Study of ANAVEX2-73 in Patients With Rett Syndrome The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. 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 Go to sections 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 Go to sections 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 Go to sections 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 Go to sections 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
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)
Glutamate Plasma Concentration [ Time Frame: 7 weeks ] Biomarker
GABA Plasma Concentration [ Time Frame: 7 weeks ] Biomarker
Eligibility Criteria Go to sections 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 Go to sections 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 rett@anavex.com
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 Go to sections
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
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