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Rett SyndromePhase 2/3EXCELLENCENCT04304482

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome

A Phase 2/3 study testing ANAVEX2-73 oral liquid in people with Rett Syndrome, run by Anavex Life Sciences Corp. at 12 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 5 to 17 years, women only
Conditions the study is for
Rett Syndrome
What is being tested?
ANAVEX2-73 oral liquid
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
92 participants
Where
12 sitesin 3 countries
Study dates
1 Jul 2020to 30 Jun 2023
Phase
Phase 2/3
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 5 to 17 years
  • Women only
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
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  • 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.

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 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.
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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.
  • 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.

Trial locations and contacts

12 sites in 3 countries.

12 sites in 3 countries

United Kingdom5 sites

  • Royal Hospital for Children

    Edinburgh, EH16 4TJ

  • Evelina London Children's Hospital

    London, SE1 7EH

  • King's College of London

    London, SE5 8AF

  • Manchester CGM, St Mary's Hospital

    Manchester, M13 9WL

  • Nottingham University Hospital NHS Trust

    Nottingham, NG7 2UH

Other trials in United Kingdom

Canada4 sites

  • Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8

  • Children's Hospital LHSC

    London, Ontario, N6A 5W9

  • Holland Bloorview Kids Hospital

    Toronto, Ontario, M5H 3W4

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3V4

Other trials in Canada

Australia3 sites

  • Queensland Children's Hospital

    Brisbane, Queensland, 4101

  • Austin Health

    Melbourne, Victoria, 3084

  • The Children's Hospital at Westmead

    Sydney, New South Wales, 2145

Other trials in Australia

About this study

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

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.

Read the 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.

Conditions
Rett Syndrome
Being tested
ANAVEX2-73 oral liquid, Placebo oral liquid
Medical subject headings
Rett Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ANAVEX2-73 oral liquidDrug

    Also called Blarcamesine

    Liquid oral solution

  • Placebo oral liquidDrug

    Liquid oral solution

Study groups in NCT04304482
Study groupRoleWhat they receive
ANAVEX2-73 ActiveExperimentalANAVEX2-73 liquid oral solution
Receives: Drug: ANAVEX2-73 oral liquid
ANAVEX2-73 PlaceboPlacebo comparatorPlacebo liquid oral solution
Receives: Drug: Placebo oral liquid

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. RSBQ

    Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score

    Measured: 12 weeks

  2. Incidents of Adverse Events

    Change from baseline to End of Treatment (EOT)

    Measured: 12 weeks

Secondary outcomes (9)

  1. CGI-I

    Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score

    Measured: 12 weeks

  2. Anxiety, Depression, and Mood Scale (ADAMS)

    Anxiety, Depression, and Mood Scale (ADAMS)

    Measured: 12 weeks

  3. Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7)

    Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7)

    Measured: 12 weeks

  4. Children's Sleep Habits Questionnaire (CSHQ)

    Children's Sleep Habits Questionnaire (CSHQ)

    Measured: 12 weeks

  5. Seizure Frequency via seizure diary

    Seizure Frequency via seizure diary

    Measured: 12 weeks

  6. Incidence of Adverse Events

    Incidence of Adverse Events

    Measured: 12 weeks

  7. RSBQ Emotional Factor-Pediatric (subset of the RSBQ)

    RSBQ Emotional Factor-Pediatric (subset of the RSBQ)

    Measured: 12 weeks

  8. Rett Syndrome Caregiver Inventory Assessment (RTT CIA)

    Rett Syndrome Caregiver Inventory Assessment (RTT CIA)

    Measured: 12 weeks

  9. Child Health Questionnaire-Parent Form 50 (CHQ-PF50)

    Child Health Questionnaire-Parent Form 50 (CHQ-PF50)

    Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Mar 2020
  2. Study start1 Jul 2020
  3. Primary completion1 Jun 2023
  4. Study completion30 Jun 2023

Resources

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
92 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04304482
Sponsor's protocol ID
ANAVEX2-73-RS-003
Acronym
EXCELLENCE
Status last verified
Aug 2023
First submitted
8 Mar 2020
Last updated
21 Aug 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

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.

    PubMed 33395098

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing ANAVEX2-73 oral liquid in people with Rett Syndrome, run by Anavex Life Sciences Corp. at 12 sites in 3 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 10 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 21 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United Kingdom, Canada, Australia. 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 combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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: 12 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?

On the ClinicalTrials.gov record for NCT04304482, which also lists 1 linked publication.

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 21 Aug 2023.

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