drugset / Trial / NCT01331018
Gene Therapy for Fanconi Anemia
Phase 1
Terminated
3 enrolled
Fred Hutchinson Cancer Center
National Heart, Lung, and Blood Institute (NHLBI) · collabRocket Pharma Limited · collab
NaSingle-groupOpen-labelTreatment
Summary
This clinical trial will access the toxicity and efficacy of infusion of gene modified cells for patients with Fanconi anemia (FA). Infusion of autologous patient blood stem cells that have been corrected in the laboratory by introduction of the normal gene may improve blood counts in patients with FA.
Timeline
- Start
- 2012-02-22
- Primary completion
- 2024-02-15
- Completion
- 2024-02-15
Outcome
Outcome not reported
Stopped (Business): “Study ended early due to end of funding.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Genetically Engineered Hematopoietic Stem Progenitor Cells | Cell therapy | — | — |
| Background | Methylprednisolone | Other / unclassified | — | Intravenous |
| Background | Plerixafor | Small molecule | — | Subcutaneous |
| Background | Prednisone | Other / unclassified | — | Oral |
| Background | filgrastim | Protein / enzyme biologic | — | Subcutaneous |