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

NCT03852498 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2024-05-24

Study results available
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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

GENETIC

Lenti-D

Participants received a single IV infusion of Lenti-D Drug Product.

Sponsors & Collaborators

  • Genetix Biotherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Himal Lal Thakar, MD · bluebird bio, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-24
Primary Completion
2023-07-24
Completion
2023-07-24
FDA Drug
Yes

Countries

  • United States
  • France
  • Germany
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03852498 on ClinicalTrials.gov