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Phase 4 N=55 Randomized Double-blind Treatment

Acthar Gel in Participants With Pulmonary Sarcoidosis

Sarcoidosis, Pulmonary

Enrolled (actual)
55
Serious AEs
12.8%
Results posted
Feb 2023
Primary outcome: Primary: DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 — 10; 7; 12; 12 Participants — p=0.5076

Study Design & Population

Study type
Interventional
Phase
Phase 4
Interventions
Acthar Gel (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Mallinckrodt
Primary completion
Nov 2021

Outcome Measures

OutcomeResultp-value
PRIMARY
DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24
10; 7; 12; 12; 1; 3 0.5076
PRIMARY
OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48
2; 6; 13; 11; 1; 3
PRIMARY
DBT: Number of Participants in Each Category of Assessment Based on the Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO), a Pulmonary Function Test Parameter at Week 24
5; 4; 11; 11; 7; 7 1.0000
PRIMARY
OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48
9; 6; 3; 6; 4; 8
PRIMARY
DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24
2; 1; 22; 18; 0; 4 0.0848
PRIMARY
OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48
1; 6; 13; 13; 1; 2
PRIMARY
DBT: Number of Participants in Each Category of Assessment Based on the King's Sarcoidosis Questionnaire (KSQ) (General Health), a Quality of Life Parameter at Week 24
11; 7; 3; 8; 10; 9 0.2005
PRIMARY
OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48
8; 8; 2; 5; 6; 9
PRIMARY
DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24
6; 6; 12; 11; 6; 7 0.9416
PRIMARY
OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48
7; 4; 5; 13; 4; 5
PRIMARY
DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24
17; 21; 8; 4; 0; 0 0.1853
PRIMARY
OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48
15; 19; 5; 5; 0; 0

Summary

The purpose of this study is to find out if Acthar Gel is safe and effective to treat pulmonary sarcoidosis. Participants will be randomly assigned (like flipping a coin) to receive a shot under their skin of Acthar Gel or a matching placebo gel that has no drug in it. They will receive their assigned shot twice a week for 24 weeks. All participants who complete the 24-week treatment period will be eligible to receive Acthar Gel for 24 more weeks, even if they were originally in the placebo group.

Eligibility Criteria

Inclusion Criteria

  • Has biopsy-confirmed sarcoidosis meeting American Thoracic Society criteria ≥ 1 year at screening (Visit 1)
  • Has protocol-defined symptomatic pulmonary disease
  • Has been receiving a stable prednisone dose between 5 mg and 40 mg (or equivalent) for pulmonary sarcoidosis, for at least 4 weeks before screening, or a stable dose of another disease-modifying anti-sarcoidosis drug for at least 3 months before screening
  • Has lung function within protocol-defined parameters

Exclusion Criteria

  • Has at least a 10% change in forced vital capacity (FVC) on spirometry between Visits 1 and 2
  • Has pulmonary arterial hypertension requiring treatment
  • Has been treated with antitumor necrosis factor-α antibody within the past 3 months
  • Has any pulmonary condition that requires treatment, therefore impeding corticosteroid tapering
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03320070). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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