Trial to Evaluate the Hormone Drug Acthar in ALS

A 40-person phase 2 clinical trial to test the safety and tolerability of Questcor Pharmaceuticals’ H.P. Acthar Gel (Acthar) in people with amyotrophic lateral sclerosis (ALS) has opened at a trial site in Phoenix, Ariz.

Acthar (also known as adrenocorticotropic hormone, ACTH, ACTH gel and corticotropin) is a formulation of hormones that causes increased production and release of other hormones called corticosteroids (also known as glucocorticoids) known to suppress the immune system.

Acthar is approved by the U.S. Food and Drug Administration for the treatment of a number of disorders including multiple sclerosis, infantile spasms, dermatomyositis (DM) and polymyositis (PM).

Note: Acthar’s effects on people with ALS are unknown. The manufacturer, Questcor, reports that Acthar can cause serious side effects. Therefore, individuals are strongly advised not to use Acthar to treat ALS until it has been proven safe and effective in people with the disease.

Investigators associated with the trial will evaluate the safety and tolerability of four different dosing regimens of Acthar to help inform dose selection of future studies of the drug in ALS.

About the Acthar trial

Participants in the eight-week trial will be randomly assigned to one of four treatment groups. Each group will be treated with one of four doses of Acthar, which is administered by subcutaneous injection. The trial is open-label, meaning all participants will receive the study drug (not a placebo), and participants and investigators will know which dose each participant receives.

Investigators will monitor participants for adverse events, and changes in vital signs (blood pressure, heart rate and body temperature) and clinical laboratory test measurements.

At the end of eight weeks, trial participants enrolled in a treatment group with a dose that is considered safe and tolerable will have the option to continue into an open-label 28-week extension study, All participants will gradually taper off the drug and return for a follow-up visit after the completion of their trial period.

To learn more about the trial, see A Study to Explore the Safety and Tolerability of Acthar in Patients with Amyotrophic Lateral Sclerosis, or search for trial ID NCT01906658 on

To participate in the phase 2 trial

Prospective trial participants must be 18 to 80 years old and have a diagnosis of clinically definite, clinically probable-laboratory supported, clinically probable, or clinically possible ALS based on the revised El Escorial Criteria. In addition, they must:

  • be within three years of symptom onset (defined as the date of first muscle weakness or speech difficulty);
  • have an upright slow vital capacity (SVC) greater than or equal to 60 percent of the predicted value;
  • not have tracheostomy, diaphragm pacing or ongoing need for assisted ventilation of any type;
  • not have participated in another therapeutic investigational study for a drug or device within 30 days of screening for this trial; and
  • meet additional criteria.

The study, led by principal investigator David Saperstein, is being conducted at Phoenix Neurological Associates, in Phoenix, Ariz. For more information, contact Shanique Smythe at (443) 973-2012.

About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more about clinical trials, see Being a Co-Adventurer. For a more refined list of ALS clinical trials, visit, a registry of federally and privately supported clinical trials in the United States and around the world. Enter your trial ID number or subject in the "Search for Studies" box on the home page.

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