MedDataX Logo

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

NCT ID: NCT00010361

Last Updated: 2014-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2000-11-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Metabolism, Inborn Errors Granulomatous Disease, Chronic

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Primary Study Purpose

TREATMENT

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

cyclosporine

Intervention Type DRUG

fludarabine

Intervention Type DRUG

mycophenolate mofetil

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion 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
Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Fred Hutchinson Cancer Center

OTHER

Sponsor Role lead

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Ann Woolfrey

Role: STUDY_CHAIR

Fred Hutchinson Cancer Center

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

FHCRC-1475.00

Identifier Type: -

Identifier Source: secondary_id

199/15577

Identifier Type: -

Identifier Source: org_study_id

NCT00144742

Identifier Type: -

Identifier Source: nct_alias