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NCT06224413 · ClinicalTrials.gov registry record
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
A clinical trial of Cerebral Adrenoleukodystrophy (CALD), sponsored by Genetix Biotherapeutics.
- Recruiting
- Registry status
- 120
- Enrollment target
- 3
- Study locations
NCT06224413 is a study of Cerebral Adrenoleukodystrophy (CALD) that is actively recruiting participants, run by Genetix Biotherapeutics. The registered enrollment target is 120 participants, above the 45-participant average among 3 other Cerebral Adrenoleukodystrophy (CALD) trials with a reported enrollment target (167% higher). The trial reports 3 study locations across 3 states.
The verdict
NCT06224413, a study of Cerebral Adrenoleukodystrophy (CALD), is actively recruiting participants, sponsored by Genetix Biotherapeutics.
- RECRUITING
- Registry status
- 120 participants
- Enrollment target
- 3
- Study locations
Study 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 Studied
Interventions
- OTHER No Intervention
Study Locations (3)
Massachusetts
- Boston Children's Hospital - Boston
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Texas
- UT Southwestern Medical Center - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 120 participants |
| Start Date | 2024-03-27 |
| Est. Completion | 2047-12-30 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06224413
The ClinicalTrials.gov registry entry for NCT06224413 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 120 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 45-participant average among 3 other Cerebral Adrenoleukodystrophy (CALD) trials with a reported enrollment target (167% higher). The listed sponsor is Genetix Biotherapeutics, which has 12 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Cerebral Adrenoleukodystrophy (CALD) appearing as the primary indexed condition, and to 1 intervention - of which No Intervention is the first listed.
NCT06224413 reports 3 study locations spanning 3 distinct geographic areas - top geographies include Massachusetts, Pennsylvania, Texas.
Frequently Asked Questions
What is clinical trial NCT06224413 about?
NCT06224413 is a clinical study titled "A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel". 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 wit...
What is the current status of trial NCT06224413?
This trial is currently recruiting. The enrollment target is 120 participants. The study started on 2024-03-27. Estimated completion is 2047-12-30.
What conditions does trial NCT06224413 study?
This clinical trial studies the following conditions: Cerebral Adrenoleukodystrophy (CALD).
What interventions are being tested in trial NCT06224413?
The interventions under investigation include: No Intervention (OTHER).
Who is sponsoring clinical trial NCT06224413?
This trial is sponsored by Genetix Biotherapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06224413 being conducted?
This trial has 3 study locations across Massachusetts, Pennsylvania, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.