Clinical Trial: A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD

Brief Summary: This trial will assess 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 using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification with the Lenti-D lentiviral vector, the cells will be transplanted back into the subject following myeloablative conditioning.

Detailed Summary:
Sponsor: bluebird bio

Current Primary Outcome:

  • The proportion of subjects who are alive and have no Major Functional Disabilities (MFDs) as determined by key measures in the Neurological Function Score (NFS). [ Time Frame: 24 months (±1 months) post-transplant ]
  • The proportion of subjects who experience either acute (≥Grade II) or chronic GVHD at Month 24. [ Time Frame: 24 months (±1 months) post-transplant ]


Original Primary Outcome: Assessment of the proportion of subjects who have no Major Functional Disabilities (MFDs) as determined by key measures in the Neurological Function Score (NFS). [ Time Frame: 24 months (±2 months) post-transplant ]

Current Secondary Outcome:

  • Incidence of resolution of gadolinium positivity on MRI (i.e., GdE-). [ Time Frame: 24 months (±1 months) post-transplant ]
  • Time to resolution of gadolinium positivity on MRI (i.e., GdE-). [ Time Frame: 24 months (±1 months) post-transplant ]
  • Change in total NFS from Baseline. [ Time Frame: 24 months (±1 months) post-transplant ]
  • MFD-free survival over time [ Time Frame: 24 months (±1 months) post-transplant ]
  • Overall survival [ Time Frame: 24 months (±1 months) post-transplant ]


Original Secondary Outcome:

  • Change from Baseline in Loes score as measured by brain MRI. [ Time Frame: 24 mon (±2 months) post-transplant ]
  • Change from Baseline in NFS. [ Time Frame: 24 mon (± 2 months) post-transplant ]
  • Resolution of gadolinium positivity on MRI [ Time Frame: 24 mon (± 2 months) post-transplant ]
    Proportion of subjects who demonstrate resolution of gadolinium positivity on MRI (i.e., who are gadolinium negative) at 24 months (± 2 months) post-transplant.


Information By: bluebird bio

Dates:
Date Received: March 22, 2013
Date Started: August 2013
Date Completion: August 2019
Last Updated: March 20, 2017
Last Verified: March 2017