Pharmacological Treatment of Rett Syndrome With Statins

Rett Syndrome Clinical Trial

— Statins  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02563860
• Other study ID #: 2014-4253
• Status: Completed
• Phase: Phase 2
• Start date: July 2015
• Est. completion date: July 2016

Study information

• Verified date: July 2019
• Source: Montefiore Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: July 2016
• Est. primary completion date: July 2016
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 3 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Rett Syndrome
• Syndrome

Intervention

Drug:
• Lovastatin
dose escallating

Locations

Country	Name	City	State
United States	Montefiore Medical center	Bronx	New York

Sponsors (2)

Lead Sponsor	Collaborator
Montefiore Medical Center	Rett Syndrome Research Trust

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Gait Velocity as Measured by GAITRite System	To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)	During final week of treatment, week 32
Secondary	Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System	Novelty score is considered a composite measure of visual memory.Test is a standardized neuropsychological test( "Rose test") adapted for eye tracking system.; The present study made use of a well-established battery of "visual paired comparison" problems .There were nine problems: five using achromatic photos of faces and four using multicolored abstract patterns paired stimuli , separated by 23°. For each problem, two identical stimuli are briefly presented side by side for familiarization, then the familiar and a new one are paired on test; Recognition(memory) is indexed by a novelty score (percentage of looking to the novel target on test).; Higher novelty scores were related to better recognition (index of better memory) Scores range from 0.0-1.0 (0-100%) and indicate 1) more overall looking at novel target and (2) more fixations to novel targets.; Score of .5 (50%) indicates looking "by change" only with no recognition.	final week of treatment, Week 32