Anavex Life Sciences Corp (AVXL)

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Study of ANAVEX2-73 in Patients With Rett Syndrome
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ClinicalTrials.gov Identifier: NCT03758924
Recruitment Status : Not yet recruiting
First Posted : November 29, 2018
Last Update Posted : December 7, 2018
See Contacts and Locations
Sponsor:
Anavex Life Sciences Corp.
Collaborator:
International Rett Syndrome Foundation Rettsyndrome.org
Information provided by (Responsible Party):
Anavex Life Sciences Corp.

Study Details Tabular ViewNo Results PostedDisclaimerHow to Read a Study Record
Study Description
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Brief 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. 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.


Condition or disease Intervention/treatment Phase
Rett Syndrome
Drug: ANAVEX2-73
Drug: Placebo
Phase 2

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
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Study Type : Interventional (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Double-Blind, Randomized, Placebo-Controlled
Primary Purpose: Treatment
Official Title: A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome
Estimated Study Start Date : December 2018
Estimated Primary Completion Date : October 1, 2019
Estimated Study Completion Date : November 30, 2019
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics: Rett syndrome
MedlinePlus related topics: Rett Syndrome
Genetic and Rare Diseases Information Center resources: Rett Syndrome
U.S. FDA Resources

Arms and Interventions
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Arm Intervention/treatment
Experimental: Active arm
Week 0-7: Take 1 ml orally of the product daily (solution of ANAVEX2-73)
Drug: ANAVEX2-73
Liquid oral solution

Placebo Comparator: Placebo arm
Week 0-7: Take 1 ml orally of the product daily (placebo)
Drug: Placebo
Liquid oral solution



Outcome Measures
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Primary Outcome Measures :
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


Secondary Outcome Measures :
RSBQ [ Time Frame: 7 weeks ]
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

CGI-I [ Time Frame: 7 weeks ]
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


Other Outcome Measures:
Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: 7 weeks ]
Anxiety, Depression, and Mood Scale (ADAMS)

Children's Sleep Habits Questionnaire (CSHQ) [ Time Frame: 7 weeks ]
Children's Sleep Habits Questionnaire (CSHQ)

Visual Analog Scale (VAS) [ Time Frame: 7 weeks ]
Visual Analog Scale (VAS)

Seizure Frequency via seizure diary [ Time Frame: 7 weeks ]
Seizure Frequency via seizure diary

Genetic variant SIGMAR1, COMT [ Time Frame: 7 weeks ]
Pre-specified endpoint

Glutamate Plasma Concentration [ Time Frame: 7 weeks ]
Biomarker

GABA Plasma Concentration [ Time Frame: 7 weeks ]
Biomarker


Eligibility Criteria
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Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study: 18 Years to 45 Years (Adult)
Sexes Eligible for Study: Female
Accepts Healthy Volunteers: No
Criteria
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.
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.
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 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.
Contacts and Locations
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Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03758924


Contacts
Contact: Walter Kaufmann, MD 844-689-3939 [e-mail]

Locations
United States, Alabama
UAB | The University of Alabama at Birmingham Not yet recruiting
Birmingham, Alabama, United States, 35294
Principal Investigator: Alan Percy, MD
Sponsors and Collaborators
Anavex Life Sciences Corp.
International Rett Syndrome Foundation Rettsyndrome.org
Investigators
Principal Investigator: Walter Kaufmann, MD Emory University SOM
More Information
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Responsible Party: Anavex Life Sciences Corp.
ClinicalTrials.gov Identifier: NCT03758924 History of Changes
Other Study ID Numbers: ANAVEX2-73-RS-001
First Posted: November 29, 2018 Key Record Dates
Last Update Posted: December 7, 2018
Last Verified: December 2018

https://clinicaltrials.gov/ct2/show/NCT03758924

https://www.childrensal.org/dr-alan-k-percy-md-neurology

http://genetics.emory.edu/faculty/adjunct/kaufmann-walter.html

Good luck and GOD bless,

George