Overview

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

Status:
Recruiting

Trial end date:
2023-02-16

Target enrollment:

Participant gender:

Summary

Study ALD-104 is an international, non-randomized, open-label, multi-site study in male subjects with CALD (≤17 years of age at enrollment). 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 cerebral adrenoleukodystrophy (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.

Phase:

Phase 3

Details

Lead Sponsor:

bluebird bio

Treatments:

Fludarabine