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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 25 January 2021
Main ID:  NCT03758924
Date of registration: 25/11/2018
Prospective Registration: Yes
Primary sponsor: Anavex Life Sciences Corp.
Public title: Study of ANAVEX2-73 in Patients With Rett Syndrome
Scientific title: A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome
Date of first enrolment: February 28, 2019
Target sample size: 31
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT03758924
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Walter Kaufmann, MD
Address: 
Telephone:
Email:
Affiliation:  Emory University SOM
Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 45 Years
Gender: Female
Health Condition(s) or Problem(s) studied
Rett Syndrome
Intervention(s)
Drug: ANAVEX2-73
Drug: Placebo
Primary Outcome(s)
Area Under the Curve [AUC] of ANAVEX2-73 [Time Frame: 7 weeks]
Incidence of Adverse Events [Time Frame: 7 weeks]
Lipid panel [Time Frame: 7 weeks]
Maximum Plasma Concentration [Cmax] of ANAVEX2-73 [Time Frame: 7 weeks]
Secondary Outcome(s)
CGI-I [Time Frame: 7 weeks]
RSBQ [Time Frame: 7 weeks]
Secondary ID(s)
ANAVEX2-73-RS-001
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
International Rett Syndrome Foundation Rettsyndrome.org
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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