A Phase 2, Safety, Tolerability, and Efficacy Study of PD 0360324 in Chronic Pulmonary Sarcoidosis

Study Overview
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Brief Summary
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The study proposes to evaluate the safety, tolerability, and efficacy of PD 0360324 in subjects with chronic pulmonary sarcoidosis inadequately treated with standard of care therapies.

Detailed Description
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The trial was terminated prematurely on Mar 24, 2014 due to a business decision. The decision to terminate the trial was not based on any clinical safety or efficacy concerns.

Official Title
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A PHASE 2 RANDOMIZED, DOUBLE BLIND, PLACEBO CONTROLLED, PARALLEL GROUP, 20-WEEK SAFETY, TOLERABILITY, AND EFFICACY STUDY OF PD 0360324 IN ADULT SUBJECTS WITH CHRONIC PULMONARY SARCOIDOSIS

Conditions
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Pulmonary Sarcoidosis

Intervention / Treatment
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* Biological: PD 0360324
* Other: Normal Saline for injection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of biopsy-proved chronic pulmonary sarcoidosis either Stage 2 or Stage 3 for at least 1 year; Forced Vital Capacity (FVC) lung assessment of >40% and < or = to 80% predicted normal values at screening; age 21-75 years of age; treatment with a stable (longer than 4 weeks) regimen of corticosteroids (between 10 and 30 mg of prednisone) for at least 3 months prior to Screening; (other anti-inflammatory drugs may be permitted as defined by the study protocol) Exclusion Criteria: History of any other pulmonary (lung) disease than sarcoidosis (ex, asthma requiring maintenance treatment, chronic obstructive pulmonary disease (COPD)); Pulmonary hypertension, significant lung fibrosis, any chronic infection (eg, TB, HIV); treatment with other biologic anti-inflammatory/immuno-modulatory drugs; active smokers; class 3 or 4 congestive heart failure; cancer, or history of cancer within past 5 years; history of ischemic heart disease, heart attack, stroke, any heart muscle disease; liver disease; history of alcohol or drug abuse

Ages Eligible for Study
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Minimum Age: 21 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PD 0360324<br> | Biological: PD 0360324<br>* 100 mg Q2W or 150 mg Q2W via intravenous infusion based on protocol determined dose escalation/de-escalation criteria; 12 weeks Active Therapy;<br>|
| Placebo Comparator: Placebo<br> | Other: Normal Saline for injection<br>* normal saline Q2W via intravenous infusion for 12 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline (Absolute) in % Predicted Forced Vital Capacity (FVC) Compared to Placebo at Week 16 | The percent predicted FVC was calculated by observed FVC/predicted FVC * 100. The predicted FVC values was calculated according to age, height, race and gender. FVC is the volume of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. | Baseline, Week 16 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Chest X-ray Global Assessment Score (5-point ) | Digital copies of Chest x-rays performed during the study were graded according to a 5 point Likert scale: 1 = markedly worsened; 2 = worsened; 3 = unchanged; 4 = improved; and 5 = markedly improved. Baseline will be defined as the last available x-ray prior to first dose. | Baseline, Week 16 | 
| Change From Baseline in FVC (Absolute ) Compared to Placebo at Weeks 2,4,8,12, 14, and 20 | FVC is the volume of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. Trough FVC was obtained from spirometry, performed before study treatment administration. | Baseline, Weeks 2, 4, 8, 12, 14, and 20 | 
| Change From Baseline Absolute in FVC (% Predicted) Compared to Placebo at Weeks 2,4,8,12,14, and 20 | The percent predicted FVC was calculated by observed FVC/predicted FVC * 100. The predicted FVC values was calculated according to age, height, race and gender. FVC is the volume of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. | Baseline, Weeks 2, 4, 8, 12, 14, and 20 | 
| Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Weeks 2,4,8,12,14, 16 and 20 | FEV1 is the maximal volume of air exhaled in the first second of a forced expiration from a position of full inspiration. Trough FEV1 was obtained from spirometry, performed before study treatment administration. | Baseline, Weeks 2, 4, 8, 12, 14, 16, and 20 | 
| Change From Baseline in % Predicted FEV1 at Weeks 2,4,8,12,14, 16 and 20 | The percent predicted FEV1 was calculated by observed FEV1/predicted FEV1 * 100. The predicted FEV1 values was calculated according to age, height, race and gender. FEV1 is the maximal volume of air exhaled in the first second of a forced expiration from a position of full inspiration. | Baseline, Weeks 2, 4, 8, 12, 14, 16, and 20 | 
| Change From Baseline in Ratio of FEV1/FVC at Weeks 2,4,8,12,14, 16 and 20 | FEV1 is the maximal volume of air exhaled in the first second of a forced expiration from a position of full inspiration. Trough FEV1 was obtained from spirometry, performed before study treatment administration. FVC is the volume of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. Trough FVC was obtained from spirometry, performed before study treatment administration. | Baseline, Weeks 2, 4, 8, 12, 14, 16, and 20 | 
| Estimated Treatment Effect Over Placebo in FVC Averaged Over 16 Weeks | FVC is the volume of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. Through FVC was obtained from spirometry, performed before study treatment administration. | Baseline, Weeks 0, 2, 4, 8, 12, 14, 16 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Phase 2, Randomized, Double Blind, Placebo Controlled, Parallel, 20 Week Safety, Tolerability, and Efficacy Study