Pharmacological Treatment of Rett Syndrome With Statins

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Rett syndrome
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Brief Title

Pharmacological Treatment of Rett Syndrome With Statins

Official Title

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

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.

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Gait Velocity as Measured by GAITRite System

Secondary Outcome

 Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System

Condition

Rett Syndrome

Intervention

Lovastatin

Study Arms / Comparison Groups

 Open label
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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

20

Start Date

July 2015

Completion Date

July 2016

Primary Completion Date

July 2016

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.

Gender

Female

Ages

3 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Aleksandra Djukic, MD PhD, ,

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT02563860

Organization ID

2014-4253

Responsible Party

Principal Investigator

Study Sponsor

Montefiore Medical Center

Collaborators

 Rett Syndrome Research Trust

Study Sponsor

Aleksandra Djukic, MD PhD, Principal Investigator, Montefiore Medical Center

Verification Date

July 2019