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CompletedNCT00010361Updated Dec 9, 2014

Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders

An interventional study of cyclosporine and fludarabine in Metabolism, Inborn Errors and Granulomatous Disease, Chronic, sponsored by Fred Hutchinson Cancer Center. Completed at 1 site in United States. Open to participants aged Up to 55 Years. Per ClinicalTrials.gov, last updated 2014-12-09.

Sponsored by Fred Hutchinson Cancer Center · Not applicable, Interventional, and Treatment

Phase
Not applicable
Study type
Interventional
Enrollment
20
Ages
Up to 55 Years
Sex
All
01

Study summary

OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclosporine and mycophenolate mofetil for establishing mixed chimerism in patients with inherited disorders.

II. Determine whether this regimen can establish mixed chimerism in these patients.

III. Determine whether mixed chimerism is sufficient to reverse disease symptoms in these patients.

IV. Determine the safety of donor lymphocyte infusions to eliminate persistent disease in these patients with mixed chimerism.

Read the detailed description

PROTOCOL OUTLINE: Patients receive fludarabine IV over 2 hours on days -4 to -2 followed by total body irradiation and peripheral blood stem cell or bone marrow transplantation on day 0. Patients also receive oral or IV cyclosporine 2-3 times daily on days -3 to 50 (related donor) or 100 (unrelated donor) and oral mycophenolate mofetil twice daily on days 0 to 28 (related donor) or 40 (unrelated donor).

Patients may also receive donor lymphocyte infusion for continued treatment of symptoms in the event of mixed chimerism and in the absence of graft-versus-host disease.

Patients are followed weekly for 1 month, monthly for 2 years, and then annually thereafter.

02

Conditions studied

  • Metabolism, Inborn Errors
  • Granulomatous Disease, Chronic
03

In context

Granulomatous Disease, Chronic

77 studies on the registry are indexed under Granulomatous Disease, Chronic; 17 are open to participants now.

This study's enrollment of 20 is above the median of 12 across 53 interventional studies indexed under Granulomatous Disease, Chronic.

Browse Granulomatous Disease, Chronic studies →

Lead sponsor

Fred Hutchinson Cancer Center is the lead sponsor of 537 studies on the registry; 79 are open to participants now.

Of its 57 completed or terminated interventional studies of FDA-regulated products, 45 (79%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 55 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

  • Inherited disorders treatable with allogeneic peripheral blood or bone marrow transplantation At high risk for regimen related toxicity with a conventional transplant
  • No severe CNS involvement of disease, defined by IQ score less than 70
  • HLA matched donor Sibling donors must be a confirmed match at HLA-A, B, and DRB1 Other related and non-related donors must be matched at HLA-A, B, C, DRB1, and DQB1 A donor homozygous for one allele only at HLA-A, B, C, DRB1, or DQB1 allowed (1 antigen mismatch for graft-versus-host disease, 0 antigen mismatch for graft-rejection)

--Prior/Concurrent Therapy--

  • No concurrent growth factors with mycophenolate mofetil

--Patient Characteristics--

  • Age: Under 55
  • Performance status: Not specified
  • Life expectancy: At least 100 days
  • Hematopoietic: Not specified
  • Hepatic: No evidence of synthetic dysfunction No severe cirrhosis
  • Renal: Not specified
  • Cardiovascular: LVEF at least 30% No poorly controlled hypertension on multiple antihypertensives
  • Other: No organ dysfunction that would preclude survival Not pregnant or nursing Fertile patients must use effective contraception during and for 12 months following study
05

Study design

Phase
Not applicable
Primary purpose
Treatment
Enrollment
20 participants

Interventions

  • Drugcyclosporine
  • Drugfludarabine
  • Drugmycophenolate mofetil
06

Study locations

1 site
  • Fred Hutchinson Cancer Research Center
    Seattle, Washington 98109, United States
07

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Dec 9, 2014, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
08

Registry details

Key details

Study ID
NCT00010361
Lead sponsor
Fred Hutchinson Cancer Center
First posted
Feb 2, 2001
Start date
Nov 2000
Primary completion
Apr 2007
Last update
Dec 9, 2014

Study contacts

Ann Woolfrey
study chair · Fred Hutchinson Cancer Center
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2006. You cannot join it, but the record below documents what was studied.

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