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

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ClinicalTrials.gov Identifier: NCT03941444
Recruitment Status : Recruiting
First Posted : May 8, 2019
Last Update Posted : May 9, 2019
Sponsor:
Information provided by (Responsible Party):
Anavex Life Sciences Corp.

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.

Condition or disease Intervention/treatment Phase
Rett Syndrome Drug: ANAVEX2-73 Drug: Placebos Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome
Actual Study Start Date : May 6, 2019
Estimated Primary Completion Date : June 30, 2020
Estimated Study Completion Date : July 31, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: Active arm
ANAVEX2-73 liquid oral solution
Drug: ANAVEX2-73
Liquid oral solution

Placebo Comparator: Placebo arm
Placebo liquid oral solution
Drug: Placebos
Liquid oral solution




Primary Outcome Measures :
  1. Incidence of Adverse Events [ Time Frame: 7 weeks ]
    Incidence of Adverse Events

  2. Maximum Plasma Concentration [Cmax] of ANAVEX2-73 [ Time Frame: 7 weeks ]
    PK of ANAVEX2-73 and metabolite

  3. Area Under the Curve [AUC] of ANAVEX2-73 [ Time Frame: 7 weeks ]
    PK of ANAVEX2-73 and metabolite

  4. Lipid panel [ Time Frame: 7 weeks ]
    Significant laboratory findings


Secondary Outcome Measures :
  1. RSBQ [ Time Frame: 7 weeks ]
    Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ)

  2. CGI-I [ Time Frame: 7 weeks ]
    Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score


Other Outcome Measures:
  1. Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: 7 weeks ]
    Anxiety, Depression, and Mood Scale (ADAMS)

  2. Children's Sleep Habits Questionnaire (CSHQ) [ Time Frame: 7 weeks ]
    Children's Sleep Habits Questionnaire (CSHQ)

  3. Seizure Frequency via seizure diary [ Time Frame: 7 weeks ]
    Seizure Frequency via seizure diary

  4. Genetic variant SIGMAR1, COMT [ Time Frame: 7 weeks ]
    Genetic variant SIGMAR1, COMT

  5. Glutamate Plasma Concentration [ Time Frame: 7 weeks ]
    Glutamate Plasma Concentration

  6. GABA Plasma Concentration [ Time Frame: 7 weeks ]
    GABA Plasma Concentration



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03941444


Contacts
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Contact: Walter Kaufmann, MD 844-689-3939 rett@anavex.com

Locations
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Australia, Victoria
Royal Melbourne Hospital (RMH) Recruiting
Melbourne, Victoria, Australia, 3050
Contact: Patrick Kwan, MD         
Principal Investigator: Patrick Kwan, MD         
The Alfred Hospital Recruiting
Melbourne, Victoria, Australia, 3181
Contact: Terry O'Brien, MD         
Principal Investigator: Terry O'Brien, MD         
Sponsors and Collaborators
Anavex Life Sciences Corp.

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Responsible Party: Anavex Life Sciences Corp.
ClinicalTrials.gov Identifier: NCT03941444     History of Changes
Other Study ID Numbers: ANAVEX2-73-RS-002
First Posted: May 8, 2019    Key Record Dates
Last Update Posted: May 9, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Rett Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Pharmaceutical Solutions