NL-OMON55425RecruitingNot Applicable
A study to explore the safety, tolerability, pharmacokinetic profile, and potential efficacy of Guanabenz in patients with early childhood onset Vanishing White Matter (VWM) - Guanabenz in VWM
Conditions
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Enrollment
- 40
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 2 to 15 (—)
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
- •5. Lives within reasonable travel distance from Amsterdam. This means that the
- •patients are expected to come from The Netherlands, Belgium, Germany, or
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.
Investigators
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