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

The recruitment status of this study is unknown because the information has not been verified recently.

Verified August 2006 by Office of Rare Diseases (ORD).
Recruitment status was Active, not recruiting

Sponsor:

Information provided by:

Office of Rare Diseases (ORD)

ClinicalTrials.gov Identifier:

NCT00010361

First received: February 2, 2001

Last updated: August 23, 2006

Last verified: August 2006

History of Changes

Tracking Information

First Received Date ^ICMJE

February 2, 2001

Last Updated Date

August 23, 2006

Start Date ^ICMJE

November 2000

Primary Completion Date

Not Provided

Current Primary Outcome Measures ^ICMJE

Not Provided

Original Primary Outcome Measures ^ICMJE

Not Provided

Change History

Complete list of historical versions of study NCT00010361 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Not Provided

Original Secondary Outcome Measures ^ICMJE

Not Provided

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

Not Provided

Brief 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.

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.

Study Type ^ICMJE

Interventional

Study Phase

Not Provided

Study Design ^ICMJE

Primary Purpose: Treatment

Condition ^ICMJE

Metabolism, Inborn Errors
Granulomatous Disease, Chronic

Intervention ^ICMJE

Drug: cyclosporine
Drug: fludarabine
Drug: mycophenolate mofetil

Study Arm (s)

Not Provided

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Active, not recruiting

Enrollment ^ICMJE

Completion Date

Not Provided

Primary Completion Date

Not Provided

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

up to 55 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT Number ^ICMJE

NCT00010361

Other Study ID Numbers ^ICMJE

199/15577, FHCRC-1475.00

Has Data Monitoring Committee

Not Provided

Responsible Party

Not Provided

Study Sponsor ^ICMJE

Fred Hutchinson Cancer Research Center

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Chair:

Ann Woolfrey

Fred Hutchinson Cancer Research Center

Information Provided By

Office of Rare Diseases (ORD)

Verification Date

August 2006

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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