NCT03852498

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

A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: Male
• Age: 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant'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 participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expected, although participants follow-up remains ongoing in the long-term follow-up Study LTF-304 (NCT02698579).

Conditions

• Cerebral Adrenoleukodystrophy (CALD)

Interventions

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

Timeline

• Start date: 2019-01-24
• Primary completion: 2023-07-24
• Completion: 2023-07-24
• First posted: 2019-02-25
• Last updated: 2024-05-24
• Results posted: 2024-05-24

Locations

8 sites across 6 countries: United States, France, Germany, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study