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

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

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.

Study Overview

• Status: Terminated
• Conditions: Pulmonary Sarcoidosis
• Intervention / Treatment: Biological: PD 0360324; Other: Normal Saline for injection

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10029
      • Mount Sinai School Of Medicine/Mount Sinai Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • University Hospital
    • Cincinnati, Ohio, United States, 45219
      • Medical Arts Building
    • Cincinnati, Ohio, United States, 45219
      • The Barrett Cancer Center at UC Health
    • Cincinnati, Ohio, United States, 45267
      • Iinterstitial Lung Disease & Sarcoidosis Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): March 15, 2013
• Primary Completion (Actual): September 23, 2013
• Study Completion (Actual): September 23, 2013

Study Registration Dates

• First Submitted: November 19, 2012
• First Submitted That Met QC Criteria: November 19, 2012
• First Posted (Estimate): November 22, 2012

Study Record Updates

• Last Update Posted (Actual): January 6, 2020
• Last Update Submitted That Met QC Criteria: December 16, 2019
• Last Verified: December 1, 2019

More Information

Terms related to this study

• Keywords: Randomized; Tolerability; Double Blind; Placebo Controlled; Phase 2; Parallel; 20 Week Safety; and Efficacy Study
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Lung Diseases; Lung Diseases, Interstitial; Sarcoidosis, Pulmonary; Sarcoidosis
• Other Study ID Numbers: A6261009

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.