A Phase 2 interventional study of Lovastatin in Rett Syndrome, sponsored by Montefiore Medical Center. Completed at 1 site in United States. Open to female participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2019-07-12.
Sponsored by Montefiore Medical Center · Phase 2, Interventional, and Treatment
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
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.
98 studies on the registry are indexed under Rett Syndrome; 20 are open to participants now.
This study's enrollment of 20 is below the median of 30 across 64 interventional studies indexed under Rett Syndrome.
Browse Rett Syndrome studies →Montefiore Medical Center is the lead sponsor of 402 studies on the registry; 70 are open to participants now.
Of its 81 completed or terminated interventional studies of FDA-regulated products, 73 (90%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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.
Drug: Lovastatin
dose escallating
Also known as: Mevacor
Gait Velocity as Measured by GAITRite System
To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)
Time frame: During final week of treatment, week 32
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.
Time frame: final week of treatment, Week 32
| Milestone | Open Label |
|---|---|
| Started | 20 |
| Completed | 19 |
| Not completed | 1 |
To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)
| cm/sec | Open Label |
|---|---|
| Gait Velocity as Measured by GAITRite System | 58.6 (43.8 to 71.1) |
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.
| percent | Open Label |
|---|---|
| Pre-treatment | 44.85 (25.85 to 68.10) |
| Post treatment | 58.93 (47.55 to 70.25) |
Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Open Label | 0/20 (0%) | 0/20 (0%) | 1/20 (5%) |
| Event | Open Label |
|---|---|
| mildPsychiatric disorders | 1/20 |
| Age, Categorical(Participants) | Open Label |
|---|---|
| <=18 years | 20 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Open Label |
|---|---|
| Female | 20 |
| Male | 0 |
| Region of Enrollment(participants) | Open Label |
|---|---|
| United States | 20 |
Plan to share: No
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Montefiore Medical Center