A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

What Was Tested

This registered phase 2 trial tested ACTH (Corticotropin).

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.

It had four arms (groups): one (experimental), one (experimental), one (experimental) and one (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 43 actual participants took part.

It was open to people of any sex aged from 18 years up to 80 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Able to provide informed consent. Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS, clinically probable ALS, or clinically possible ALS based on the revised El Escorial criteria. Patients with ALS ≤ 3 years since symptom onset. Symptom onset is defined as date of first muscle weakness or dysarthria. If taking riluzole and/or Nuedexta®, stable regimen is required for ≥ 30 days prior to screening. Medically (either independently or with caregiver assistance) able to comply with study procedures, including subcutaneous (SC) injections of study medication and adherence to concomitant medication restrictions.

The full record lists 6 inclusion and 15 exclusion criteria; among the exclusions: recorded diagnosis or evidence of major psychiatric disorder; therapies and/or medications:; history of prior sensitivity to acthar or other porcine protein products.

How the Study Was Run

Design: randomized, parallel-group and open-label.

Random assignment meant chance, not the researchers, decided who got ACTH (Corticotropin) and who got the comparison, which limits bias.

The ACTH (Corticotropin) group and the comparison group ran side by side rather than switching.

Being open-label, the ACTH (Corticotropin) study did not mask who received what.

It started in 2013-07 with completion listed for 2014-11.

It ran at 17 sites in United States (17).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication (time frame: Baseline to Week 8).

It also tracked 3 secondary outcomes, for example Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation, Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication and Proportion of Subjects With Treatment Emergent Suicidality.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of ACTH (Corticotropin) in 43 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the ACTH (Corticotropin) compound page, all human trials.