Brief Title
ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
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
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.
Detailed Description
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 Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
RSBQ
Secondary Outcome
Anxiety, Depression, and Mood Scale (ADAMS)
Condition
Rett Syndrome
Intervention
ANAVEX2-73 oral liquid
Study Arms / Comparison Groups
ANAVEX2-73 Active
Description: ANAVEX2-73 liquid oral solution
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
84
Start Date
July 1, 2020
Completion Date
December 1, 2022
Primary Completion Date
November 1, 2022
Eligibility Criteria
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.
Gender
Female
Ages
5 Years - 17 Years
Accepts Healthy Volunteers
No
Contacts
, +1(844)-689-3939, [email protected]
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT04304482
Organization ID
ANAVEX2-73-RS-003
Responsible Party
Sponsor
Study Sponsor
Anavex Life Sciences Corp.
Collaborators
Anavex Australia Pty Ltd.
Study Sponsor
, ,
Verification Date
May 2022