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

Bluebird Bio

Status: Active, not recruiting

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

Treatments: Genetic: No interventional drug product utilized in this follow-up study

Study type: Observational

Funder types: Industry

Identifiers

NCT02698579

2015-002805-13 (EudraCT Number)

LTF-304

2024-513904-33-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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.

Enrollment: 64 patients

Sex: Male

Ages: Under 19 years old

Volunteers: No Healthy Volunteers

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

Trial design

64 participants in 1 patient group

Long-term followup

Description:

Participants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical studies ALD-102 and ALD-104) and who meet the eligibility criteria for the Study LTF-304 will be followed in this long-term followup study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies).

Treatment:

Genetic: No interventional drug product utilized in this follow-up study