Mallinckrodt Starts Phase 4 Trial for Acthar Gel to Treat Pulmonary Sarcoidosis

Alice Melão, MSc avatar

by Alice Melão, MSc |

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The first patient was recently enrolled in a Phase 4 trial to further evaluate the effectiveness of H.P. Acthar Gel as a therapy for pulmonary sarcoidosis, according to Mallinckrodt.

Acthar Gel (repository corticotropin injection) is a U.S. Food and Drug Administration (FDA)-approved therapy for 19 different indications, including respiratory symptomatic sarcoidosis.

The Phase 4 clinical trial (NCT03320070) is being conducted in research sites across the United States. It is expected to include about 100 adult patients with a confirmed diagnosis of sarcoidosis with symptomatic pulmonary disease who are receiving prednisone treatment.

“The enrollment of the first patient in this study is an important milestone in our further assessment of Acthar’s efficacy as a treatment option for sarcoidosis patients,” Tunde Otulana, MD, chief medical officer at Mallinckrodt, said in a press release.

“We believe this exploratory evaluation of the more challenging patients who may still have disease activity after previous therapies can potentially help physicians better understand which individuals may benefit from the drug as a treatment alternative,” Otulana added.

Participants will be randomized to receive either 1 mL (corresponding to 80 units) of Acthar Gel or placebo, which will be administrated subcutaneously twice a week over 24 weeks.

The efficacy of the treatment will be evaluated by the Sarcoidosis Treatment Score (STS), which is a new composite score that combines the data of pulmonary function tests with high-resolution CT scans, quality of life analysis, and gradual reduction of corticosteroid therapy. STS baseline results (before treatment starts) will be compared with data collected at the end of the 24 weeks of treatment.

“I am pleased that the first patient has been enrolled in this important Phase 4 study, the results of which may potentially provide evidence to further support Acthar as a treatment option in appropriate symptomatic sarcoidosis patients,” said Robert Baughman, MD, professor in the Department of Internal Medicine at the University of Cincinnati College of Medicine in Ohio.

“In my experience, a considerable number of sarcoidosis patients can have persistent disease that may not be resolved by first-line treatment,” he added.

Patients who complete the 24 weeks of the trial will be eligible to continue treatment in an optional 24-week open-label extension.

For additional information about the trial, visit the trial’s registry webpage here.