A study to explore the safety, tolerability, pharmacokinetic profile, and potential efficacy of Guanabenz in patients with early childhood onset Vanishing White Matter (VWM)

Recruiting

Conditions: CACH
10029299
10012303

Registration Number: NL-OMON55425

Lead Sponsor: Vrije Universiteit Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 40

Inclusion Criteria

1. Genetically proven VWM with 2 clinically relevant mutations in one of the
EIF2B1-5 genes and a brain MRI compatible with the diagnosis.
2. Male or female whose disease duration at trial entry is 8 years or shorter.
3. Disease onset before the age of 6 years.
4. Able to stand up and walk at least 10 steps with or without some support of
one hand.
5. Lives within reasonable travel distance from Amsterdam. This means that the
patients are expected to come from The Netherlands, Belgium, Germany, or
France.

Exclusion Criteria

1. Clinically asymptomatic.
2. Comorbidity with another genetic defect.
3. Presence of an unrelated serious condition (e.g., developmental anomaly,
cardiac, liver or kidney disease).
4. Participation in another clinical study with therapeutic intervention.
5. Unable or unwilling to come to the VUmc site as required by the protocol.
6. Unable to undergo MRI due to metal-containing implants, such as cochlea
implant, neurostimulator or pacemaker.
7. Family situation in which adherence to the study medication or follow-up
procedures cannot be guaranteed.
8. Known allergy or hypersensitivity to guanabenz or to any of the other
components of the formulation used in this study.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>All adverse events and serious adverse events collected from the start of study<br /><br>treatment until the end of the study, applying the most recent version of the<br /><br>National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events<br /><br>(CTCAE, version 5.0, 27-Nov-2017).</p><br>

Secondary Outcome Measures