Last updated on August 2019

Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)


Brief description of study

Study ALD-104 is an international, non-randomized, open-label, multi-site study in male subjects (17 years of age at enrollment) with cerebral adrenoleukodystrophy (CALD). Approximately 20 subjects will be infused with Lenti-D Drug Product after myeloablative conditioning with busulfan and fludarabine.

This trial will evaluate the efficacy and safety of autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of CALD. A subject's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the subject following myeloablative conditioning.

Clinical Study Identifier: NCT03852498

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H pital Robert Debr

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