Results From a Phase 4, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Repository Corticotropin Injection for the Treatment of Pulmonary Sarcoidosis.
Acthar Gel
Clinical trial
Pulmonary sarcoidosis
RCI
Repository corticotropin injection
Journal
Pulmonary therapy
ISSN: 2364-1746
Titre abrégé: Pulm Ther
Pays: United States
ID NLM: 101687144
Informations de publication
Date de publication:
Jun 2023
Jun 2023
Historique:
received:
30
12
2022
accepted:
20
03
2023
medline:
19
4
2023
pubmed:
19
4
2023
entrez:
18
04
2023
Statut:
ppublish
Résumé
Long-term treatment of pulmonary sarcoidosis with glucocorticoids has been associated with toxicity and other adverse events, highlighting the need for alternative therapies. The goal of this study was to evaluate the efficacy and safety of repository corticotropin injection (RCI, Acthar In this multicenter, randomized, placebo-controlled trial, subjects received subcutaneous RCI (80 U) twice weekly or matching placebo through 24 weeks in a double-blind treatment phase, followed by an optional 24-week open-label extension. Efficacy was measured by glucocorticoid tapering, pulmonary function tests, chest imaging, patient-reported outcomes, and a novel sarcoidosis treatment score (STS). Safety was assessed by adverse events, physical examinations, vital signs, clinical laboratory abnormalities, and imaging. The study was terminated early due to low enrollment caused by the COVID-19 pandemic, thereby precluding statistical analysis. Fifty-five subjects were randomized to receive either RCI (n = 27) or placebo (n = 28). Mean STS at week 24 showed greater improvement with RCI (1.4) compared with placebo (0.7). At week 48, those who remained on RCI had an STS of 1.8 compared with 0.9 in those who switched from placebo to RCI. More subjects in the RCI group discontinued glucocorticoids at week 24 compared to the placebo group. Glucocorticoid discontinuation was comparable at week 48 for those who switched from placebo to RCI and those who continued RCI. Similar trends in favor of RCI over placebo were observed with the other efficacy endpoints. No new or unexpected safety signals were identified. RCI was safe and well tolerated, with trends in efficacy data suggesting greater improvement with RCI compared to placebo in patients receiving standard-of-care therapy for pulmonary sarcoidosis. The study also provided validation of efficacy endpoints that may be used in larger trials for pulmonary sarcoidosis. ClinicalTrials.gov identifier: NCT03320070. Pulmonary sarcoidosis is a disease characterized by inflammation of the lungs. Standard treatments include glucocorticoids, which may have harmful side effects. This clinical trial investigated whether repository corticotropin injection (RCI, Acthar
Autres résumés
Type: plain-language-summary
(eng)
Pulmonary sarcoidosis is a disease characterized by inflammation of the lungs. Standard treatments include glucocorticoids, which may have harmful side effects. This clinical trial investigated whether repository corticotropin injection (RCI, Acthar
Identifiants
pubmed: 37072607
doi: 10.1007/s41030-023-00222-2
pii: 10.1007/s41030-023-00222-2
pmc: PMC10113127
doi:
Banques de données
ClinicalTrials.gov
['NCT03320070']
Types de publication
Journal Article
Langues
eng
Pagination
237-253Informations de copyright
© 2023. The Author(s).
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