A study to evaluate safety and PK of SFX-01 tablets in healthy volunteers
Research type
Research Study
Full title
A Phase 1, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Repeated Daily Doses of SFX-01 Tablets in Healthy Male Participants
IRAS ID
1005951
Contact name
Gabrielle Brill
Contact email
Sponsor organisation
Evgen Pharma plc
Eudract number
2022-001601-43
ISRCTN Number
ISRCTN96285652
Research summary
Research Summary
The purpose of this study is to investigate the study drug SFX-01 (active compound sulforaphane - SFN). The main objectives of the study are as follows:
- To determine the safety and tolerability (degree to which side effects of a drug can be tolerated) of SFN (delivered in the form of SFX-01 tablets) when it is administered as single and multiple doses at different dose strengths over a period of 7 days.
- To investigate the concentration of SFN (from SFX-01) and its’ metabolites in the blood and urine, how this changes over a period of time and to evaluate whether there are differences in the concentrations when different dose strengths are given. Metabolites are by-products which are produced when a drug is broken down in the body.
- To investigate the effect of SFN (from SFX-01) on the body (known as pharmacodynamics (PD) by analysing the effect of SFN (from SFX-01) on the levels of specific biomarkers in the body. Biomarkers are markers within the body such as a gene, molecule or characteristic which can be used to identify the presence of a particular biological process occurring in the body or a particular disease.
The study will consist of 4 planned groups of up to 8 participants (with the option to include additional participants/groups up to a maximum of 56 participants overall). Each group will first receive a single dose of the study drug (planned as either 300 milligrams (mg) or 600 mg) once or twice per day (dependent on study group) before taking SFX-01 once or twice daily for a further 6 days (up to Day 7) .The study will consist of a screening visit (between 36 and 9 days prior to first dose), a pre-dose skin biopsy visit (Day -8), 1 treatment period (consisting of 9 days with 8 overnight stays) and a post-study follow-up visit on Day 14.
Summary of Results
The purpose of this study was to investigate the study drug SFX-01 active compound sulforaphane – SFN). The main objectives of this study were as follows:
To determine the safety and tolerability (degree to which side effects of a drug can be tolerated) of SFN (delivered in the form of SFX-01 enteric tablets) when administered as single and multiple doses at different dose strengths over a period of 7 days to healthy male volunteers.
To investigate the concentration of SFN (from SFX-01 tablets) and its’ metabolites in the blood and urine, how this changed over the 7 days of dosing and to evaluate whether there were differences in the concentrations when different dose strengths were given. Metabolites are by-products which are produced when a drug is broken down in the body.
To investigate the effect of SFN (from SFX-01 tablets) on the body (known as pharmacodynamics (PD) by analysing the effect of SFX-01 on the levels of specific biomarkers in the body. Biomarkers are markers within the body such as a gene, molecule or characteristics which can be used to identify the presence of a particular biological process occurring in the body or a particular disease.
The study intended to enrol up to 32 healthy male participants within 4 dosing groups; however only 24 participants were enrolled with completion of 3 dosing groups. Following review of all of the data generated in the previous 3 groups, the study Sponsor determined that conduct of dosing group 4 was not required and therefore, only 3 of the 4 planned groups completed the study. Blood and urine samples were taken at set time points throughout the study in order to measure the concentration of SFN (from SFX-01 tablets) and its’ metabolites in the blood and urine. The results from each of the groups have been analysed to determine if there are any significant differences in the safety profile of SFX-01, the concentration of SFN (from SFX-01) and its’ metabolites in the blood and urine and to determine whether there are any differences between different dose strengths, single and multiple doses or taking the drug once versus twice per day.
The purpose of the data generated in this study was to provide further information and guidance to support the study Sponsor in development of the study drug.
With respect to the safety objectives of the study, it was determined that SFX-01 was considered to be safe and well tolerated at all dose levels evaluated (300 mg once daily, 300 mg twice daily and 600 mg once daily for 7 days). Of the side effects (adverse events) reported, all were considered to be related to the study drug and were aligned to similar types of effects reported for the study drug in previous clinical trials and the known action/expected effects for SFN (the active compound in SFX-01). The majority of adverse events reported were considered to be mild in severity and resolved before the study completed.
With respect to the other objectives of the study, (namely related to the levels of the study drug in the blood and urine), the following outcomes were reported:
Following administration of SFX-01 tablets at all dose levels, it was identified that sulforaphane (SFN) was rapidly absorbed, broken down (metabolised) and removed (excreted) from the body.
With respect to the metabolite products of SFN, the levels of the metabolites in the blood were higher than the levels of SFN.
The time taken for the levels of SFN and metabolites to reach their highest concentration was consistent with the expected profile for this type of enteric coated tablet form of administration and there was minimal evidence to show that following multiple dose administration, the drug accumulated in the body.
With respect to the levels of SFN and metabolites in the urine, it was observed that these levels did not appear to be influenced by the dose strength which was administered.
In summary, the data gathered during the study was considered sufficient to meet the objectives of the study and warrant further clinical trials and investigations of the study drug.REC name
Wales REC 1
REC reference
22/WA/0218
Date of REC Opinion
27 Sep 2022
REC opinion
Further Information Favourable Opinion