Know Cancer

or
forgot password

Multicenter Phase II Trial-Hematopoietic Stem Cell Transplantation for Treatment of Patients With FA Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine


Phase 2
N/A
N/A
Not Enrolling
Both
Fanconi Anemia

Thank you

Trial Information

Multicenter Phase II Trial-Hematopoietic Stem Cell Transplantation for Treatment of Patients With FA Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine


Inclusion Criteria:



- Diagnosis: Patients must have a diagnosis of Fanconi anemia (confirmed by mitomycin C
or diepoxybutane [DEB] chromosomal breakage testing at an approved laboratory)

- Hematologic Diagnosis and Status: Patients must have one of the following hematologic
diagnoses:

1. Severe Aplastic Anemia (SAA), or Severe Isolated Single lineage

Cytopenia with bone marrow cellularity of < 25% AND at least one of the
following features:

- Platelet count < 20 x 109/L or platelet transfusion dependence

- ANC < 1000 x 109/L

- Hgb < 8 gm/dl or red cell transfusion dependence

2. Myelodysplatic Syndrome (MDS) (Appendix 1: MDS Classification). MDS at any
stage, based on either one of the following classifications:

- WHO Classification

- Refractory anemia and transfusion dependence

- Any of other stages

- IPSS Classification

- Low risk (score 0) and transfusion dependence

- Any other risk groups Score > 0.5

3. Acute Myelogenous Leukemia: Patients with acute leukemia are included in this
trial in remission, refractory or relapsed disease

Exclusion Criteria:

- Active CNS leukemic involvement

- Female patients who are pregnant or breast-feeding

- Active uncontrolled viral, bacterial or fungal infection

- Patient seropositive for HIV-I/II; HTLV -I/II

Type of Study:

Interventional

Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

the incidence and quality of engraftment and hematopoietic reconstitution, the incidence of early transplant related mortality, the incidence and severity of acute GvHD and chronic GVHD.

Outcome Time Frame:

2 Years

Safety Issue:

Yes

Principal Investigator

David A Williams, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Hospital Boston

Authority:

United States: Food and Drug Administration

Study ID:

08-377

NCT ID:

NCT00850317

Start Date:

April 2009

Completion Date:

Related Keywords:

  • Fanconi Anemia
  • Fanconi Anemia
  • FA
  • Stem Cell Transplant
  • T-Cell
  • Anemia
  • Fanconi Anemia
  • Fanconi Syndrome

Name

Location

Dana-Farber Cancer InstituteBoston, Massachusetts  02115