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Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

NCT00272857 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2017-06-23

No results posted yet for this study

Summary

Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the bone marrow cells that produce various types of blood cells. Individuals with FA may experience fatigue, bleeding, and increased infections. The purpose of this study is to evaluate the safety and effectiveness of a gene transfer procedure in generating new, healthy cells in individuals with FA.

Conditions

  • Fanconi Anemia

Interventions

GENETIC

Retrovirus Construct

This study will use an experimental gene transfer procedure performed in a laboratory to insert a new FA gene into the participant's bone marrow cells. The gene-corrected bone marrow cells will then be re-infused into the participant and participants will be observed for successful gene transfer

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Children's Hospital Medical Center, Cincinnati

    collaborator OTHER

  • Boston Children's Hospital

    lead OTHER

Principal Investigators

  • Franklin O. Smith, MD · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-08-31
Primary Completion
2007-10-31
Completion
2007-10-31

Countries

  • United States

Study Locations

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Entities

Companies

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 NCT00272857 on ClinicalTrials.gov