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Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia

NCT00005896 · Status: UNKNOWN · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2005-06-24

No results posted yet for this study

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.

Conditions

  • Fanconi's Anemia

Interventions

DRUG

filgrastim

GENETIC

Autologous stem cells transduced with FACC retroviral vector

PROCEDURE

Autologous Stem Cell Transplantation

Sponsors & Collaborators

Principal Investigators

  • John E. Wagner, Jr. · Masonic Cancer Center, University of Minnesota

Study Design

Purpose
TREATMENT

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2000-03-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00005896 on ClinicalTrials.gov