ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome (EXCELLENCE)
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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: NCT04304482 |
Recruitment Status :
Active, not recruiting
First Posted : March 11, 2020
Last Update Posted : February 8, 2023
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Rett Syndrome | Drug: ANAVEX2-73 oral liquid Drug: Placebo oral liquid | Phase 2 Phase 3 |
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 5-17 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 Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 92 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | ANAVEX2-73-RS-003 is a Phase 2/3, Double-blind, Randomized, Placebo-controlled Safety and Efficacy Study in Pediatric Patients With RTT |
Actual Study Start Date : | July 1, 2020 |
Estimated Primary Completion Date : | April 30, 2023 |
Estimated Study Completion Date : | June 1, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: ANAVEX2-73 Active
ANAVEX2-73 liquid oral solution
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Drug: ANAVEX2-73 oral liquid
Liquid oral solution
Other Name: Blarcamesine |
Placebo Comparator: ANAVEX2-73 Placebo
Placebo liquid oral solution
|
Drug: Placebo oral liquid
Liquid oral solution |
- RSBQ [ Time Frame: 12 weeks ]Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
- Incidents of Adverse Events [ Time Frame: 12 weeks ]Change from baseline to End of Treatment (EOT)
- CGI-I [ Time Frame: 12 weeks ]Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score
- Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: 12 weeks ]Anxiety, Depression, and Mood Scale (ADAMS)
- Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7) [ Time Frame: 12 weeks ]Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7)
- Children's Sleep Habits Questionnaire (CSHQ) [ Time Frame: 12 weeks ]Children's Sleep Habits Questionnaire (CSHQ)
- Seizure Frequency via seizure diary [ Time Frame: 12 weeks ]Seizure Frequency via seizure diary
- Incidence of Adverse Events [ Time Frame: 12 weeks ]Incidence of Adverse Events
- RSBQ Emotional Factor-Pediatric (subset of the RSBQ) [ Time Frame: 12 weeks ]RSBQ Emotional Factor-Pediatric (subset of the RSBQ)
- Rett Syndrome Caregiver Inventory Assessment (RTT CIA) [ Time Frame: 12 weeks ]Rett Syndrome Caregiver Inventory Assessment (RTT CIA)
- Child Health Questionnaire-Parent Form 50 (CHQ-PF50) [ Time Frame: 12 weeks ]Child Health Questionnaire-Parent Form 50 (CHQ-PF50)
- Glutamate Plasma Concentration [ Time Frame: 12 weeks ]Glutamate Plasma Concentration
- GABA Plasma Concentration [ Time Frame: 12 weeks ]GABA Plasma Concentration
- Genetic variant SIGMAR1, COMT [ Time Frame: 12 weeks ]Genetic variant SIGMAR1, COMT
- Maximum Plasma Concentration [Cmax] [ Time Frame: 12 weeks ]Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
- Maximum Plasma Concentration [Cmax] relationship with RSBQ [ Time Frame: 12 weeks ]Number of participants with positive Maximum Plasma Concentration [Cmax] relationship with RSBQ
- Other Amino Acid Plasma concentrations [ Time Frame: 12 weeks ]Other Amino Acid Plasma concentrations
- Measure of gene DNA variants and gene RNA expressions [ Time Frame: 12 weeks ]Number of participants with active dose compared gene DNA variants and gene RNA expressions

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: | 5 Years to 17 Years (Child) |
Sexes Eligible for Study: | Female |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Aged ≥ 5 years to 17 (inclusive).
- 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.
- The subject's caregiver/LAR is English-speaking and has sufficient language skills to complete the caregiver assessments and has the ability to keep accurate seizure diaries.
- If participant is a woman of childbearing potential (WOCBP#), a negative urine or serum pregnancy test is required to confirm she is not pregnant.
- 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.
- Treatment with strong inhibitors or inducers of CYP3A4 or CYP2C19 is not stable (drug, dose) for 30 days prior to screening. Although these medications are not excluded, caution is advised when enrolling participants on potent CYP3A4 or CYP2C19 inducers or inhibitors (see respective section).
- 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): NCT04304482
Australia, New South Wales | |
The Children's Hospital at Westmead | |
Sydney, New South Wales, Australia, 2145 | |
Australia, Queensland | |
Queensland Children's Hospital | |
Brisbane, Queensland, Australia, 4101 | |
Australia, Victoria | |
Austin Health | |
Melbourne, Victoria, Australia, 3084 | |
Canada, Alberta | |
Alberta Children's Hospital | |
Calgary, Alberta, Canada, T3B 6A8 | |
Canada, British Columbia | |
British Columbia Children's Hospital | |
Vancouver, British Columbia, Canada, V6H 3V4 | |
Canada, Ontario | |
Children's Hospital LHSC | |
London, Ontario, Canada, N6A 5W9 | |
Holland Bloorview Kids Hospital | |
Toronto, Ontario, Canada, M5H 3W4 | |
United Kingdom | |
Royal Hospital for Children | |
Edinburgh, United Kingdom, EH16 4TJ | |
Evelina London Children's Hospital | |
London, United Kingdom, SE1 7EH | |
King's College of London | |
London, United Kingdom, SE5 8AF | |
Manchester CGM, St Mary's Hospital | |
Manchester, United Kingdom, M13 9WL | |
Nottingham University Hospital NHS Trust | |
Nottingham, United Kingdom, NG7 2UH |
Responsible Party: | Anavex Life Sciences Corp. |
ClinicalTrials.gov Identifier: | NCT04304482 |
Other Study ID Numbers: |
ANAVEX2-73-RS-003 |
First Posted: | March 11, 2020 Key Record Dates |
Last Update Posted: | February 8, 2023 |
Last Verified: | February 2023 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
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 |