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Completed

NCT Number: NCT02563860

Pharmacological Treatment of Rett Syndrome With Statins

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

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Key information

Conditions

Rett Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations X-Linked Intellectual Disability

Age range

3 year and older

Sex eligibility

Female

Study type

Interventional

Phase

Phase 2

Primary location

Montefiore Medical center

The Bronx, New York, 10467, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

lovastatin

Drug

dose escallating

Other names: Mevacor

Primary outcomes

  1. 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)

Secondary outcomes

  1. 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.

Sponsors and collaborators

Lead sponsor

Montefiore Medical Center

Other

Collaborators

  • Rett Syndrome Research Trust

Registry information

Official study title

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

Acronym: Statins

Important dates

Study start
2015
Primary completion
2016
Study completion
2016
First posted
Sep 30, 2015
Registry last updated
Jul 12, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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