# Gene Therapy for Fanconi Anemia

> **NCT01331018** · PHASE1 · TERMINATED · sponsor: **Fred Hutchinson Cancer Center** · enrollment: 3 (actual)

## Conditions studied

- Fanconi Anemia

## Interventions

- **PROCEDURE:** Bone Marrow Aspiration
- **BIOLOGICAL:** Filgrastim
- **BIOLOGICAL:** Genetically Engineered Hematopoietic Stem Progenitor Cells
- **OTHER:** Laboratory Biomarker Analysis
- **PROCEDURE:** Leukapheresis
- **DRUG:** Methylprednisolone
- **DRUG:** Plerixafor
- **DRUG:** Prednisone

## Key facts

- **NCT ID:** NCT01331018
- **Lead sponsor:** Fred Hutchinson Cancer Center
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2012-02-22
- **Primary completion:** 2024-02-15
- **Final completion:** 2024-02-15
- **Target enrollment:** 3 (ACTUAL)
- **Why stopped:** Study ended early due to end of funding.
- **Last updated:** 2024-05-17

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01331018

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01331018, "Gene Therapy for Fanconi Anemia". Retrieved via AI Analytics 2026-06-14 from https://api.ai-analytics.org/clinical/NCT01331018. Licensed CC0.

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