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

NCT02698579 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 64

Last updated 2025-02-20

No results posted yet for this study

Summary

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.

Conditions

  • Cerebral Adrenoleukodystrophy (CALD)
  • Adrenoleukodystrophy (ALD)
  • X-Linked Adrenoleukodystrophy (X-ALD)

Interventions

GENETIC

No interventional drug product utilized in this follow-up study

Participants received a single IV infusion of Lenti-D Drug Product (also known as elivaldogene autotemcel or eli-cel) in either parent Study ALD-102 or ALD-104. The objectives of this long-term follow-up study are to assess long-term safety and efficacy following completion of participation in parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant are conducted in this study.

Sponsors & Collaborators

  • Genetix Biotherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Vinod K Prasad, MD, FRCP · bluebird bio, Inc.

Eligibility

Max Age
19 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-22
Primary Completion
2038-08-31
Completion
2038-08-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Brazil
  • France
  • Germany
  • Italy
  • Netherlands
  • 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 NCT02698579 on ClinicalTrials.gov