NCT03852498View on ClinicalTrials.gov

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

PHASE3CompletedINTERVENTIONAL
Enrollment

35

Participants

Timeline

Start Date

January 24, 2019

Primary Completion Date

July 24, 2023

Study Completion Date

July 24, 2023

Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
GENETIC

Lenti-D

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

Trial Locations (8)

55455

University of Minnesota, Minneapolis

75019

Hôpital Robert Debré, Paris

94304

Lucile Packard Children's Hospital, Palo Alto

02215

Boston Children's Hospital/Massachusetts General Hospital, Boston

04103

Universitätsklinikum Leipzig AöR, Leipzig

00165

Ospedale Pediatrico Bambino Gesù, Rome

3508AB

Prinses Maxima Center, Utrecht

WC1N3JH

UCL-ICH/Great Ormond Street Hospital, London

Sponsors
All Listed Sponsors
lead

Genetix Biotherapeutics Inc.

INDUSTRY

NCT03852498 - A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD) | Biotech Hunter | Biotech Hunter