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Acthar Gel in Participants With Pulmonary Sarcoidosis (PULSAR)

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ClinicalTrials.gov Identifier: NCT03320070
Recruitment Status : Active, not recruiting
First Posted : October 25, 2017
Last Update Posted : September 16, 2021
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt

Tracking Information
First Submitted Date  ICMJE October 20, 2017
First Posted Date  ICMJE October 25, 2017
Last Update Posted Date September 16, 2021
Actual Study Start Date  ICMJE January 24, 2018
Estimated Primary Completion Date January 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 31, 2020)
  • Number of Participants in each Category of Assessment based on Forced Vital Capacity, a Pulmonary Function Test Parameter [ Time Frame: 24 weeks ]
    Based on absolute change of percent predicted, FVC is 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]
  • Number of Participants in each Category of Assessment based on Forced Vital Capacity, a Pulmonary Function Test Parameter [ Time Frame: 48 weeks ]
    Based on absolute change of percent predicted, FVC is 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]
  • 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 [ Time Frame: 24 weeks ]
    Based on absolute change of percent predicted, DLCO is evaluated to determine if the condition is:
    • Improved (+1) [≥ 5% absolute change]
    • Unchanged (0) [>- 5% to < 5% absolute change],
    • Worse (-1) [≤ -5% absolute change]
  • 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 [ Time Frame: 48 weeks ]
    Based on absolute change of percent predicted, DLCO is evaluated to determine if the condition is:
    • Improved (+1) [≥ 5% absolute change]
    • Unchanged (0) [>- 5% to < 5% absolute change],
    • Worse (-1) [≤ -5% absolute change]
  • Number of Participants in each Category of Assessment based on High Resolution Computer Tomography (HRCT) [ Time Frame: 24 weeks ]
    HRCT imaging will be evaluated by the investigator/radiology and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1).
  • Number of Participants in each Category of Assessment based on High Resolution Computer Tomography (HRCT) [ Time Frame: 48 weeks ]
    HRCT imaging will be evaluated by the investigator/radiology and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1).
  • Number of participants in each Category of Assessment based on the King's Sarcoidosis Questionnaire (General Health), a Quality of Life Parameter [ Time Frame: 24 weeks ]
    King's Sarcoidosis Questionnaire (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale is 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
  • Number of participants in each Category of Assessment based on the King's Sarcoidosis Questionnaire (General Health), a Quality of Life Parameter [ Time Frame: 48 weeks ]
    King's Sarcoidosis Questionnaire (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale is 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
  • Number of participants in each Category of Assessment based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter [ Time Frame: 24 weeks ]
    The FAS is a 10-item checklist for participants to indicate the level of their fatigue. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale is 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
  • Number of participants in each Category of Assessment based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter [ Time Frame: 48 weeks ]
    The FAS is a 10-item checklist for participants to indicate the level of their fatigue. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale is 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
  • Number of Participants Receiving each Dose of Prednisone [ Time Frame: 24 weeks ]
    Corticosteroids are typically the first-line when treatment of sarcoidosis is required. Concerns of significant corticosteroid toxicity results in efforts to taper as early as possible. Participants are evaluated at each visit following randomization, and an algorithm is used to taper them off prednisone using incremental doses of 40, 30, 20, 10, 7.5, 5, 2.5 and 0 mg. When the clinical status is:
    • Improvement, they go down by one level
    • First stable visit without toxicity, they continue the same dose
    • Second stable visit without toxicity, the go down by one level
    • Stable visit with toxicity, their toxicity is treated and they may go down by one level
    • Worsening, they go up by one or two levels, but do not exceed 40 mg/day
  • Number of Participants Receiving each Dose of Prednisone [ Time Frame: 48 weeks ]
    Corticosteroids are typically the first-line when treatment of sarcoidosis is required. Concerns of significant corticosteroid toxicity results in efforts to taper as early as possible. Participants are evaluated at each visit following randomization, and an algorithm is used to taper them off prednisone using incremental doses of 40, 30, 20, 10, 7.5, 5, 2.5 and 0 mg. When the clinical status is:
    • Improvement, they go down by one level
    • First stable visit without toxicity, they continue the same dose
    • Second stable visit without toxicity, the go down by one level
    • Stable visit with toxicity, their toxicity is treated and they may go down by one level
    • Worsening, they go up by one or two levels, but do not exceed 40 mg/day
Original Primary Outcome Measures  ICMJE
 (submitted: October 20, 2017)
Response will be evaluated by Sarcoidosis Treatment Score (STS) [ Time Frame: Baseline to 24 weeks ]
STS is an exploratory newly developed composite score that combines the results of pulmonary function tests (forced vital capacity; diffusing capacity of the lungs for carbon monoxide), high-resolution computed tomography, Quality of life (King's Sarcoidosis Questionnaire; Fatigue Assessment Scale) and corticosteroid tapering.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Acthar Gel in Participants With Pulmonary Sarcoidosis
Official Title  ICMJE A Phase 4, Multicenter, Randomized, Double Blind, Placebo Controlled Pilot Study to Assess the Efficacy and Safety of Acthar Gel in Subjects With Pulmonary Sarcoidosis
Brief 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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Sarcoidosis, Pulmonary
Intervention  ICMJE
  • Drug: Acthar Gel
    Acthar Gel for subcutaneous (SC) injection (80 units per 1 mL)
    Other Names:
    • H.P. Acthar Gel
    • Repository Corticotropin Injection
  • Drug: Placebo
    Placebo gel for SC injection
    Other Name: Matching Placebo
Study Arms  ICMJE
  • Experimental: Acthar Gel
    Participants receive Acthar Gel as a 1 mL injection under the skin twice weekly
    Intervention: Drug: Acthar Gel
  • Placebo Comparator: Placebo
    Participants receive Placebo as a 1 mL injection under the skin twice weekly
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: December 2, 2020)
55
Original Estimated Enrollment  ICMJE
 (submitted: October 20, 2017)
100
Estimated Study Completion Date  ICMJE January 2022
Estimated Primary Completion Date January 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 90 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03320070
Other Study ID Numbers  ICMJE MNK14344100
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Mallinckrodt
Study Sponsor  ICMJE Mallinckrodt
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Team Leader Mallinckrodt
PRS Account Mallinckrodt
Verification Date September 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP