Recruiting

Elivaldogene Autotemcel

Sponsor:

bluebird bio

Code:

NCT06224413

Conditions

Cerebral Adrenoleukodystrophy (CALD)

Eligibility Criteria

Sex: Male

Age: 0+

Healthy Volunteers: Not accepted

Interventions

No Intervention

Study Details

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.

Conditions

Cerebral Adrenoleukodystrophy (CALD)

Study ID

NCT06224413

Start date

Mar 27, 2024

Status verified date

Jun, 2025

Completion date

Dec 30, 2047

Anticipated

Primary completion date

Dec 30, 2047

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 0+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

120 participants

Anticipated

Interventions and Outcome Measures

Arms

All Participants

Participants with CALD treated with eli-cel in the post marketing setting will be followed in this registry study for up to 15 years after eli-cel infusion to collect real-world longitudinal data, and evaluate the outcomes.

Interventions

No Intervention

This is non-interventional study.

Primary outcome measure

  • Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) [ Time Frame: Through 15 years postinfusion ]
  • Number of Participants with Newly Diagnosed Malignancies [ Time Frame: Through 15 years postinfusion ]
  • Number of Participants with Insertional Oncogenesis [ Time Frame: Through 15 years postinfusion ]
  • Major Functional Disability (MFD)-Free Survival [ Time Frame: Through 15 years postinfusion ]

Central Contacts and Locations

Central contacts

bluebird bio

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

Locations

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

UT Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

More Information

Sponsor

bluebird bio

Last update posted

Jul 2, 2025

Last verified

Jun, 2025

Keywords

  • Adrenoleukodystrophy
  • Adrenoleukodystrophy protein

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by bluebird bio on 2025-07-02.