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Gene Therapy

Single arm for 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 3months, 6 months and yearly up to 15 years post gene transfer
Awards & highlights

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.

Eligible Conditions
  • Fanconi Anemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3months, 6 months and yearly up to 15 years post gene transfer
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3months, 6 months and yearly up to 15 years post gene transfer for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety of gene transfer methods
Short-term and long-term engraftment of gene-corrected autologous hematopoietic cells (all measured at Year 1)
Secondary outcome measures
Development of myelodysplastic syndrome or overt leukemia (all measured at Year 1)
Efficiency of engraftment of multilineage gene corrected clones
Frequency and function of the integrated recombinant Fanconi vector
+1 more

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
766 Previous Clinical Trials
5,580,786 Total Patients Enrolled
4 Trials studying Fanconi Anemia
1,781 Patients Enrolled for Fanconi Anemia
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,847 Previous Clinical Trials
47,818,247 Total Patients Enrolled
10 Trials studying Fanconi Anemia
334 Patients Enrolled for Fanconi Anemia
Children's Hospital Medical Center, CincinnatiOTHER
815 Previous Clinical Trials
6,530,733 Total Patients Enrolled
9 Trials studying Fanconi Anemia
257 Patients Enrolled for Fanconi Anemia

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia
David Williams 2004. "Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT00272857.
~0 spots leftby May 2025
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