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

NCT01896102 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2022-04-25

Study results available
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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.

Conditions

  • Cerebral Adrenoleukodystrophy (CALD)

Interventions

GENETIC

Lenti-D Drug Product (eli-cel)

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

Sponsors & Collaborators

  • Genetix Biotherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Jakob Sieker, MD. · bluebird bio, Inc.

  • David Williams, MD · Boston Children's Hospital

  • Christine Duncan, MD · Boston Children's Hospital

  • Florian Eichler, MD · Massachusetts General Hospital

  • Satiro de Oliveira, MD · University of California, Los Angeles

  • Paul Orchard, MD · University of Minnesota

  • Adrian Thrasher, MD, PhD · Great Ormond Street Hospital for Chidren NHS Foundation Trust

  • Patrick Aubourg, MD, PhD · Hôpital Bicêtre

  • Jorn-Sven Kuhl, MD · University of Leipzig

  • Nicholas Smith, MD · Women and Children's Hospital

  • Hernan Amartino, MD · Medeos SRL

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-08-21
Primary Completion
2021-03-26
Completion
2021-03-26
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • France
  • Germany
  • 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 NCT01896102 on ClinicalTrials.gov