NCT00317876

Brief Summary

RATIONALE: Giving low doses of chemotherapy, such as cyclophosphamide, before a donor bone marrow transplant helps stop the growth of abnormal cells. It also stops the patient's immune system from rejecting the donor's bone marrow. The donated bone marrow stem cells may replace the patient's immune system and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and methotrexate before or after transplant may stop this from happening. PURPOSE: This phase I trial is studying the side effects and best dose of cyclophosphamide in treating patients who are undergoing a donor bone marrow transplant for Fanconi's anemia.

Trial Health

85
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at P25-P50 for phase_1

Geographic Reach
2 countries

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 1998

Completed
5.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2003

Completed
2.8 years until next milestone

First Submitted

Initial submission to the registry

April 24, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 25, 2006

Completed
Last Updated

April 20, 2012

Status Verified

April 1, 2012

Enrollment Period

5.1 years

First QC Date

April 24, 2006

Last Update Submit

April 18, 2012

Conditions

Fanconi Anemia

Keywords

Fanconi anemia

Outcome Measures

Primary Outcomes (2)

  • Conditioning-related toxicity

    100 days post-transplant

  • Graft rejection

    100 days post-transplant

Interventions

cyclophosphamideDRUG
cyclosporineDRUG
methotrexateDRUG
allogeneic bone marrow transplantationPROCEDURE
nonmyeloablative allogeneic hematopoietic stem cell transplantationPROCEDURE

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
DISEASE CHARACTERISTICS: * Diagnosis of Fanconi's anemia by chromosome fragility with a diepoxybutane (DEB) or mitomycin C test * Hemoglobin ≤ 8.0 g/dL, absolute granulocyte count ≤ 1,000/mm\^3, or platelet count ≤ 50,000/mm\^3 * No refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute leukemia * HLA-identical related donor available PATIENT CHARACTERISTICS: * Glomerular filtration rate ≥ 30% predicted for age * No liver disease (e.g., active hepatitis or moderate to severe portal fibrosis/cirrhosis by biopsy) * No symptomatic cardiac insufficiency or symptomatic arrhythmia * No other diseases that would severely limit the probability of survival * No HIV seropositivity * Not pregnant or nursing * Fertile patients must use effective contraception PRIOR CONCURRENT THERAPY: * Not specified

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (3)

Seattle Cancer Care Alliance

Seattle, Washington, 98109-1023, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109-1024, United States

Location

Universidade Federal do Parana

Curitiba, Paraná, 80.060-000, Brazil

Location

MeSH Terms

Conditions

Fanconi Anemia

Interventions

CyclophosphamideCyclosporineMethotrexate

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsCyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and ProteinsAminopterinPterinsPteridinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds

Study Officials

  • Hans-Peter Kiem, MD

    Fred Hutchinson Cancer Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Masking
NONE
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

April 24, 2006

First Posted

April 25, 2006

Study Start

June 1, 1998

Primary Completion

July 1, 2003

Last Updated

April 20, 2012

Record last verified: 2012-04

Locations

US(2)BR(1)