Conditions: Amyotrophic Lateral Sclerosis
Keywords: H.P. Acthar Gel Acthar amyotrophic lateral sclerosis ALS
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 43
Healthy volunteers: no

Brief summary:
This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis (ALS). The study will also investigate the mean rate of change in the ALSFRS-R total score as an exploratory endpoint to help design future studies.

This study will enroll up to 40 patients and include an optional 28-week open-label extension period plus a 3-week treatment taper and 1-week follow up period. After completion of Week 8, patients enrolled in a treatment group that is considered safe and tolerable at that time have the option to continue into the open-label extension period. A 3-week treatment taper and a follow-up visit are planned for all patients enrolled in the study, beginning either at Week 8 or at Week 36 if a patient continues into the optional open-label extension period.

Detailed description:

Arms:
arm 1: Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly arm 2: Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily arm 3: Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly arm 4: Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily

Interventions: intervention 1: Repository corticotropin injection
Intervention descriptions: intervention 1: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks

Protocol document: