Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)

What Was Tested

The trial (phase 2, phase 3) is built around ACTH (Corticotropin).

About 213 people with ALS will participate in this study.

There will be locations in North and South America.

During the first part, participants will be randomly assigned to a group (like by flipping a coin).

It had four arms (groups): Arm A: Treatment Period Acthar (experimental), Arm B: Treatment Period Placebo (placebo comparator), Arm C: Extension Period Acthar-Acthar (experimental) and Arm D: Extension Period Placebo-Acthar (experimental).

The registry classes the interventions as drug.

More on the Plan

Approximately 213 participants will be enrolled.

Who Took Part

Enrollment: 143 actual participants took part.

It was open to people of any sex aged from 18 years up to 75 years.

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

Examples of who could join: Is 18-75 years of age at Screening. Has ALS symptom onset within 2 years prior to Screening.

The full record lists 4 inclusion and 6 exclusion criteria; among the exclusions: has tracheostomy, diaphragm pacing, or an ongoing need for assisted ventilation of any type; has used any medication within a time period not allowed per protocol; used edaravone less than 1 week before screening.

How the Study Was Run

Design: randomized, parallel-group and double-blind.

Because people in this ACTH (Corticotropin) study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Groups ran in parallel: each of the participants stayed in one arm throughout.

As a double-blind ACTH (Corticotropin) study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant and investigator.

It started in 2017-06-22 with completion listed for 2019-11-25.

It ran at 69 sites in United States (39), Argentina (10), Mexico (7), Peru (5), Canada (4) and Chile (3).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) (time frame: Baseline, Week 36). Number of Participants Experiencing an Adverse Event During the Treatment Period (time frame: by the end of the treatment period (within 36 Weeks)). Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period (time frame: by the time of database lock (within 84 weeks)).

It also tracked 3 secondary outcomes, for example Treatment Period: Spirometry (%), Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R and Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3, randomized trial of ACTH (Corticotropin) in 143 people.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

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