NCT06224413 A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
| NCT ID | NCT06224413 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Genetix Biotherapeutics Inc. |
| Condition | Cerebral Adrenoleukodystrophy (CALD) |
| Study Type | OBSERVATIONAL |
| Enrollment | 120 participants |
| Start Date | 2024-03-27 |
| Primary Completion | 2047-12-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 120 participants in total. It began in 2024-03-27 with a primary completion date of 2047-12-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
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.
Contact & Investigator
Kiran Bhirangi, MD
STUDY DIRECTOR
bluebird bio, Inc.
Frequently Asked Questions
Who can join the NCT06224413 clinical trial?
This trial is open to male participants only, studying Cerebral Adrenoleukodystrophy (CALD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06224413 currently recruiting?
Yes, NCT06224413 is actively recruiting participants. Contact the research team at clinicaltrials@bluebirdbio.com for enrollment information.
Where is the NCT06224413 trial being conducted?
This trial is being conducted at Boston, United States, Philadelphia, United States, Dallas, United States.
Who is sponsoring the NCT06224413 clinical trial?
NCT06224413 is sponsored by Genetix Biotherapeutics Inc.. The principal investigator is Kiran Bhirangi, MD at bluebird bio, Inc.. The trial plans to enroll 120 participants.