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Clinical Trial Summary

This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02563860
Study type Interventional
Source Montefiore Medical Center
Contact
Status Completed
Phase Phase 2
Start date July 2015
Completion date July 2016

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