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Trial record 3 of 5 for:    Anavex

Study of ANAVEX2-73 in Patients With Rett Syndrome

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ClinicalTrials.gov Identifier: NCT03758924
Recruitment Status : Recruiting
First Posted : November 29, 2018
Last Update Posted : March 8, 2019
Sponsor:
Collaborator:
International Rett Syndrome Foundation Rettsyndrome.org
Information provided by (Responsible Party):
Anavex Life Sciences Corp.

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.


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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 : February 28, 2019
Estimated Primary Completion Date : November 1, 2019
Estimated Study Completion Date : December 30, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

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




Primary Outcome Measures :
  1. Incidence of Adverse Events [ Time Frame: 7 weeks ]
    Incidence of Adverse Events

  2. Maximum Plasma Concentration [Cmax] of ANAVEX2-73 [ Time Frame: 7 weeks ]
    PK of ANAVEX2-73 and metabolite

  3. Area Under the Curve [AUC] of ANAVEX2-73 [ Time Frame: 7 weeks ]
    PK of ANAVEX2-73 and metabolite

  4. Lipid panel [ Time Frame: 7 weeks ]
    Significant laboratory findings


Secondary Outcome Measures :
  1. 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

  2. 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:
  1. Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: 7 weeks ]
    Anxiety, Depression, and Mood Scale (ADAMS)

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

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

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

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

  6. Glutamate Plasma Concentration [ Time Frame: 7 weeks ]
    Biomarker

  7. GABA Plasma Concentration [ Time Frame: 7 weeks ]
    Biomarker



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.


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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.

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
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Contact: Walter Kaufmann, MD 844-689-3939 rett@anavex.com

Locations
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United States, Alabama
UAB | The University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35294
Principal Investigator: Alan Percy, MD         
United States, Illinois
Rush University Medical Center Not yet recruiting
Chicago, Illinois, United States, 60612
Principal Investigator: Peter Heydemann, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
Principal Investigator: Shannon Standridge, DO, MPH         
Sponsors and Collaborators
Anavex Life Sciences Corp.
International Rett Syndrome Foundation Rettsyndrome.org
Investigators
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Principal Investigator: Walter Kaufmann, MD Emory University SOM

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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: March 8, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Syndrome
Rett Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System