NL-OMON51993尚未招募2 期
A Proof-of-Concept Study to Explore the Potential Efficacy of Deferiprone in Patients With Pelizaeus-Merzbacher disease (PMD) - Deferiprone as possible treatment for Pelizaeus-Merzbacher disease
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 11（—）
入选标准
- •- Genetically proven PMD with a clinically relevant mutation in PLP1 (missense
- •mutation or duplication/triplication) and an MRI compatible with the diagnosis.
- •- Present age between 6 months and 8 years of age.
- •- Connatal or classic form of the disease (defined as not being able to sit
- •without support and/or a mutation predicting this form, e.g. PLP1 duplication
- •or higher copy numbers; known missense mutations associated with severe forms).
排除标准
- •- important comorbidity as another genetic disease
- •- liver or kidney disease
- •- neutropenia in patient's history
- •- severe iron deficiency
研究者
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