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Gene Therapy

Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

Phase 1
Waitlist Available
Led By Franklin O. Smith, MD
Research Sponsored by Boston Children's Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

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.

Eligible Conditions
  • Fanconi Anemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Single armExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Retrovirus Construct
2004
Completed Phase 1
~10

Find a Location

Who is running the clinical trial?

Boston Children's HospitalLead Sponsor
789 Previous Clinical Trials
5,582,903 Total Patients Enrolled
4 Trials studying Fanconi Anemia
1,781 Patients Enrolled for Fanconi Anemia
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,944 Previous Clinical Trials
47,799,829 Total Patients Enrolled
10 Trials studying Fanconi Anemia
334 Patients Enrolled for Fanconi Anemia
Children's Hospital Medical Center, CincinnatiOTHER
839 Previous Clinical Trials
6,565,600 Total Patients Enrolled
9 Trials studying Fanconi Anemia
250 Patients Enrolled for Fanconi Anemia
Franklin O. Smith, MDPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati
~0 spots leftby Jan 2026
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