Safety, Tolerability and PK of CORT113176 in Healthy Subjects
Research type
Research Study
Full title
A Phase 1 Adaptive Dose, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Orally Administered CORT113176 in Healthy Subjects, with an optional Pharmacological Effects Cohort
IRAS ID
271965
Contact name
Hazel Hunt
Contact email
Sponsor organisation
Corcept Therapeutics
Eudract number
2019-004258-27
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 8 months, 4 days
Research summary
Research Summary
The Sponsor is developing the test medicine, CORT113176, for the potential treatment of amyotrophic lateral sclerosis (ALS). ALS is characterised by progressive degeneration of motor neurons in the brain and spinal cord, leading to muscle weakness and difficulty walking, speaking and swallowing.
The study will try to identify the safety and tolerability of single (Part 1) and multiple (Part 2) ascending doses of the test medicine. It will also try to identify how the test medicine is taken up by the body (pharmacokinetics). Optional Part 3 may assess what the test medicine does to the body (pharmacodynamic effect).
The study will consist of up to 3 parts, involving up to 122 healthy male and female [of non-childbearing potential] volunteers.
In Part 1, up to 64 healthy volunteers in up to 8 cohorts will receive a single dose of the test medicine or matching placebo. Volunteers will remain in clinic until 72-hours post-dose. They will return 96-hours post-dose for a final PK sample. A follow-up visit will take place on approximately Day 7.
In Part 2, up to 48 healthy volunteers in up to 4 cohorts will receive multiple doses of the test medicine or matching placebo for 14 days. Volunteers will remain in clinic until 48-hours post-final dose (Day 16). They will return 72-hours and 96-hours post-final dose for PK samples and safety assessments. A follow-up visit will take place approximately 7 days post-final dose.
Part 3 consists of two periods of up to 10 volunteers in a single cohort, where they will receive one dose of the test medicine to investigate its effects on two doses of a marketed medicine. In period 1, volunteers will remain in clinic until 24-hours post-dose. In period 2 they will remain until 72-hours post-dose. A follow-up visit will take place on approximately Day 7.
Summary of Results
Quotient Sciences (a clinical research organisation) conducted the study in Nottingham, UK, between 27th January and 15th October 2020.
Corcept Therapeutics sponsored the study. Corcept is a small pharmaceutical company based in the US. We all produce the hormone, cortisol, when stressed. Corcept aims to develop novel medicines for the treatment of diseases in which cortisol is important. CORT113176 is one of Corcept’s new drugs. It is hoped it will be effective in treating amyotrophic lateral sclerosis (ALS), a devastating disease with few treatment options.
This study was the first time that CORT113176 was given to people. It was done to find out if the drug is safe for human use, and to help decide what doses should be used in later studies in patients with ALS. 108 healthy men and women took part. They took capsules of either CORT113176 or placebo by mouth. A placebo is a capsule that looks like the CORT113176 capsule but contains no medicine.
Some volunteers took the study medicine just once. This was safe and well tolerated. Most side effects were mild. Other volunteers took the study medicine for 14 days. The highest dose was associated with side effects including abdominal pain and back pain.
The amount of CORT113176 present in blood samples collected during the study was measured, to find out how well it is absorbed into the blood stream. Three different capsule types were tested to find out which was absorbed best. Two of the capsule types were found to be very similar to each other. The third was not absorbed so well.
Based on the results of this study, the capsule type and doses for a study in patients with ALS have been selected. The successful development of a new drug for this disease would be of great benefit to patients suffering from ALS.REC name
Wales REC 2
REC reference
19/WA/0359
Date of REC Opinion
20 Dec 2019
REC opinion
Further Information Favourable Opinion