treatment, patients are followed up at 6 months and then every
year for 5 years.


Inclusion Criteria:



- Patients with Severe Combined Immunodeficiency Syndrome:

- SCID with presence of B lymphocytes

- X-linked SCID (presence of B lymphocytes)

- Autosomal recessive SCID

- Patients with Severe Combined Immunodeficiency Syndrome:

- SCID with absence of T and B lymphocytes

- Patients with Severe Combined Immunodeficiency Syndrome:

- Purine metabolite deficiencies, deficiencies of the purine metabolites

- Adenosine deaminase (ADA) deficiency

- Purine nucleoside phosphorylase (PNP) deficiency

- DONOR: Related donor who is human leukocyte antigen (HLA) genotypically identical at
least at one haplotype and may be genotypically or phenotypically identical for
serological typing for HLA-A, B, C, and at the allele level for DRB1 and DQB1;
related donors other than siblings must be matched at HLA-A, B, and C (at highest
resolution available at the time of donor selection) and at DRB1 and DQB1 by DNA
typing; if more than one HLA-identical sibling is available, priority will be given
to the oldest normal donor

- DONOR: Unrelated donors who are prospectively matched for HLA-A, B, C, DRB1 and DQB1
by DNA typing at the highest resolution routinely available at the time of donor
selection; only a single allele disparity will be allowed for HLA-A, B, or C as
defined by high resolution typing

Exclusion Criteria:

- Patients with viral associated T cell immunodeficiency disorders, such as human
immunodeficiency virus (HIV)

- Patients with other disease or organ dysfunction that would limit survival to less
than 30 days

- DONOR: Identical twin

- DONOR: Pregnancy

- DONOR: HIV seropositive

- DONOR: A positive anti-donor cytotoxic cross match is absolute donor exclusion

- DONOR: Patient and donor pairs homozygous at a mismatched allele in the graft
rejection vector are considered a two-HLA allele mismatch, i.e., the patient is
A*0201, and this type of mismatch is not allowed

- DONOR: < 6 months old, > 75 years old

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Extent of chimerism in specific subpopulations by assessing T (CD3+3), B (CD19/20+), NK (CD56+), and myeloid (CD33+/13+) of bone marrow aspirate and peripheral blood

Outcome Time Frame:

Up to 5 years

Safety Issue:

No

Principal Investigator

Lauri Burroughs

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Authority:

United States: Food and Drug Administration

Study ID:

1227.00

NCT ID:

NCT00008450

Start Date:

August 1997

Completion Date:

Related Keywords:

  • Immune System Disorder
  • Severe Combined Immunodeficiency
  • Immunologic Deficiency Syndromes
  • Immune System Diseases
  • Severe Combined Immunodeficiency

Name

Location

Fred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumSeattle, Washington  98109