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COMPLETEDPHASE3

ANAVEX2-73 Study in Patients With Rett Syndrome

A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome

NCT03941444•Anavex Life Sciences Corp.

Summary

ANAVEX2-73-RS-002 is a Phase 3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 18 years and older with RTT using endpoints including multiple clinical and exploratory molecular and biochemical measures.

Detailed Description

This Phase 3 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study. This is a 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients to continue a 48-week open label extension.

Eligibility

Age

18 - 45 years

Sex

FEMALE

Healthy Volunteers

Inclusion Criteria

•Aged ≥ 18 years, inclusive.
•Diagnosis of classic RTT, according to 2010 criteria (Neul et al., 2010), and a MECP2 mutation.
•Current pharmacological treatment regimen, including supplements, has been stable for at least 4 weeks.
•If on antiepileptic drugs (AEDs), 1-4 AEDs allowed. Treatment must be stable (drug, dose, interval of administration) for 30 days prior to enrollment.
•If the subject is already receiving stable non-pharmacologic educational, behavioral, and/or dietary interventions, participation in these programs must have been continuous during the 90 days prior to the screening visit and subjects or their parent/caregiver/legally authorized representative (LAR) will not electively initiate new or modify ongoing interventions for the duration of the study. 'Study duration' is defined as lasting from the screening visit until the treatment is terminated. For participants in the 16-21 years range, typical school vacations are not considered modifications of stable programming.
•Ability to keep accurate seizure diaries or have caregiver who can keep accurate seizure diaries.
•Confirmation from the participant that, if of childbearing potential is not pregnant through urine pregnancy testing. Female patients of childbearing potential and at risk for pregnancy must agree to abstinence.
•Prior to the conduct of study-specific procedures, the subject's parent/caregiver/LAR must provide written informed consent. If applicable, the research team

Exclusion Criteria

•Patients who have a progressive medical or neurological condition that in the opinion of the Investigator would interfere with the conduct of the study.
•Current clinically significant systemic illness that is likely to result in deterioration of the patient's condition or affect the patient's safety during the study.
•History of clinically evident stroke or clinically significant carotid or vertebrobasilar stenosis or plaque or other history of neurologic (e.g., head trauma with loss of consciousness) or psychiatric condition that the Investigator deems may interfere with interpretability of data.
•Indication of liver disease, defined by serum levels of ALT (SGPT), AST (SGOT), or alkaline phosphatase above 3x upper limit of normal (ULN) as determined during screening.
•Treatment with immunosuppressive medications (e.g., systemic corticosteroids) within the last 90 days (topical and nasal corticosteroids and inhaled corticosteroids for asthma are permitted) or chemotherapeutic agents for malignancy within the last 3 years.
•Other clinically significant abnormality on physical, neurological, laboratory, or electrocardiogram (ECG) examination (e.g., atrial fibrillation) that could compromise the study or be detrimental to the participant.
•Any known hypersensitivity to any of the excipients contained in the study drug or placebo formulation.
•Other co-morbid or chronic illness beyond that known to be associated with RTT.
•Subjects who plan to initiate or change pharmacologic or nonpharmacologic intervention during the course of the study.
•Subjects taking another investigational drug currently or within the last 30 days.
+3 more criteria

Locations (7)

HammondCare

Greenwich, New South Wales, Australia

Mater Misericordiae Ltd

South Brisbane, Queensland, Australia

Royal Melbourne Hospital (RMH)

Melbourne, Victoria, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

The Keogh Institute for Medical Research

Nedlands, Western Australia, Australia

King's College of London

London, UK, United Kingdom

Manchester CGM, St. Mary's Hospital

Manchester, UK, United Kingdom

Key Dates

Start Date

May 6, 2019

Primary Completion Date

September 30, 2021

Completion Date

September 30, 2021

Last Updated

January 27, 2022

Enrollment

ACTUAL participants

Conditions

Rett Syndrome

Interventions

ANAVEX2-73

DRUG

Placebo

DRUG

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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: January 27, 2022Data 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.

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ANAVEX2-73 Study in Patients With Rett Syndrome

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