Phase 1 study in healthy volunteers of a treatment to improve and/or normalize symptoms and deficits in patients with Fragile X Syndrome (FXS) Not Applicable
Conditions
Interventions
Current intervention as of 21/01/2022:
Part A1: Single Ascending Dose, cross-over design
subjects will be dosed in up to 4 sessions.
Group 1 males - 6 subjects AUT00206 active
2 subjects placebo to match
Group 2 males - 6 subjects AUT00206 active
2 subjects placebo to match
Optional Part A2: Single Ascending Dose. Cross-over design
subjects will be dosed in up to 2 sessions.
Group 1 females - 6 subjects AUT00206 active
2 subjects placebo to match
Optional PART B: Multiple Ascending Dose (7 - 10 days, based on predicted time to steady state)
Group 1 - 6 subjects AUT00206 active
2 placebo
Group 2 - 6 subjects AUT00206 active
2 placebo
Group 3 - 6 subjects AUT00206 active
2 placebo
Group 4 - 6 subjects AUT00206 active
2 placebo
All subjects will be dosed orally. Whether subjects receive either a single dose or 2 single doses (one in the morning and one in the evening) of AUT00206 or placebo, and whether the dose i
2 placebo
All subjects will be dosed orally. Whether subjects receive either a single do
Sponsors
Autifony Therapeutics (United Kingdom)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Current participant inclusion criteria as of 21/01/2022: 1. Healthy male volunteer (Parts A1 and B only) or healthy female volunteer (Part A2 only). 2. Aged 18 - 45 years (at [first] dosing) (Part A and Part B Groups 1 and 2) or aged >18 years and targeted towards those aged >45 years (Part B Groups 3 and 4) 3. A BMI (Quetelet index) in the range 18.0 - 31.0 kg/m² 4. Sufficient intelligence to understand the nature of the trial and any hazards of participating in it. Ability to communicate satisfactorily with the investigator and to participate in, and comply with the requirements of, the entire trial. 5. Willingness to give written consent to participate after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or their delegate. 6. Agree to follow the contraception requirements of the trial as described in the protocol/ICF. 7. Agree not to donate blood or blood products during the study and for up to 3 months after the administration of the trial medication. 8. Willingness to give written consent to have data entered into The Overvolunteering Prevention System (TOPS). Previous participant inclusion criteria: 1. Healthy male volunteer (Parts A1 and B only) or healthy female volunteer (Part A2 only). 2. Aged 18 - 45 years (at [first] dosing). 3. A BMI (Quetelet index) in the range 18.0 - 31.0 kg/m² 4. Sufficient intelligence to understand the nature of the trial and any hazards of participating in it. Ability to communicate satisfactorily with the investigator and to participate in, and comply with the requirements of, the entire trial. 5. Willingness to give written consent to participate after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or their delegate. 6. Agree to follow the contraception requirements of the trial as described in the protocol/ICF. 7. Agree not to donate blood or blood products during the study and for up to 3 months after the administration of the trial medication. 8. Willingness to give written consent to have data entered into The Overvolunteering Prevention System (TOPS).
Exclusion criteria
Exclusion criteria: 1. Woman who is pregnant or lactating, or pre-menopausal woman who is sexually active and not using a highly-effective method of contraception. 2. Clinically relevant abnormal history, physical findings, ECG, or laboratory values at the pre-trial screening assessment that could interfere with the objectives of the trial or the safety of the volunteer. 3. Presence of acute or chronic illness or history of chronic illness sufficient to invalidate the volunteer’s participation in the trial or make it unnecessarily hazardous. 4. Impaired endocrine, thyroid, hepatic, respiratory or renal function, diabetes mellitus, coronary heart disease, post-COVID-19 syndrome (‘long COVID’), or history of any psychotic mental illness. 5. Surgery (eg stomach bypass) or medical condition that might affect absorption of medicines. 6. Presence or history of severe adverse reaction to any drug, or a history of sensitivity to AUT00206 or its excipients. 7. Use of a prescription medicine (except oral contraceptives in females) during the 30 days before the first dose of trial medication, or use of an over-the-counter medicine, with the exception of acetaminophen (paracetamol), during the 7 days before the first dose of trial medication. 8. Use of vaccine (including COVID-19 vaccine) during the 7 days, before first receipt of trial medication until the end of the study. 9. History of epilepsy or seizures. 10. Receipt of an investigational product (including prescription medicines) as part of another clinical trial within the 3 months before [first] admission to this study; in the follow-up period of another clinical trial at the time of screening for this study. 11. Presence or history of drug or alcohol abuse, or intake of more than 14 units of alcohol weekly. 12. Smoking of more than 5 cigarettes daily. 13. Blood pressure and heart rate in supine position at the screening examination outside the ranges: blood pressure 90–140 mm Hg systolic, 40–90 mm Hg diastolic; heart rate 40–100 beats/min. 14. QT value, measured at screening visit, greater than 450 msec (men) or 470 msec (women) on 12-lead ECG, using Fridericia’s formula (QTcF) for correction. 15. Alanine aminotransferase (AST), aspartate aminotransferase (ALT) or alkaline phosphatase (AP) = 1.5 x the upper limit of normal (ULN). A repeat is allowed on one occasion for determination of eligibility. 16. Possibility that the volunteer will not cooperate with the requirements of the protocol. 17. Positive test for hepatitis B, hepatitis C or HIV consistent with ongoing infection. 18. Vegans or people who are on a restricted diet for medical reasons (eg lactose intolerant); or unwilling to eat a high-fat breakfast (Part A1 and A2 only). 19. Positive result for suicidal ideation or behaviour using the C-SSRS, including a positive response to items 3 to 5; or a history of suicidal behaviour in the past year 20. Loss of more than 400 mL blood during the 3 months before the trial, eg as a blood donor. 21. Medical objection by General Practitioner (GP) to volunteer entering trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A1: 1.1 Pharmacokinetic (PK) profile of AUT00206 after single oral doses of AUT00206 in healthy men measured from blood samples taken before and frequently after dosing up to 192h 1.2 Effect of food on the bioavailability of single oral doses of AUT00206 in healthy men measured from blood samples taken before and frequently after dosing Part A2 (optional): 2. PK profile of AUT00206 after single oral doses of AUT00206 in healthy women measured from blood samples taken before and frequently after dosing up to 192h Part B (optional): 3. PK profile of AUT00206 after repeated oral doses of AUT00206 in healthy men measured from blood samples taken before and frequently after dosing up to 192h | — |
Secondary
| Measure | Time frame |
|---|---|
| Part A1: 1. Safety and tolerability of single oral doses of AUT00206 in healthy men measured by collecting vital signs (blood pressure, heart rate, tympanic temperature, and respiratory rate), 12-lead electrocardiogram (ECG)s, physical examinations, and laboratory safety tests (haematology, clinical chemistry, thyroid function, and urinalysis), at frequent intervals; the Columbia-Suicide Severity Rating Scale (C-SSRS) will be conducted at Screening and follow-up, and collecting reports of adverse events (AEs) throughout the study Part A2 (optional): 2. Safety and tolerability of single oral doses of AUT00206 in healthy women measured by collecting vital signs (blood pressure, heart rate, tympanic temperature, and respiratory rate), 12-lead electrocardiogram (ECG)s, physical examinations, and laboratory safety tests (haematology, clinical chemistry, thyroid function, and urinalysis), at frequent intervals; the Columbia-Suicide Severity Rating Scale (C-SSRS) will be conducted at Screening and follow-up, and collecting reports of adverse events (AEs) throughout the study Part B (optional): 3. To assess the safety and tolerability of repeated oral doses of AUT00206 in healthy men measured by collecting vital signs (blood pressure, heart rate, tympanic temperature, and respiratory rate), 12-lead electrocardiogram (ECG)s, physical examinations, and laboratory safety tests (haematology, clinical chemistry, thyroid function, and urinalysis), at frequent intervals; the Columbia-Suicide Severity Rating Scale (C-SSRS) will be conducted at Screening and follow-up, and collecting reports of adverse events (AEs) throughout the study | — |
Countries
England, United Kingdom
Outcome results
None listed