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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT04304482
Date of registration: 08/03/2020
Prospective Registration: Yes
Primary sponsor: Anavex Life Sciences Corp.
Public title: ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome EXCELLENCE
Scientific title: A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome
Date of first enrolment: July 1, 2020
Target sample size: 69
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT04304482
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 2/Phase 3
Countries of recruitment
Australia
Contacts
Name:     Walter Kaufmann, MD
Address: 
Telephone: +1(844)-689-3939
Email: rett@anavex.com
Affiliation: 
Key inclusion & exclusion criteria

Inclusion Criteria:

- Aged = 5 years to <18.

- 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.

- 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 (or serum) 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 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.

- Subjects on potent CYP 3A4 and CYP2C19 inhibitors and inducers (e.g., oxcarbazepine).

- Patients with hepatic and renal impairment.



Age minimum: 5 Years
Age maximum: 18 Years
Gender: Female
Health Condition(s) or Problem(s) studied
Rett Syndrome
Intervention(s)
Drug: ANAVEX2-73 oral liquid
Drug: Placebo oral liquid
Primary Outcome(s)
CGI-I [Time Frame: 12 weeks]
RSBQ [Time Frame: 12 weeks]
Secondary Outcome(s)
Anxiety, Depression, and Mood Scale (ADAMS) [Time Frame: 12 weeks]
Children's Sleep Habits Questionnaire (CSHQ) [Time Frame: 12 weeks]
Incidence of Adverse Events [Time Frame: 12 weeks]
Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7) [Time Frame: 12 weeks]
Seizure Frequency via seizure diary [Time Frame: 12 weeks]
Secondary ID(s)
ANAVEX2-73-RS-003
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Anavex Australia Pty Ltd.
Anavex Germany GmbH
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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