The use of ANAVEX2-73 as a new therapeutic treatment for Patients with Rett Syndrome

Phase 1

• Conditions: Rett Syndrome; MedDRA version: 20.0Level: PTClassification code 10077709Term: Rett syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2019-004345-32-GB
• Lead Sponsor: Anavex Germany GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-02-07
• Last Update Posted: 30 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Female
• Target Recruitment: 30

Inclusion Criteria

1.Aged = 18 years, inclusive.
2. Diagnosis of classic Rett Syndrome, according to 2010 criteria (Neul et al., 2010), and a MECP2 mutation.
3. Rett Syndrome Behaviour Questionnaire (RSBQ) total score =40.
4. Patients with a Clinical Global Impression – Severity (CGI-S) score of 4 or greater at Screening.
5. Current pharmacological treatment regimen, including supplements, has been stable for at least 4 weeks.
6. If on antiepileptic drugs (AEDs), 1-4 AEDs allowed. Treatment must be stable (drug, dose, interval of administration) for 30 days prior to enrollment.
7. 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/legally authorized representative (LAR) will not electively initiate new or modify ongoing interventions for the duration of the study. ‘Study duration’ is defined as lasting from the screening visit until the treatment is terminated. For participants in the 18-21 years range, typical school vacations are not considered modifications of stable programming.
8. Ability to keep accurate seizure diaries or have caregiver who can keep accurate seizure diaries.
9.If participant is a woman of childbearing potential, a negative urine or serum pregnancy test is required to confirm she is not pregnant.
Female patients of childbearing potential are to use adequate contraception as recommended by their health care provider. For participants that are sexually active, highly effective contraception may be used as recommended by their health care provider.Highly effective methods of contraception may include:
•Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: Oral, intravaginal or transdermal
•Progestogen-only hormonal contraception associated with inhibition of ovulation: Oral, injectable, implantable
•Intrauterine device
•Intrauterine hormone-releasing system
•Bilateral tubal occlusion
10. 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.

Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1. Patients who have a progressive medical or neurological condition that in the opinion of the Investigator would interfere with the conduct of the study.
2. Patients with a 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.
3. 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.
4. 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.
5. 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.
6. 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.
7. Any known hypersensitivity to any of the excipients contained in the study drug or placebo formulation.
8. Other co-morbid or chronic illness beyond that known to be associated with Rett Syndrome.
9. Subjects who plan to initiate or change pharmacologic or nonpharmacologic intervention during the course of the study.
10. Subjects taking another investigational drug currently or within the last 30 days.
11. 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.
12. 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.
13. Patients with hepatic and renal impairment.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified