Rett SyndromePhase 3AVATARNCT03941444
ANAVEX2-73 Study in Patients With Rett Syndrome
A Phase 3 study testing ANAVEX2-73 in people with Rett Syndrome, run by Anavex Life Sciences Corp. at 7 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- ANAVEX2-73
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 33 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 6 May 2019to 30 Sep 2021
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
Australia5 sites
HammondCare
Greenwich, New South Wales, 2065
Royal Melbourne Hospital (RMH)
Melbourne, Victoria, 3050
The Alfred Hospital
Melbourne, Victoria, 3181
The Keogh Institute for Medical Research
Nedlands, Western Australia, 6009
Mater Misericordiae Ltd
South Brisbane, Queensland, 4101
United Kingdom2 sites
King's College of London
London, UK, SE5 8AF
Manchester CGM, St. Mary's Hospital
Manchester, UK, M13 9WL
About this study
Official title: A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome
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.
Read the detailed description
This Phase 3 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study.
This is a 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients to continue a 48-week open label extension.
- Conditions
- Rett Syndrome
- Being tested
- ANAVEX2-73, Placebo
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANAVEX2-73Drug
Liquid oral solution
PlaceboDrug
Liquid oral solution
| Study group | Role | What they receive |
|---|---|---|
| Active arm | Experimental | ANAVEX2-73 liquid oral solution Receives: Drug: ANAVEX2-73 |
| Placebo arm | Placebo comparator | Placebo liquid oral solution Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
RSBQ
Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
Measured: 7 weeks
Incidence of Adverse Events
Incidence of Adverse Events
Measured: 7 weeks
Secondary outcomes (4)
CGI-I
Drug exposure-dependent response of the Clinical Global Impression of Improvement Scale (CGI-I) score
Measured: 7 weeks
Anxiety, Depression, and Mood Scale (ADAMS)
Drug exposure-dependent response of the Anxiety, Depression, and Mood Scale (ADAMS)
Measured: 7 weeks
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
PK of ANAVEX2-73 and metabolite
Measured: 7 weeks
Area Under the Curve [AUC] of ANAVEX2-73
PK of ANAVEX2-73 and metabolite
Measured: 7 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2019
- Study start6 May 2019
- Primary completion30 Sep 2021
- Study completion30 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 36 participants: 3 PK open-label followed by 33 double-blind, randomized, placebo-controlled
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Anavex Life Sciences Corp.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03941444
- Sponsor's protocol ID
- ANAVEX2-73-RS-002
- Acronym
- AVATAR
- Status last verified
- Jan 2022
- First submitted
- 6 May 2019
- Last updated
- 27 Jan 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome
- ClinicalTrials.gov ID
- NCT03941444
- Acronym / sponsor ID
- AVATAR / ANAVEX2-73-RS-002
- Conditions
- Rett Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Anavex Life Sciences Corp.Industry (pharma, biotech, devices)
- Study start
- 6 May 2019 (actual)
- Primary completion
- 30 Sep 2021 (actual)
- Study completion
- 30 Sep 2021 (actual)
- First posted
- 8 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This Phase 3 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study.
This is a 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients to continue a 48-week open label extension.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- 36 participants: 3 PK open-label followed by 33 double-blind, randomized, placebo-controlled
| Arm | Type | Receives |
|---|---|---|
| Active arm | Experimental | Drug: ANAVEX2-73ANAVEX2-73 liquid oral solution |
| Placebo arm | Placebo comparator | Drug: PlaceboPlacebo liquid oral solution |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ANAVEX2-73Liquid oral solution | Drug | Active arm |
| PlaceboLiquid oral solution | Drug | Placebo arm |
Primary outcome
RSBQ
Time frame: 7 weeks
Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
Incidence of Adverse Events
Time frame: 7 weeks
Incidence of Adverse Events
4 secondary outcomes
CGI-I
Time frame: 7 weeks
Anxiety, Depression, and Mood Scale (ADAMS)
Time frame: 7 weeks
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
Time frame: 7 weeks
Area Under the Curve [AUC] of ANAVEX2-73
Time frame: 7 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ANAVEX2-73 in people with Rett Syndrome, run by Anavex Life Sciences Corp. at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Australia, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
RSBQ (time frame: 7 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jan 2022.