Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant

• Register: ClinicalTrials.gov
• 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
• URL: https://clinicaltrials.gov/show/NCT00008450
• 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

Contacts

• 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

Severe Combined Immunodeficiency With Absence of T and B Cells

Autosomal Recessive Disorder

Immune System Disorder

Adenosine Deaminase Deficiency

Severe Combined Immunodeficiency

Purine-Nucleoside Phosphorylase Deficiency

X-Linked Severe Combined Immunodeficiency

Intervention(s)

Drug: Cyclosporine

Other: Laboratory Biomarker Analysis

Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation

Drug: Mycophenolate Mofetil

Radiation: Total-Body Irradiation

Procedure: Allogeneic Bone Marrow Transplantation

Primary Outcome(s)

Mixed hematopoietic chimerism in a population of pediatric patients with immunodeficiency diseases [Time Frame: Up to 5 years]

Secondary ID(s)

P01HL036444

P30CA015704

NCI-2010-02045

1227.00

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Heart, Lung, and Blood Institute (NHLBI)

National Cancer Institute (NCI)