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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Genetix Biotherapeutics Inc.
Study ID
NCT06224413
Status
Recruiting
Apply to this trial

Conditions

  • Cerebral Adrenoleukodystrophy (CALD)

Eligibility Criteria

Sex
MALE
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • No Intervention — OTHER
    This is non-interventional study.

Study Details

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.

Key Dates

Start date
Mar 27, 2024
Status verified
Jun 2025
Primary completion
Dec 30, 2047
Completion
Dec 30, 2047

Study Design

Enrollment
120 participants (estimated)

Arms

  • Arm: 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.

Primary Outcome Measure

Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) [ Time Frame: Through 15 years postinfusion ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02115-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
UT Southwestern Medical CenterDallasTexas75390-

Find similar trials in Boston, MA

By research site
Boston Children's Hospital· Boston, MAChildren's Hospital of Philadelphia· Philadelphia, PAUT Southwestern Medical Center· Dallas, TX

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