Fanconi Anemia Transplant Lacking Genotypically Identical Donor

Fanconi Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT00850317
• Other study ID #: 08-377
• Status: Not yet recruiting
• Phase: Phase 2
• First received: February 24, 2009
• Last updated: February 24, 2009
• Start date: April 2009

Study information

• Verified date: February 2009
• Source: Children's Hospital Boston
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

A research study for patients with Fanconi Anemia whose bone marrow has changed and now failed, giving rise to a pre-leukemia or leukemia. This study is a Phase II clinical trial in which patients will undergo allogenic transplant of stem cells, meaning they will receive bone marrow cells from a healthy donor. The purpose of this study is to see if transplant course of treatment will lower the risk of graft vs. host disease.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 25
• Est. primary completion date: April 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Syndrome

Intervention

Device:
• Miltenyi CliniMACs device
CD34 selection

Locations

Country	Name	City	State
United States	Dana-Farber Cancer Institute	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Children's Hospital Boston	Dana-Farber Cancer Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		2 Years	Yes
Secondary	The occurrence of severe post transplant regimen-related severe morbidity (grade III/IV toxicity) and/or mortality		2 years	Yes