Trial Profile

A Phase 1b/2 Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following Fosigotifator Administration in Adult and Pediatric Subjects With Vanishing White Matter Disease

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 29 May 2025

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At a glance

07 Jan 2025 Treatment arms increased from 4 to 5. Primary endpoints updated. Age changed from ≥ 6 years to ≥ 6 months.
07 Jan 2025 Planned number of patients changed from 38 to 50.
07 Jan 2025 Planned End Date changed from 31 Dec 2027 to 1 Nov 2029.

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