Acthar Gel in Participants With Pulmonary Sarcoidosis

Study at a glance

From the registry record, last updated by the sponsor on 27 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

A Multicenter, Randomized, Double Blind, Placebo Controlled Exploratory Study to Assess the Efficacy and Safety of Acthar Gel in Subjects With Pulmonary Sarcoidosis

ClinicalTrials.gov ID

NCT03320070

Acronym / sponsor ID

PULSAR / MNK14344100

Conditions

Sarcoidosis, Pulmonary

Phase

Phase 4

Study type

Interventional

Recruitment status

Completed

Enrolment

55 participants (actual)

Lead sponsor

MallinckrodtIndustry (pharma, biotech, devices)

Study start

21 Feb 2018 (actual)

Primary completion

15 Nov 2021 (actual)

Study completion

15 Nov 2021 (actual)

First posted

25 Oct 2017 (actual)

Results

Posted 27 Feb 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Randomised

Intervention model

Parallel groups

Masking

Double blind (participant, investigator)

Number of arms

2

Who may qualify

Ages

18 to 90 years

Sex

All

Healthy volunteers

Not accepted

Age groups

Adult, Older adult

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24

   Time frame: Week 24

   Forced vital capacity (FVC) is a pulmonary function test parameter that indicates the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It's measured by spirometry, which is a common breathing test to check lung function. Based on the absolute change of percent predicted, FVC was evaluated to determine if the condition is: * Improved (+1) [≥ 5% absolute change] * Unchanged (0) [>- 5% to < 5% absolute change], or * Worse (-1) [≤ -5% absolute change] An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

2. OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48

   Time frame: Week 48

   Forced vital capacity (FVC) is a pulmonary function test parameter that indicates the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It's measured by spirometry, which is a common breathing test to check lung function. Based on the absolute change of percent predicted, FVC was evaluated to determine if the condition is: * Improved (+1) [≥ 5% absolute change] * Unchanged (0) [>- 5% to < 5% absolute change], or * Worse (-1) [≤ -5% absolute change] An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

3. 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

   Time frame: Week 24

   The diffusing capacity of the lungs for carbon monoxide (DLCO) measures the ability of the lungs to transfer gas from inhaled air to the red blood cells in the blood. Participants are asked to fully inhale a low concentration of carbon monoxide and an inert tracer gas. Based on the absolute change of percent predicted, DLCO was evaluated to determine if the condition is: * Improved (+1) [≥ 5% absolute change] * Unchanged (0) [>- 5% to < 5% absolute change], * Worse (-1) [≤ -5% absolute change] An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

4. OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48

   Time frame: Week 48

   The diffusing capacity of the lungs for carbon monoxide (DLCO) measures the ability of the lungs to transfer gas from inhaled air to the red blood cells in the blood. Participants are asked to fully inhale a low concentration of carbon monoxide and an inert tracer gas. Based on the absolute change of percent predicted, DLCO was evaluated to determine if the condition is: * Improved (+1) [≥ 5% absolute change] * Unchanged (0) [>- 5% to < 5% absolute change], * Worse (-1) [≤ -5% absolute change] An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

5. DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24

   Time frame: Week 24

   High-resolution computer tomography (HRCT) is a type of computed tomography (CT) with specific techniques to enhance image resolution. It is used in the diagnosis of various health problems, most commonly for lung disease. These images show cross sections (slices) through the lungs. HRCT imaging was evaluated by the investigator/radiologist and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1). An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

6. OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48

   Time frame: Week 48

   High-resolution computer tomography (HRCT) is a type of computed tomography (CT) with specific techniques to enhance image resolution. It is used in the diagnosis of various health problems, most commonly for lung disease. These images show cross sections (slices) through the lungs. HRCT imaging was evaluated by the investigator/radiologist and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1). An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

7. 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

   Time frame: Week 24

   King's sarcoidosis questionnaire (KSQ) (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Each item was answered on a 7-point scale where 1 means the participant experiences the symptom all the time and 7 means the participant does not experience the symptom at all. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: * Improved (+1) based on a change of ≥ 4 points * Unchanged (0) based on a change of >- 4 to < 4 points * Worse (-1) based on a change of ≤ -4 points An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

8. OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48

   Time frame: Week 48

   King's sarcoidosis questionnaire (KSQ) (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Each item was answered on a 7-point scale where 1 means the participant experiences the symptom all the time and 7 means the participant does not experience the symptom at all. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: * Improved (+1) based on a change of ≥ 4 points * Unchanged (0) based on a change of >- 4 to < 4 points * Worse (-1) based on a change of ≤ -4 points An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

9. DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24

   Time frame: Week 24

   The fatigue assessment score (FAS) is a 10-item checklist for participants to indicate the level of their fatigue. Each item was answered on a 5-point scale where 1 means the participant does not experience the symptom all and 5 means the participant experiences the symptom all the time. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: * Improved (+1) based on a change of ≤ -4 points * Unchanged (0) based on a change of >- 4 to < 4 points * Worse (-1) based on a change of ≥ 4 points An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

10. OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48

    Time frame: Week 48

    The fatigue assessment score (FAS) is a 10-item checklist for participants to indicate the level of their fatigue. Each item was answered on a 5-point scale where 1 means the participant does not experience the symptom all and 5 means the participant experiences the symptom all the time. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: * Improved (+1) based on a change of ≤ -4 points * Unchanged (0) based on a change of >- 4 to < 4 points * Worse (-1) based on a change of ≥ 4 points An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.

11. DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24

    Time frame: Week 24

    Corticosteroids are first line treatment for sarcoidosis. Concerns of corticosteroid toxicity led to efforts to taper dose sooner. Participants were clinically evaluated at each visit by investigator and categorized by their condition; dose was tapered using algorithm to take them off prednisone using incremental doses of 40,30,20,10,7.5,5,2.5,0 mg, as explained with each category below. * Improved: When condition improved, reduce dose by 1 level * Unchanged: 1. When stable condition without toxicity: At first stable visit they continue same dose; at second stable visit, reduce dose by 1 level 2. When stable condition with toxicity: toxicity is treated; reduce dose by 1 level * Deteriorate: When worsening condition, increase dose by 1 or 2 levels but not >40mg/day Dose tapering was done based on the participant's clinical condition. Category "Missing Assessment" indicates participants who had a missing assessment for this outcome measure.

12. OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48

    Time frame: Week 48

    Corticosteroids are first line treatment for sarcoidosis. Concerns of corticosteroid toxicity led to efforts to taper dose sooner. Participants were clinically evaluated at each visit by investigator and categorized by their condition; dose was tapered using algorithm to take them off prednisone using incremental doses of 40,30,20,10,7.5,5,2.5,0 mg, as explained with each category below. * Improved: When condition improved, reduce dose by 1 level * Unchanged: 1. When stable condition without toxicity: At first stable visit they continue same dose; at second stable visit, reduce dose by 1 level 2. When stable condition with toxicity: toxicity is treated; reduce dose by 1 level * Deteriorate: When worsening condition, increase dose by 1 or 2 levels but not >40mg/day Dose tapering was done based on the participant's clinical condition. Category "Missing Assessment" indicates participants who had a missing assessment for this outcome measure.

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2023.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
• Derived from this study: PubMed 37072607Mirsaeidi M, Baughman RP, Sahoo D, Tarau E. Results From a Phase 4, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Repository Corticotropin Injection for the Treatment of Pulmonary Sarcoidosis. Pulm Ther. 2023 Jun;9(2):237-253. doi: 10.1007/s41030-023-00222-2. Epub 2023 Apr 19.
• Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

29 sites in 1 country: see every site and its status.