Loading clinical trials...

COMPLETEDPHASE2

Pharmacological Treatment of Rett Syndrome With Statins

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

NCT02563860•Montefiore Medical Center

View on ClinicalTrials.gov

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.

Eligibility

Age

3 - No limit years

Sex

FEMALE

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.

Locations (1)

Montefiore Medical center

The Bronx, New York, United States

Key Dates

Start Date

July 1, 2015

Primary Completion Date

July 1, 2016

Completion Date

July 1, 2016

Last Updated

July 12, 2019

Enrollment

ACTUAL participants

Conditions

Rett Syndrome

Interventions

Lovastatin

DRUG

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT06338267

Rett Syndrome

View study

RECRUITINGEARLY_PHASE1

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

NCT06856759

Rett Syndrome

View study

RECRUITING

GROWing Up With Rare GENEtic Syndromes

NCT04463316

Prader-Willi Syndrome

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 12, 2019Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Pharmacological Treatment of Rett Syndrome With Statins

Inclusion Criteria

Exclusion Criteria

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

GROWing Up With Rare GENEtic Syndromes

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome