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

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01732211

Enrollment

Registered

2012-11-22

Start date

2013-03-15

Completion date

2013-09-23

Last updated

2020-01-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pulmonary Sarcoidosis

Keywords

Phase 2, Randomized, Double Blind, Placebo Controlled, Parallel, 20 Week Safety, Tolerability, and Efficacy Study

Brief summary

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

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.

Interventions

BIOLOGICALPD 0360324

100 mg Q2W or 150 mg Q2W via intravenous infusion based on protocol determined dose escalation/de-escalation criteria; 12 weeks Active Therapy;

OTHERNormal Saline for injection

normal saline Q2W via intravenous infusion for 12 weeks

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

BASIC_SCIENCE

Masking

TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender

ALL

Age

21 Years to 75 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Change From Baseline (Absolute) in % Predicted Forced Vital Capacity (FVC) Compared to Placebo at Week 16	Baseline, 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.

Secondary

Measure	Time frame	Description
Change From Baseline in FVC (Absolute ) Compared to Placebo at Weeks 2,4,8,12, 14, and 20	Baseline, 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.
Change From Baseline Absolute in FVC (% Predicted) Compared to Placebo at Weeks 2,4,8,12,14, and 20	Baseline, 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.
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Weeks 2,4,8,12,14, 16 and 20	Baseline, 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.
Change From Baseline in Chest X-ray Global Assessment Score (5-point )	Baseline, Week 16	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.
Change From Baseline in Ratio of FEV1/FVC at Weeks 2,4,8,12,14, 16 and 20	Baseline, 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.
Estimated Treatment Effect Over Placebo in FVC Averaged Over 16 Weeks	Baseline, Weeks 0, 2, 4, 8, 12, 14, 16	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.
Change From Baseline in % Predicted FEV1 at Weeks 2,4,8,12,14, 16 and 20	Baseline, 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.

Countries

United States

Participant flow

Pre-assignment details

In Part A, approximately 22 subjects were to be enrolled to receive PD 0360324 100 mg once every 2 weeks (Q2W)/150 mg Q2W. In Part B, approximately 66 subjects were planned to be enrolled to receive PD 0360324 (either 100 or 150 mg Q2W defined in Part A) B.

Participants by arm

Arm	Count
PD 0360324 100 mg Q2W / 150 mg Q2W PD-0360324 100 milligram (mg) was administered as intravenous infusion on Day 1 of every 2 weeks for Week 0 and Week 2. PD-0360324 150 mg was administered as intravenous infusion on Day 1 of every 2 weeks for Weeks 4, 6, 8, 10, and 12.	1
Total	1

Baseline characteristics

Characteristic	PD 0360324 100 mg Q2W / 150 mg Q2W
Age, Continuous	58 years
Sex: Female, Male Female	0 Participants
Sex: Female, Male Male	1 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	1 / 1
serious Total, serious adverse events	0 / 1

Outcome results

Primary

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.

Time frame: Baseline, Week 16