SAD, MAD Study of TQS-168 in Healthy Male Volunteers (QSC202400)
Research type
Research Study
Full title
A Randomised, Double-Blind, Placebo-Controlled, Single- and Multiple-, Ascending-Dose Study of the Safety, Tolerability and Pharmacokinetics of TQS-168 in Healthy Male Adults
IRAS ID
300388
Contact name
Jonas Hannestead MD, PhD
Contact email
Sponsor organisation
Tranquis Therapeutics, Inc.
Eudract number
2021-003069-37
Duration of Study in the UK
0 years, 5 months, 26 days
Research summary
Summary of Research
The Sponsor is developing the test medicine, TQS-168, for the potential treatment of motor neurone disease (MND) (also known as amyotrophic lateral sclerosis (ALS)) and Parkinson’s disease. These are neurological diseases where the nerve cells in the brain become damaged, impacting the movement of the body.The study will assess the safety and tolerability of single (Part 1) and multiple (Part 2) ascending doses of the test medicine. It will also try to identify what the body does to the test medicine (pharmacokinetics, PK) and the effect of food on the PK. Optional Part 3 may assess the taste attributes of the test medicine.
The study will consist of up to 3 parts involving healthy male volunteers.
In Part 1, 48 volunteers in 6 cohorts will receive a single dose of the test medicine or placebo. In Cohort 3, volunteers may receive an additional two doses of the test medicine or placebo (dummy medicine) on two more occasions. Volunteers will remain in the clinical unit until 48 hours post-[final] dose. A follow-up visit will take place 2 – 6 days after discharge.
In Part 2, up to 30 volunteers in up to 3 cohorts will receive multiple doses of the test medicine or placebo, for 7 days. Volunteers may be dosed once or twice daily. Volunteers will remain in the clinical unit until 48 hours post-final dose. A follow-up visit will take place 3 – 7 days after discharge.
Part 3 may enroll a single cohort of 9 volunteers who will complete three taste tests, using two test medicine formulations (recipes) and a placebo. Volunteers will remain in the clinical unit until 24 hours post-final taste test. A follow-up phone call will be carried out 2 – 4 days after discharge.
Summay of Results
We thank the people who participated in this first-in-human study of TQS-168, A Randomised, Double-Blind, Placebo-Controlled, Single- and Multiple-, Ascending-Dose Study of the Safety, Tolerability and Pharmacokinetics of TQS-168 in Healthy Male Adults. The study was sponsored by Tranquis Therapeutics, Inc. and was conducted at Quotient Sciences in Nottingham, UK in September 2021 to June 2022.The Phase 1 first-in-human study was conducted to evaluate the safety, tolerability, and blood levels of TQS-168, a new molecule being developed for the treatment of neurological disorders such as amyotrophic lateral sclerosis and Parkinson’s disease. First-in-human studies provide information on acceptable doses of the study medicine and what side effects can be expected. The subjects who participated in this study were males between 19 and 51 years of age. Most subjects were Caucasian.
The study had two parts. In Part 1, 47 participants received a single dose of TQS-168 or placebo (dummy medicine) given by mouth (oral dosing). Groups of participants received different doses, and the doses ranged from 60 mg to 540 mg. In Part 2, 30 subjects were given 1 dose of TQS-168 or placebo by mouth every morning for 7 days in a row. The doses in Part 2 ranged from 90 mg per day to 300 mg per day.
No participants experienced adverse events (side effects) of moderate or severe intensity, and there were no serious adverse events. Participants who received TQS-168 experienced mild adverse events which were reversible and tended to occur more frequently at higher dose levels. No participants stopped taking TQS-168 due to side effects. There were no indications that any safety parameters were being affected by TQS-168, including cardiac electrical signals (electrocardiogram), laboratory values measuring liver and kidney function, the number of red and white blood cells and platelets, or blood pressure and heart rate.
TQS-168 was present in the blood stream after oral dosing indicating that taking TQS-168 by mouth is an acceptable way to give the study medication. The results of this study will help us to design future clinical trials of TQS-168 in patients with neurological and other diseases.
REC name
London - Riverside Research Ethics Committee
REC reference
21/LO/0513
Date of REC Opinion
29 Jul 2021
REC opinion
Further Information Favourable Opinion