NCT01896102

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

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)

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: Male
• Age: 17 Years
• Healthy volunteers: Not accepted

Summary

This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were 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 were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.

Detailed description

For study ALD-102 the Transplant Population (TP), Neutrophil Engraftment Population (NEP), and Intent-to-Treat Population (ITT) were identical.

Conditions

• Cerebral Adrenoleukodystrophy (CALD)

Interventions

Type	Name	Description
GENETIC	Lenti-D Drug Product (eli-cel)	Participants received a single IV infusion of Lenti-D Drug Product.

Timeline

• Start date: 2013-08-21
• Primary completion: 2021-03-26
• Completion: 2021-03-26
• First posted: 2013-07-11
• Last updated: 2022-04-25
• Results posted: 2022-04-25

Locations

8 sites across 6 countries: United States, Argentina, Australia, France, Germany, United Kingdom

Regulatory

• FDA-regulated drug study