Post hoc analysis using PRO-ACT database to evaluate Repository Corticotropin Injection (H.P. Acthar® Gel) as a potential treatment for ALS (P5.327)

Objective: To explore the effect of repository corticotropin injection (RCI) on functional progression of amyotrophic lateral sclerosis (ALS).

Background: Data from a pilot study of RCI and the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database were used to assess the potential effects of RCI on ALS progression via 2 post hoc analyses: 1) a case-matched control analysis using placebo-treated patients from PRO-ACT and 2) a slope analysis comparing observed ALS progression in the RCI pilot study with disease progression predicted by an algorithm developed using PRO-ACT data.

Design/Methods: During the pilot study, 43 patients with ALS were treated with RCI for up to 36 weeks and the ALS Functional Rating Scale (ALSFRS) assessment was completed monthly. For the case-matched control analysis, RCI-treated patients were paired with up to 3 patients from PRO-ACT using 7 variables associated with disease progression. The prediction algorithm used baseline features to generate a 36-week slope estimate, which was compared with the observed slope for change in ALSFRS score from the RCI pilot study.

Results: At baseline, mean ALSFRS scores were 27.8 (SD, 5.55) in the RCI-treated group and 27.2 (SD, 6.31) in the PRO-ACT control group. In the case-match control analysis, ALSFRS scores at Week 36 significantly declined from baseline by a mean of 4.3 (SD, 4.71) points in the RCI-treated group and by 6.6 (SD, 5.57) points in the control group (P=0.025). For the 21 patients who completed the pilot study, the observed mean absolute value of the slope of change for progression in ALSFRS score(s) (−0.51; SD, 0.57) was smaller than that predicted by the algorithm (−0.75; SD, 0.26), although not statistically significant (P=0.087).

Conclusions: These results suggest potential RCI efficacy in the treatment of ALS and support further study of RCI for ALS in controlled trials.

Disclosure: Dr. Vanmeter has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of Mallinckrodt ARD, Inc. Dr. Vanmeter has received compensation for serving on the Board of Directors of Stockholder of Mallinckrodt ARD, Inc., and of GlaxoSmithKline. Dr. Becker has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of Mallinckrodt ARD, Inc. Dr. Becker has received compensation for serving on the Board of Directors of Stockholder of Mallinckrodt ARD, Inc. Dr. Mackey, PhD has nothing to disclose. Dr. Fang, MS, JD has nothing to disclose. Dr. Zhao, MS has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of Mallinckrodt ARD, Inc. Dr. Zhao, MS has received compensation for serving on the Board of Directors of Stockholder of Mallinckrodt ARD, Inc.