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Phase 3 study of eli-cel after busulfan/fludarabine conditioning in pediatric cerebral adrenoleukodystrophy
NL-OMON54719Phase 3Completed
- Sponsor
- bluebird bio, Inc.
- Start
- Jan 2019
- Modality
- Cell Therapies, Gene Therapies
- Countries
- 1
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Overview
Phase 3, open-label, single-arm trial of elivaldogene autotemcel in cerebral adrenoleukodystrophy (CALD), sponsored by bluebird bio, Inc. Primary endpoint: Alive and free of major functional disabilities at Month 24. Started Jan 2019; status: completed.
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Drugs
elivaldogene autotemcel
03
Conditions
cerebral adrenoleukodystrophy (CALD)
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