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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04304482




Registration number
NCT04304482
Ethics application status
Date submitted
8/03/2020
Date registered
11/03/2020
Date last updated
21/08/2023

Titles & IDs
Public title
ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
Scientific title
ANAVEX2-73-RS-003 is a Phase 2/3, Double-blind, Randomized, Placebo-controlled Safety and Efficacy Study in Pediatric Patients With RTT
Secondary ID [1] 0 0
ANAVEX2-73-RS-003
Universal Trial Number (UTN)
Trial acronym
EXCELLENCE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ANAVEX2-73 oral liquid
Treatment: Drugs - Placebo oral liquid

Experimental: ANAVEX2-73 Active - ANAVEX2-73 liquid oral solution

Placebo comparator: ANAVEX2-73 Placebo - Placebo liquid oral solution


Treatment: Drugs: ANAVEX2-73 oral liquid
Liquid oral solution

Treatment: Drugs: Placebo oral liquid
Liquid oral solution
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
RSBQ
Timepoint [1] 0 0
12 weeks
Primary outcome [2] 0 0
Incidents of Adverse Events
Timepoint [2] 0 0
12 weeks
Secondary outcome [1] 0 0
CGI-I
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Anxiety, Depression, and Mood Scale (ADAMS)
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7)
Timepoint [3] 0 0
12 weeks
Secondary outcome [4] 0 0
Children's Sleep Habits Questionnaire (CSHQ)
Timepoint [4] 0 0
12 weeks
Secondary outcome [5] 0 0
Seizure Frequency via seizure diary
Timepoint [5] 0 0
12 weeks
Secondary outcome [6] 0 0
Incidence of Adverse Events
Timepoint [6] 0 0
12 weeks
Secondary outcome [7] 0 0
RSBQ Emotional Factor-Pediatric (subset of the RSBQ)
Timepoint [7] 0 0
12 weeks
Secondary outcome [8] 0 0
Rett Syndrome Caregiver Inventory Assessment (RTT CIA)
Timepoint [8] 0 0
12 weeks
Secondary outcome [9] 0 0
Child Health Questionnaire-Parent Form 50 (CHQ-PF50)
Timepoint [9] 0 0
12 weeks

Eligibility
Key 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.
Minimum age
5 Years
Maximum age
17 Years
Sex
Females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/07/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/06/2023
Sample size
Target
Accrual to date
Final
92
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Sydney
Recruitment hospital [2] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [3] 0 0
Austin Health - Melbourne
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
4101 - Brisbane
Recruitment postcode(s) [3] 0 0
3084 - Melbourne
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Alberta
Country [2] 0 0
Canada
State/province [2] 0 0
British Columbia
Country [3] 0 0
Canada
State/province [3] 0 0
Ontario
Country [4] 0 0
United Kingdom
State/province [4] 0 0
Edinburgh
Country [5] 0 0
United Kingdom
State/province [5] 0 0
London
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Manchester
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Nottingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Anavex Life Sciences Corp.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Anavex Australia Pty Ltd.
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Commercial sector/industry
Name [2] 0 0
Anavex Germany GmbH
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
ANAVEX2-73-RS-003 is a Phase 2/3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 5-17 years of age with RTT using endpoints including multiple clinical and exploratory molecular and biochemical measures.
Trial website
https://clinicaltrials.gov/study/NCT04304482
Trial related presentations / publications
Heussler HS. Emerging Therapies and challenges for individuals with Angelman syndrome. Curr Opin Psychiatry. 2021 Mar 1;34(2):123-128. doi: 10.1097/YCO.0000000000000674. Erratum In: Curr Opin Psychiatry. 2021 Sep 1;34(5):514. doi: 10.1097/YCO.0000000000000738.
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04304482