NCT01896102View on ClinicalTrials.gov

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

PHASE2/PHASE3CompletedINTERVENTIONAL
Enrollment

32

Participants

Timeline

Start Date

August 21, 2013

Primary Completion Date

March 26, 2021

Study Completion Date

March 26, 2021

Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
GENETIC

Lenti-D Drug Product (eli-cel)

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

Trial Locations (8)

5006

Women and Children's Hospital, North Adelaide

55455

University of Minnesota, Minneapolis

90095

Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center, Los Angeles

94275

Hôpital Bicêtre, Le Kremlin-Bicêtre

02115

Boston Children's Hospital/Massachusetts General Hospital, Boston

C1022

Medeos SRL, Buenos Aires

04103

University of Leipzig, Leipzig

WC1N3JH

Great Ormond Street Hospital for Children NHS Foundation Trust, London

Sponsors
All Listed Sponsors
lead

Genetix Biotherapeutics Inc.

INDUSTRY

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) | Biotech Hunter | Biotech Hunter