Rett Syndrome Clinical Trial
Official title:
Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
Excess neuronal cholesterol plays a role in the pathophysiology of Rett syndrome(RTT) and the
investigators hypothesise that inhibition of cholesterol synthesis in the CNS will reduce
neuronal cholesterol and lead to improvement in Rett syndrome related symptoms.
Goal: To determine the optimal dosing regiment of Lovastatin for patients with RTT and to
identify the most appropriate primary outcome measure for the subsequent Phase 3 study.
Phase 2, dose escalation study.
Primary outcome: Gait speed Secondary outcomes: respiratory function, cognition, EEG
(encephalopathy), Rett syndrome severity scale, neurodevelopmental assessment (QOL)
20 ambulatory female patients with genetically confirmed.
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