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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03941444




Registration number
NCT03941444
Ethics application status
Date submitted
6/05/2019
Date registered
8/05/2019
Date last updated
27/01/2022

Titles & IDs
Public title
ANAVEX2-73 Study in Patients With Rett Syndrome
Scientific title
A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome
Secondary ID [1] 0 0
ANAVEX2-73-RS-002
Universal Trial Number (UTN)
Trial acronym
AVATAR
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
Treatment: Drugs - Placebo

Experimental: Active arm - ANAVEX2-73 liquid oral solution

Placebo Comparator: Placebo arm - Placebo liquid oral solution


Treatment: Drugs: ANAVEX2-73
Liquid oral solution

Treatment: Drugs: Placebo
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
7 weeks
Primary outcome [2] 0 0
Incidence of Adverse Events
Timepoint [2] 0 0
7 weeks
Secondary outcome [1] 0 0
CGI-I
Timepoint [1] 0 0
7 weeks
Secondary outcome [2] 0 0
Anxiety, Depression, and Mood Scale (ADAMS)
Timepoint [2] 0 0
7 weeks
Secondary outcome [3] 0 0
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
Timepoint [3] 0 0
7 weeks
Secondary outcome [4] 0 0
Area Under the Curve [AUC] of ANAVEX2-73
Timepoint [4] 0 0
7 weeks

Eligibility
Key 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
Minimum age
18 Years
Maximum age
45 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 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.

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 3
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
6/05/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/09/2021
Sample size
Target
Accrual to date
Final
33
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
HammondCare - Greenwich
Recruitment hospital [2] 0 0
Mater Misericordiae Ltd - South Brisbane
Recruitment hospital [3] 0 0
Royal Melbourne Hospital (RMH) - Melbourne
Recruitment hospital [4] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [5] 0 0
The Keogh Institute for Medical Research - Nedlands
Recruitment postcode(s) [1] 0 0
2065 - Greenwich
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3050 - Melbourne
Recruitment postcode(s) [4] 0 0
3181 - Melbourne
Recruitment postcode(s) [5] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United Kingdom
State/province [1] 0 0
UK

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Anavex Life Sciences Corp.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
ANAVEX2-73-RS-002 is a Phase 3, 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03941444
Trial related presentations / publications
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/ct2/show/NCT03941444