Pharmacological Treatment of Rett Syndrome With Statins (Statins)
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|ClinicalTrials.gov Identifier: NCT02563860|
Recruitment Status : Completed
First Posted : September 30, 2015
Results First Posted : May 7, 2019
Last Update Posted : July 12, 2019
|Condition or disease||Intervention/treatment||Phase|
|Rett Syndrome||Drug: Lovastatin||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)|
|Study Start Date :||July 2015|
|Actual Primary Completion Date :||July 2016|
|Actual Study Completion Date :||July 2016|
Experimental: Open label
Treatment with Lovastatin, dose escalating trial according to the following schedule:
10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.
Other Name: Mevacor
- Gait Velocity as Measured by GAITRite System [ Time Frame: During final week of treatment, week 32 ]To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)
- Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System [ Time Frame: final week of treatment, Week 32 ]
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02563860
|United States, New York|
|Montefiore Medical center|
|Bronx, New York, United States, 10467|
|Principal Investigator:||Aleksandra Djukic, MD PhD||Montefiore Medical Center|