A Study to Explore the Safety, Tolerability, Pharmacokinetic Profile, and Potential Efficacy of Guanabenz in Patients With Early-Childhood Onset Vanishing White Matter (VWM)

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-503320-89-00

Acronym

VWM1

Enrollment

Registered

2024-09-26

Start date

2021-05-31

Completion date

2025-12-29

Last updated

2025-11-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Vanishing White Matter

Brief summary

All adverse events and serious adverse events collected from the start of study treatment until the end of the study, applying the most recent version of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, version 5.0, 27-Nov-2017), as well as applying

Detailed description

Guanabenz PK parameters in plasma, such as Cmax, AUC, half-life, and predicted trough concentration (Ctrough) at steady state, Quantitative brain MRI parameters:, Clinical parameters:, Overall survival

Interventions

DRUGGuanabenz capsule 2 mg in 180 mg blend 1

DRUGGuanabenz capsule 8 mg in 160 mg blend 2

DRUGGuanabenz capsule 6 mg in 120 mg blend 2

DRUGGuanabenz capsule 24 mg in 480 mg blend 2

DRUGGuanabenz capsule 1 mg in 90 mg blend 1

DRUGGuanabenz capsule 16 mg in 320 mg blend 2

DRUGGuanabenz capsule 4 mg in 360 mg blend 1

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
All adverse events and serious adverse events collected from the start of study treatment until the end of the study, applying the most recent version of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, version 5.0, 27-Nov-2017), as well as applying	—

Secondary

Measure	Time frame
Guanabenz PK parameters in plasma, such as Cmax, AUC, half-life, and predicted trough concentration (Ctrough) at steady state, Quantitative brain MRI parameters:, Clinical parameters:, Overall survival	—

Countries

Netherlands

Outcome results

None listed

Source: EU CTIS · Data processed: Sep 1, 2026