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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03941444|
Recruitment Status : Completed
First Posted : May 8, 2019
Last Update Posted : January 27, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Rett Syndrome||Drug: ANAVEX2-73 Drug: Placebo||Phase 3|
This Phase 3 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 to continue a 48-week open label extension.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||33 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||36 participants: 3 PK open-label followed by 33 double-blind, randomized, placebo-controlled|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Actual Primary Completion Date :||September 30, 2021|
|Actual Study Completion Date :||September 30, 2021|
Experimental: Active arm
ANAVEX2-73 liquid oral solution
Liquid oral solution
Placebo Comparator: Placebo arm
Placebo liquid oral solution
Liquid oral solution
- RSBQ [ Time Frame: 7 weeks ]Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
- Incidence of Adverse Events [ Time Frame: 7 weeks ]Incidence of Adverse Events
- CGI-I [ Time Frame: 7 weeks ]Drug exposure-dependent response of the Clinical Global Impression of Improvement Scale (CGI-I) score
- Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: 7 weeks ]Drug exposure-dependent response of the Anxiety, Depression, and Mood Scale (ADAMS)
- 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
- 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
- Lipid panel [ Time Frame: 7 weeks ]Significant laboratory findings
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.
|Ages Eligible for Study:||18 Years to 45 Years (Adult)|
|Sexes Eligible for Study:||Female|
|Accepts Healthy Volunteers:||No|
- 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
- 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.
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
|Australia, New South Wales|
|Greenwich, New South Wales, Australia, 2065|
|Mater Misericordiae Ltd|
|South Brisbane, Queensland, Australia, 4101|
|Royal Melbourne Hospital (RMH)|
|Melbourne, Victoria, Australia, 3050|
|The Alfred Hospital|
|Melbourne, Victoria, Australia, 3181|
|Australia, Western Australia|
|The Keogh Institute for Medical Research|
|Nedlands, Western Australia, Australia, 6009|
|King's College of London|
|London, UK, United Kingdom, SE5 8AF|
|Manchester CGM, St. Mary's Hospital|
|Manchester, UK, United Kingdom, M13 9WL|
|Responsible Party:||Anavex Life Sciences Corp.|
|Other Study ID Numbers:||
|First Posted:||May 8, 2019 Key Record Dates|
|Last Update Posted:||January 27, 2022|
|Last Verified:||January 2022|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Mental Retardation, X-Linked
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System