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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 12 December 2020
Main ID:  NCT00008450
Date of registration: 06/01/2001
Prospective Registration: No
Primary sponsor: Fred Hutchinson Cancer Research Center
Public title: Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant
Scientific title: 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
Date of first enrolment: August 11, 1997
Target sample size: 6
Recruitment status: Completed
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1
Countries of recruitment
United States
Name:     Lauri Burroughs
Affiliation:  Fred Hutch/University of Washington Cancer Consortium
Key inclusion & exclusion criteria

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 deoxyribonucleic
acid (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

Age minimum: N/A
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Adenosine Deaminase Deficiency
Autosomal Recessive Disorder
Immune System Disorder
Purine-Nucleoside Phosphorylase Deficiency
Severe Combined Immunodeficiency
Severe Combined Immunodeficiency With Absence of T and B Cells
X-Linked Severe Combined Immunodeficiency
Drug: Cyclosporine
Drug: Mycophenolate Mofetil
Other: Laboratory Biomarker Analysis
Procedure: Allogeneic Bone Marrow Transplantation
Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation
Radiation: Total-Body Irradiation
Primary Outcome(s)
Mixed hematopoietic chimerism in a population of pediatric patients with immunodeficiency diseases [Time Frame: Up to 5 years]
Secondary Outcome(s)
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
National Cancer Institute (NCI)
National Heart, Lung, and Blood Institute (NHLBI)
Ethics review
Results available:
Date Posted:
Date Completed:
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