ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

92 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-07-01

Study Completion Date

2023-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

ANAVEX2-73-RS-003 is a Phase 2/3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 5-17 years of age with RTT using endpoints including multiple clinical and exploratory molecular and biochemical measures.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Anakinra Pilot 2 - A Study to Optimise Dose and Route of Administration of Anakinra in Preterm Infants

NCT07254000

Premature Infants Very Premature Infants Inflammation

RECRUITING PHASE2

Comparing Reversal With Neostigmine and Sugammadex in Paediatric

NCT03137290

Neuromuscular Blockade

COMPLETED NA

Dose Escalating Study of Rotigotine in Pediatric Subjects With Restless Legs Syndrome

NCT01495793

Restless Legs Syndrome

COMPLETED PHASE2

Intravenous (IV) Solutions for Dehydration in Children With Gastroenteritis

NCT01234883

Dehydration Gastroenteritis

TERMINATED PHASE4

Trial of 70% Ethanol Versus Heparin to Reduce the Rate of Central Line Infections in Children With Short Bowel Syndrome

NCT01263574

WITHDRAWN PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This Phase 2/3 efficacy study is designed as a double-blind, randomized, placebo-controlled study.

This is a 12-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 5-17 years of age. A voluntary option will be offered for all patients who meet the exposure criteria for ANAVEX2-73 to continue a 48-week open label extension.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Rett Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

ANAVEX2-73 Active

ANAVEX2-73 liquid oral solution

Group Type EXPERIMENTAL

ANAVEX2-73 oral liquid

Intervention Type DRUG

Liquid oral solution

ANAVEX2-73 Placebo

Placebo liquid oral solution

Group Type PLACEBO_COMPARATOR

Placebo oral liquid

Intervention Type DRUG

Liquid oral solution

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ANAVEX2-73 oral liquid

Liquid oral solution

Intervention Type DRUG

Placebo oral liquid

Liquid oral solution

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Blarcamesine

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Aged ≥ 5 years to 17 (inclusive).
* Diagnosis of classic RTT, according to 2010 criteria, and a MECP2 mutation.
* Post-regression stage, defined as ≥ 6 months since last loss of spoken language or motor (fine or gross) skills.
* Clinical Global Impression - Severity (CGI-S) score of 4 or greater at Screening.
* Current pharmacological treatment regimen, including supplements, has been stable for at least 4 weeks.
* If on 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/LAR will not electively initiate new or modify ongoing interventions for the duration of the study.
* The subject's caregiver/LAR is English-speaking and has sufficient language skills to complete the caregiver assessments and has the ability to keep accurate seizure diaries.
* If participant is a woman of childbearing potential (WOCBP#), a negative urine or serum pregnancy test is required to confirm she is not pregnant.
* Prior to the conduct of study-specific procedures, the subject's parent/caregiver/LAR must provide written informed consent. If applicable, the research team must attempt to obtain consent from both parents.

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 or clinically evident 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., long QT) 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.
* Any other criteria (such as a clinically significant screening blood test result), which in the opinion of the Investigator could interfere with the study conduct or outcome.
* Treatment with strong inhibitors or inducers of CYP3A4 or CYP2C19 is not stable (drug, dose) for 30 days prior to screening. Although these medications are not excluded, caution is advised when enrolling participants on potent CYP3A4 or CYP2C19 inducers or inhibitors (see respective section).
* Patients with hepatic and renal impairment.

Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No