Rett SyndromePhase 2NCT03758924
Study of ANAVEX2-73 in Patients With Rett Syndrome
A Phase 2 study testing ANAVEX2-73 in people with Rett Syndrome, run by Anavex Life Sciences Corp. at 8 sites in United States.
Trial summary
Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study. 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. 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
- 31 participants
- Where
- 8 sitesUnited States
- Study dates
- 28 Feb 2019to 30 Oct 2020
- Phase
- Phase 2
- 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.
- 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 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 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 on potent CYP 3A4 and CYP2C19 inhibitors and inducers.
- 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.
- Patients with hepatic and renal impairment.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
UAB | The University of Alabama at Birmingham
Birmingham, Alabama, 35294
Boston Children's Hospital
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Greenwood Genetic Center
Greenwood, South Carolina, 29646
Baylor College of Medicine
Houston, Texas, 77030
UC Davis University of California - Davis MIND Institute
Sacramento, California, 95817
Washington University in St. Louis | Saint Louis Children's Hospital
St Louis, Missouri, 63110
About this study
Official title: A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome
Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study.
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 who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.
Read the detailed description
This Phase 2 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 who meet the exposure criteria for ANAVEX2-73 to continue a 12-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 | Week 0-7: Take 1 ml orally of the product daily (solution of ANAVEX2-73) Receives: Drug: ANAVEX2-73 |
| Placebo arm | Placebo comparator | Week 0-7: Take 1 ml orally of the product daily (placebo) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events
Incidence of Adverse Events
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
Lipid panel
Significant laboratory findings
Measured: 7 weeks
Secondary outcomes (2)
RSBQ
Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ). Total score and a pre-specified subset of the RSBQ
Measured: 7 weeks
CGI-I
Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score. Total score and a pre-specified subset of the CGI-I
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 posted29 Nov 2018
- Study start28 Feb 2019
- Primary completion30 Oct 2020
- Study completion30 Oct 2020
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 31 participants: 6 PK open-label followed by 25 double-blind, randomized, placebo-controlled
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Anavex Life Sciences Corp.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- International Rett Syndrome Foundation
- Responsible party
- Sponsor
- Investigators
- Walter Kaufmann, MD, Principal investigator, Emory University SOM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03758924
- Sponsor's protocol ID
- ANAVEX2-73-RS-001
- Status last verified
- Jan 2021
- First submitted
- 25 Nov 2018
- Last updated
- 12 Jan 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome
- ClinicalTrials.gov ID
- NCT03758924
- Acronym / sponsor ID
- ANAVEX2-73-RS-001
- Conditions
- Rett Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Anavex Life Sciences Corp.Industry (pharma, biotech, devices)
- Collaborators
- International Rett Syndrome Foundation
- Study start
- 28 Feb 2019 (actual)
- Primary completion
- 30 Oct 2020 (actual)
- Study completion
- 30 Oct 2020 (actual)
- First posted
- 29 Nov 2018 (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.
Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study.
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 who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.
Detailed description
This Phase 2 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 who meet the exposure criteria for ANAVEX2-73 to continue a 12-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
- 31 participants: 6 PK open-label followed by 25 double-blind, randomized, placebo-controlled
| Arm | Type | Receives |
|---|---|---|
| Active arm | Experimental | Drug: ANAVEX2-73Week 0-7: Take 1 ml orally of the product daily (solution of ANAVEX2-73) |
| Placebo arm | Placebo comparator | Drug: PlaceboWeek 0-7: Take 1 ml orally of the product daily (placebo) |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 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.
- Ability to keep accurate seizure diaries or have caregiver who can keep accurate seizure diaries.
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
Incidence of Adverse Events
Time frame: 7 weeks
Incidence of Adverse Events
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
Time frame: 7 weeks
PK of ANAVEX2-73 and metabolite
Area Under the Curve [AUC] of ANAVEX2-73
Time frame: 7 weeks
PK of ANAVEX2-73 and metabolite
Lipid panel
Time frame: 7 weeks
Significant laboratory findings
2 secondary outcomes
RSBQ
Time frame: 7 weeks
CGI-I
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 12 Jan 2021.
- Individual participant dataSharing planned: No
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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.
Walter Kaufmann, MD principal investigator
Emory University SOM
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ANAVEX2-73 in people with Rett Syndrome, run by Anavex Life Sciences Corp. at 8 sites in United States.
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 7 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 12 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Incidence of Adverse Events (time frame: 7 weeks). The study lists 4 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 12 Jan 2021.