Pharmacological Treatment of Rett Syndrome With Statins

• Status: Completed
• Trial end date: 2016-07-01
• Target enrollment: 0
• Participant gender: Female

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Montefiore Medical Center
• Collaborator: Rett Syndrome Research Trust
• Treatments: Dihydromevinolin; Hydroxymethylglutaryl-CoA Reductase Inhibitors; L 647318; Lovastatin

Criteria

Inclusion Criteria:

• Females patients,

• Genetically confirmed RTT,

• Ambulatory.

Exclusion Criteria:

• Presence of co morbid non-Rett related disease,

• History of adverse reaction/hypersensitivity to statins,

• Levels of aspartate-amino trans-ferase (AST), alanine-amino transferase (ALT) or creatine kinase (CK) triple the normal values,

• Active liver disease,

• Concomitant use of strong CYP3A4 inhibitors,

• Condition predis-posing to renal failure secondary to rhabdomyolysis (scoliosis surgery within 6 months),

• Oral contraceptives use.