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Active (not recruiting) NCT02698579

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Conditions: Cerebral Adrenoleukodystrophy (CALD) Adrenoleukodystrophy (ALD) X-Linked Adrenoleukodystrophy (X-ALD)

Sponsor: Genetix Biotherapeutics Inc.

trial.available_in: БГ
Overview
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study ALD-104). After completing a parent clinical study (approximately 2 years), eligible participants will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
Who can participate
Inclusion Criteria: * Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable * Have received eli-cel in a parent clinical study Exclusion Criteria: * There are no exclusion criteria for this study
Locations 9
Argentina (1)
Instituto Neurogenia
Caba
Australia (1)
Women's and Children's Hospital
North Adelaide
Brazil (1)
Hospital das Clínicas da Universidade de São Paulo
São Paulo
France (1)
Hôpital Bicêtre
Le Kremlin-Bicêtre , Cedex
Germany (1)
Universitätsklinikum Leipzig AöR
Leipzig
Italy (1)
Ospedale Pediatrico Bambino Gesù
Rome
Netherlands (1)
Prinses Maxima Center
Utrecht
United Kingdom (1)
Royal Free London Hospital
London , England
United States (1)
Mattel Children's Hospital-UCLA
Los Angeles , California
Technical details
Status
Active (not recruiting)
Study type
OBSERVATIONAL
Sex
Male only
Maximum age
19 Years
Healthy volunteers
No
Start date
22.01.2016
Completion date
01.08.2038
Registry ID
NCT02698579
Source
anzctr
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