ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome

Brief Title

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome

Official Title

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

Brief Summary

      ANAVEX2-73-RS-003 is a Phase 2/3, double-blind, randomized, placebo-controlled dose
      escalation safety, tolerability and efficacy study in patients 5-18 years of age with RTT
      using endpoints including multiple clinical and exploratory molecular and biochemical
      measures.
    

Detailed Description

      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 5-18 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.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional

Primary Outcome

RSBQ

Secondary Outcome

 Anxiety, Depression, and Mood Scale (ADAMS)

Condition

Rett Syndrome

Intervention

ANAVEX2-73 oral liquid

Study Arms / Comparison Groups

 ANAVEX2-73 Active

Description:  ANAVEX2-73 liquid oral solution

Publications

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

Recruitment Status

Drug

Estimated Enrollment

69

Start Date

June 2020

Completion Date

July 2021

Primary Completion Date

June 2021

Eligibility Criteria

        Inclusion Criteria:

          -  Aged ≥ 5 years to <18.

          -  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.

          -  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 (or serum) 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 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.

          -  Subjects on potent CYP 3A4 and CYP2C19 inhibitors and inducers (e.g., oxcarbazepine).

          -  Patients with hepatic and renal impairment.
      

Gender

Female

Ages

5 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

, +1(844)-689-3939, [email protected]

Location Countries

Australia

Location Countries

Australia

NCT ID

NCT04304482

Organization ID

ANAVEX2-73-RS-003

Responsible Party

Sponsor

Study Sponsor

Anavex Life Sciences Corp.

Collaborators

 Anavex Australia Pty Ltd.

Study Sponsor

, , 

Verification Date

June 2020