Clinical trial NCT06224413: A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
Official title: A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cerebral Adrenoleukodystrophy (CALD)
Keywords: Adrenoleukodystrophy
Adrenoleukodystrophy protein
Lead sponsor: bluebird bio
Sponsor class: INDUSTRY
Collaborators: name: Center for International Blood and Marrow Transplant Research
First posted: 2024-01-25
Enrollment: 120
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.

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.

Primary outcomes:
measure: Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Newly Diagnosed Malignancies
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Insertional Oncogenesis
timeFrame: Through 15 years postinfusion
measure: Major Functional Disability (MFD)-Free Survival
timeFrame: Through 15 years postinfusion
Secondary outcomes:
measure: Overall Survival (OS)
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Serious Adverse Events (SAEs)
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Eli-cel Related AEs
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy
timeFrame: Through 15 years postinfusion
measure: Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
timeFrame: Through 15 years postinfusion
measure: Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy
timeFrame: Through 15 years postinfusion
measure: Number of Participants with Clinically Significant Abnormalities on Fluorescence In-situ Hybridization (FISH) and Next Generation Sequencing (NGS) in Subpopulation with Newly Diagnosed Hematologic Malignancy
timeFrame: Through 15 years postinfusion
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: bluebird bio, Inc.
name: Himal Lal Thakar, MD
role: STUDY_DIRECTOR
References: