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Pharmacological Treatment of Rett Syndrome With Statins (Statins)

Primary Purpose

Rett Syndrome

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Lovastatin
Sponsored by
Montefiore Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Rett Syndrome focused on measuring Treatment trial, Lovastatin

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)FemaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Montefiore Medical center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open label

Arm Description

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.

Outcomes

Primary Outcome Measures

Gait Velocity as Measured by GAITRite System
To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)

Secondary Outcome Measures

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.

Full Information

First Posted
July 20, 2015
Last Updated
July 11, 2019
Sponsor
Montefiore Medical Center
Collaborators
Rett Syndrome Research Trust
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1. Study Identification

Unique Protocol Identification Number
NCT02563860
Brief Title
Pharmacological Treatment of Rett Syndrome With Statins
Acronym
Statins
Official Title
Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
July 2015 (undefined)
Primary Completion Date
July 2016 (Actual)
Study Completion Date
July 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Montefiore Medical Center
Collaborators
Rett Syndrome Research Trust

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Rett Syndrome
Keywords
Treatment trial, Lovastatin

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Open label
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Lovastatin
Other Intervention Name(s)
Mevacor
Intervention Description
dose escallating
Primary Outcome Measure Information:
Title
Gait Velocity as Measured by GAITRite System
Description
To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)
Time Frame
During final week of treatment, week 32
Secondary Outcome Measure Information:
Title
Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System
Description
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

10. Eligibility

Sex
Female
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Aleksandra Djukic, MD PhD
Organizational Affiliation
Montefiore Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Montefiore Medical center
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Pharmacological Treatment of Rett Syndrome With Statins

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