NCT02563860

Pharmacological Treatment of Rett Syndrome With Statins

Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Montefiore Medical Center · Academic / Other
• Sex: Female
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Rett Syndrome

Interventions

Type	Name	Description
DRUG	Lovastatin	dose escallating

Timeline

• Start date: 2015-07-01
• Primary completion: 2016-07-01
• Completion: 2016-07-01
• First posted: 2015-09-30
• Last updated: 2019-07-12
• Results posted: 2019-05-07

Locations

1 site across 1 country: United States