Trial Outcomes & Findings for Pharmacological Treatment of Rett Syndrome With Statins (NCT NCT02563860)

Results Overview

To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

During final week of treatment, week 32

Results posted on

2019-07-12

Participant Flow

Participant milestones

Participant milestones
Measure	Open Label Treatment with Lovastatin, dose escalating trial according to the following schedule: All participants were assigned to the specified sequence of interventions and all participants received all the three dosing regimen please make this explicit in the Arm/Group Description. In either case, separate Period 1-10 mg daily for 8 week Period 2-20 mg daily for 8 weeks Period 3-40 mg daily for 16 weeks. Lovastatin: dose escallating
Overall Study STARTED	20
Overall Study COMPLETED	19
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pharmacological Treatment of Rett Syndrome With Statins

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open Label n=20 Participants Female patients with genetically confirmed RTT, treatment arm only. 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. Lovastatin: dose escallating
Age, Categorical <=18 years	20 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	20 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: During final week of treatment, week 32

To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)

Outcome measures

Outcome measures
Measure	Open Label n=19 Participants 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. Lovastatin: dose escallating
Gait Velocity as Measured by GAITRite System	58.6 cm/sec Interval 43.8 to 71.1

SECONDARY outcome

Timeframe: final week of treatment, Week 32

Population: Calculation done on intent-to-treat population

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.

Outcome measures

Outcome measures
Measure	Open Label n=19 Participants 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. Lovastatin: dose escallating
Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System Pre-treatment	44.85 percent Interval 25.85 to 68.1
Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System Post treatment	58.93 percent Interval 47.55 to 70.25

Adverse Events

Open Label

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Open Label n=20 participants at risk 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. Lovastatin: dose escallating
Psychiatric disorders mild	5.0% 1/20 • Number of events 1

Additional Information

Aleksandra Djukic

Montefiore MC

Phone: 7189204378

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place