Recently, Mallinckrodt Pharmaceuticals announced the opening of enrollment of their Phase 2 clinical trial of H.P. Acthar Gel in people with ALS. Mallinckrodt’s product has received fast track designation and orphan drug status from the FDA for the treatment of ALS. The clinical trial enrolled its first patients in June 2017, and the company expects to complete the trial and read out results by the end of 2019.
The company believes that H.P. Acthar Gel may be able to trigger changes in the way that the immune cells in a person with ALS activate. Research has been building over the last decade suggesting that ALS has specific neuro-immunologic aspects to it. H.P. Acthar Gel contains the adrenocorticotropic hormone (ACTH), which may signal changes in the way that endogenous corticosteroids in a person’s body act, in turn, modifying immune cell activity. It is important to note that H.P. Acthar Gel has been approved by the FDA as a treatment for disorders other than ALS, including lupus, multiple sclerosis, sarcoidosis, among others.
A previous Phase 1 clinical trial of H.P. Acthar Gel in people with ALS was completed in 2016. That two-month long study enrolled 43 people with ALS, who were broken into four cohorts, each being treated with a different dosing regimen of H.P. Acthar Gel. Based on data from several outcome measures predetermined for the Phase 1 study, Mallinckrodt has chosen 0.2 mL of the treatment daily as the dose to be explored in the Phase 2 study.
The clinical trial, led by principal investigator Roman Bilyk, M.D., has been dubbed “The PENNANT trial” by Mallinchrodt. It aims to enroll 195 people through three enrollment sites currently open in Colorado Springs, Colorado, Tampa, Florida and Dallas, Texas. One of the unique aspects of this clinical trial is that the data used to determine if the trial reaches its primary endpoint – change in ALS Functional Rating Scale (ALSFRS-R) – will be collected via telephone calls. Clinical investigators will collect the ALSFRS-R and other data during intermittent clinical visits through the trial as well during the nine-month long Phase 2 clinical trial.
Two out of every 3 people enrolled will be given the active compound, H.P. Acthar Gel, and the remainder will receive the placebo. Enrollment is limited to only those individuals with ALS that meet certain criteria, such as starting the trial within 24 months of their first ALS symptoms and having a forced vital capacity measure of at least 60% of the predicted value, among others. Researchers involved in this study have also named specific exclusion criteria that should be reviewed by anyone considering this study.
A full description of this clinical trial including contact information can be found online here. Updates on site enrollment status and other changes to the study are monitored by ALS TDI and sent monthly via email to those subscribed to the clinical trial mailing list.
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