In this research we investigate the safety of the new drug prosetin for the treatment of ALS. Furthermore, we measure the level of study drug in your blood after taking multiple doses of prosetin to evaluate how the drug distributes and behaves in the body. This drug research is developed on behalf of ProJenX Inc.
Prosetin
The study drug prosetin is a potent and selective small molecule (MAP4K) inhibitor that has shown to promote motor neuron survival in vitro (in glass, outside living organism). It is thought to maintain function of nerve cells in people with ALS. Prosetin is an investigational drug being developed to potentially treat ALS.
Study design
This study is called a Phase 1 study. The main goal of a Phase 1 study is to see whether a new drug is safe. The study consists of 4 parts (A, B, C, and D). Parts A and B, conducted with healthy volunteers, has been completed. Parts C and D is conducted with ALS patients.
Part C is conducted randomized, double-blind and placebo-controlled. Placebo is an oral solution that looks like prosetin, but has no active ingredients. Researchers use a placebo to see if a research medicine works better or is safer than not taking anything at all. Double-blind means you will be randomized (by chance, like flipping a coin) to either the study drug prosetin or placebo, where neither you nor your doctor know that you receive medication or placebo. You will have a 75% (3 in 4) chance of receiving prosetin and a 25% (1 in 4) chance of receiving placebo. The purpose of the double-blind phase is to compare the effects of the study drug, prosetin, with placebo.
Both the study drug and placebo are fluids that should be taken orally once per day. For Part C, your total study duration is up to 56 days, in which there are up to 7 visits to the hospital. During the dosing period you will take study drug for 14 days. Most visits vary in duration from 1 hour to approximately 8,5 hours. However, the visit at which you will receive study drug for the first time will last for approximately 25 hours, including an overnight stay.
Part D is conducted open-label and is only available if you complete Part C. Open-label means that nobody receives placebo, so you will receive prosetin in this part of the study. For Part D, your total study duration is up to 54 weeks, or a little over a year, in which there are 17 visits to the hospital. During the dosing period you will take the drug for 1 year. Most visits vary in duration from 1 hour to a few hours, but the visit at which you will receive open-label study drug for the first time will last for approximately 8,5 hours.
Can I participate in the study?
The main criteria to participate in this study are:
- Age ≥ 18 years
- Diagnosis of ALS
- Slow Vital Capacity (breathing test) >50% predicted
- Able to swallow liquid in order to digest the study medication
- If you are currently receiving medication for ALS (riluzole or edaravone) on a stable dose for at least 30 days prior to participation
- No participation in another investigation study drug trial within 30 days prior to first dose of study medication
- Able to complete long visit days in the ALS Centre
Note: This list is not exhaustive, but these are the main criteria.
Registration and more information
If you are interested in participating in the PRO-101 study and fit the above profile, please contact one of the participating TRICALS centres below (contact details can be found by clicking on ‘read more’). They can provide you with more information and answer any questions you may have. If you experience difficulty contacting a centre, you may reach out directly to the TRICALS operational team at info@tricals.org for support in this process. More information about the study can be found at clinicaltrials.gov.
