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ANAVEX2-73 Study in Patients With Rett Syndrome (AVATAR)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03941444
Recruitment Status : Recruiting
First Posted : May 8, 2019
Last Update Posted : July 2, 2020
Sponsor:
Information provided by (Responsible Party):
Anavex Life Sciences Corp.

Tracking Information
First Submitted Date  ICMJE May 6, 2019
First Posted Date  ICMJE May 8, 2019
Last Update Posted Date July 2, 2020
Actual Study Start Date  ICMJE May 6, 2019
Estimated Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 6, 2019)
  • Incidence of Adverse Events [ Time Frame: 7 weeks ]
    Incidence of Adverse Events
  • Maximum Plasma Concentration [Cmax] of ANAVEX2-73 [ Time Frame: 7 weeks ]
    PK of ANAVEX2-73 and metabolite
  • Area Under the Curve [AUC] of ANAVEX2-73 [ Time Frame: 7 weeks ]
    PK of ANAVEX2-73 and metabolite
  • Lipid panel [ Time Frame: 7 weeks ]
    Significant laboratory findings
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 6, 2019)
  • RSBQ [ Time Frame: 7 weeks ]
    Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ)
  • CGI-I [ Time Frame: 7 weeks ]
    Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: May 6, 2019)
  • Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: 7 weeks ]
    Anxiety, Depression, and Mood Scale (ADAMS)
  • Children's Sleep Habits Questionnaire (CSHQ) [ Time Frame: 7 weeks ]
    Children's Sleep Habits Questionnaire (CSHQ)
  • Seizure Frequency via seizure diary [ Time Frame: 7 weeks ]
    Seizure Frequency via seizure diary
  • Genetic variant SIGMAR1, COMT [ Time Frame: 7 weeks ]
    Genetic variant SIGMAR1, COMT
  • Glutamate Plasma Concentration [ Time Frame: 7 weeks ]
    Glutamate Plasma Concentration
  • GABA Plasma Concentration [ Time Frame: 7 weeks ]
    GABA Plasma Concentration
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE ANAVEX2-73 Study in Patients With Rett Syndrome
Official Title  ICMJE A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome
Brief Summary ANAVEX2-73-RS-002 is a Phase 2, 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 2 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 who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Rett Syndrome
Intervention  ICMJE
  • Drug: ANAVEX2-73
    Liquid oral solution
  • Drug: Placebos
    Liquid oral solution
Study Arms  ICMJE
  • Experimental: Active arm
    ANAVEX2-73 liquid oral solution
    Intervention: Drug: ANAVEX2-73
  • Placebo Comparator: Placebo arm
    Placebo liquid oral solution
    Intervention: Drug: Placebos
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 6, 2019)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2020
Estimated Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
  • 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 CYP3A4 and CYP2C19 inhibitors and inducers.
  • Patients with hepatic and renal impairment.
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE 18 Years to 45 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Walter Kaufmann, MD 844-689-3939 rett@anavex.com
Listed Location Countries  ICMJE Australia,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03941444
Other Study ID Numbers  ICMJE ANAVEX2-73-RS-002
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Anavex Life Sciences Corp.
Study Sponsor  ICMJE Anavex Life Sciences Corp.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Anavex Life Sciences Corp.
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP