A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

Launched by BLUEBIRD BIO · Jan 16, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the safety and effectiveness of a treatment called elivaldogene autotemcel (also known as eli-cel) for patients with a condition known as Cerebral Adrenoleukodystrophy (CALD). The goal is to track how well patients do over time after receiving this treatment and to monitor any new health issues that may arise, including serious complications like cancer. The study will include 120 participants who have received the treatment, and it will continue to follow them for 15 years to gather important information about their health and quality of life.

To be eligible for the study, participants must have been treated with eli-cel at a participating center in the United States and must be able to provide consent to join. Specifically, 24 of the participants will have more advanced CALD and must meet additional criteria based on MRI results. There are no exclusion criteria, meaning that as long as participants meet the inclusion requirements, they can join. Throughout the study, participants can expect regular follow-ups with their doctors to monitor their progress and any potential side effects from the treatment.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Participants who fulfill the following criteria will be eligible for inclusion in this Registry Study.
• • Participant must be treated with eli-cel in the post marketing setting at a center in the United States (US) that participates in the Registry Study.
• • Participant must have provided an informed consent and/or assent to participate in Center for International Blood and Marrow Transplant Research (CIBMTR) registry.
• • Participant must have provided an informed consent and/or assent to participate in the Registry Study.
• • Participant must receive follow up care by a US-based physician with the ability to submit REG-502 data.
• Registry Study Subpopulation inclusion:
• Twenty-four of the 120 patients in the Registry Study must meet the following inclusion criteria which will be used to create the more advanced early active CALD subpopulation:
• • • Participant must meet the above inclusion criteria and have a Loes score of 4.5 through 9.0 with GdE+ (gadolinium enhancement positivity) from an MRI performed before treatment with eli-cel and with NFS of 0 or 1 at baseline.
• Exclusion Criteria:
• • There are no exclusion criteria for this Registry Study.