RecruitingNot Applicable

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

United States120 participantsStarted 2024-03-27

Plain-language summary

The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.

Who can participate

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)

Timeframe: Through 15 years postinfusion

Number of Participants with Newly Diagnosed Malignancies

Timeframe: Through 15 years postinfusion

Number of Participants with Insertional Oncogenesis

Timeframe: Through 15 years postinfusion

Major Functional Disability (MFD)-Free Survival

Timeframe: Through 15 years postinfusion

Trial details

NCT IDNCT06224413

SponsorGenetix Biotherapeutics Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2047-12-30

Contact for this trial

bluebird bio

+1-833-999-6378 clinicaltrials@bluebirdbio.com

View on ClinicalTrials.gov More Cerebral Adrenoleukodystrophy (CALD) trials

Plain-language summary

Who can participate

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)

Timeframe: Through 15 years postinfusion

Number of Participants with Newly Diagnosed Malignancies

Timeframe: Through 15 years postinfusion

Number of Participants with Insertional Oncogenesis

Timeframe: Through 15 years postinfusion

Major Functional Disability (MFD)-Free Survival

Timeframe: Through 15 years postinfusion