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Study of ANAVEX2-73 in Patients With Rett Syndrome

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ClinicalTrials.gov Identifier: NCT03758924
Recruitment Status : Recruiting
First Posted : November 29, 2018
Last Update Posted : March 28, 2019
Sponsor:
Collaborator:
International Rett Syndrome Foundation Rettsyndrome.org
Information provided by (Responsible Party):
Anavex Life Sciences Corp.

Tracking Information
First Submitted Date  ICMJE November 25, 2018
First Posted Date  ICMJE November 29, 2018
Last Update Posted Date March 28, 2019
Actual Study Start Date  ICMJE February 28, 2019
Estimated Primary Completion Date November 1, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 28, 2018)
  • 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 Complete list of historical versions of study NCT03758924 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 28, 2018)
  • RSBQ [ Time Frame: 7 weeks ]
    Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ). Total score and a pre-specified subset of the 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. Total score and a pre-specified subset of the CGI-I
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: November 28, 2018)
  • 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)
  • Visual Analog Scale (VAS) [ Time Frame: 7 weeks ]
    Visual Analog Scale (VAS)
  • Seizure Frequency via seizure diary [ Time Frame: 7 weeks ]
    Seizure Frequency via seizure diary
  • Genetic variant SIGMAR1, COMT [ Time Frame: 7 weeks ]
    Pre-specified endpoint
  • Glutamate Plasma Concentration [ Time Frame: 7 weeks ]
    Biomarker
  • GABA Plasma Concentration [ Time Frame: 7 weeks ]
    Biomarker
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Study of ANAVEX2-73 in Patients With Rett Syndrome
Official Title  ICMJE A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome
Brief Summary

Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study.

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.

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: Triple (Participant, Care Provider, Investigator)
Masking Description:
Double-Blind, Randomized, Placebo-Controlled
Primary Purpose: Treatment
Condition  ICMJE Rett Syndrome
Intervention  ICMJE
  • Drug: ANAVEX2-73
    Liquid oral solution
  • Drug: Placebo
    Liquid oral solution
Study Arms  ICMJE
  • Experimental: Active arm
    Week 0-7: Take 1 ml orally of the product daily (solution of ANAVEX2-73)
    Intervention: Drug: ANAVEX2-73
  • Placebo Comparator: Placebo arm
    Week 0-7: Take 1 ml orally of the product daily (placebo)
    Intervention: Drug: Placebo
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: November 28, 2018)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 30, 2019
Estimated Primary Completion Date November 1, 2019   (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.
  • 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 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 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 on potent CYP 3A4 and CYP2C19 inhibitors and inducers.
  • 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.
  • 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 United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03758924
Other Study ID Numbers  ICMJE ANAVEX2-73-RS-001
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Anavex Life Sciences Corp.
Study Sponsor  ICMJE Anavex Life Sciences Corp.
Collaborators  ICMJE International Rett Syndrome Foundation Rettsyndrome.org
Investigators  ICMJE
Principal Investigator: Walter Kaufmann, MD Emory University SOM
PRS Account Anavex Life Sciences Corp.
Verification Date February 2019

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