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Clinical Trial Summary

OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C.

II. Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients.

III. Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients.


Clinical Trial Description

PROTOCOL OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily on days 0-6 followed by apheresis to collect peripheral blood stem cells (PBSC) on days 5-7. PBSCs are processed in vitro for enrichment of CD34 cells and transduced with a Fanconi's anemia complementation C (FACC) retroviral vector on days 5-10. Patients receive transduced PBSCs IV over no more than 2 hours on days 8-10. PBSC infusions may be repeated no more than every 2 months for up to 4 courses total.

Patients are followed monthly for 3 months, every 3 months for 9 months, every 6 months for the next year, and then yearly thereafter. ;


Study Design

Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT00005896
Study type Interventional
Source Office of Rare Diseases (ORD)
Contact
Status Active, not recruiting
Phase Phase 1
Start date March 2000

See also
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Completed NCT00005898 - Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia Phase 1/Phase 2
Completed NCT00001399 - Gene Therapy for the Treatment of Fanconi's Anemia Type C Phase 1
Completed NCT00004787 - Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes Phase 2
Active, not recruiting NCT00006127 - Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia Phase 1