NCT03324503

A Study to Estimate the Effect Sizes of HRCT Endpoints in Response to Glucocorticoid Induction Therapy in Subjects With Pulmonary Sarcoidosis

A Multicenter, Open-Label Study to Estimate the Effect Sizes of HRCT Endpoints in Response to GLUCOCORTICOID Induction Therapy in Subjects With Pulmonary Sarcoidosis

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Celgene · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, single-arm, unblinded/open-label study of the effect size of HRCT endpoints in response to glucocorticoid induction therapy in subjects with a diagnosis of pulmonary sarcoidosis who have not received glucocorticoid as initial sarcoidosis therapy (≥ 20 mg/day prednisone or prednisolone) or other sarcoidosis therapy for at least 3 months prior to enrollment. This study will enroll a total of approximately 24 subjects.

Detailed description

Optimal development of novel treatments for sarcoidosis requires clinical study endpoints that efficiently reflect changes in disease activity over short treatment durations. Such endpoints enable preliminary assessment of candidate drug efficacy in small studies - prior to exposing large cohorts to experimental compounds in pursuit of registrational data. This study will accordingly investigate the ways that certain HRCT-based functional respiratory imaging (FRI) measures can serve as more quantitative and sensitive endpoints compared to pulmonary function tests to measure drug effect in a short duration small sample size study in patients with pulmonary sarcoidosis. The study is designed to minimize impact on sarcoid care that the patient would have otherwise received. Glucocorticoid (≥ 30 mg/day prednisone or prednisolone) as per local clinical practice of sarcoidosis initial induction therapy will be taken orally in this study. The glucocorticoid dose may be modified depending on ongoing assessment as a part of standard local clinical practice.

Conditions

• Sarcoidosis, Pulmonary

Interventions

Type	Name	Description
DRUG	Glucocorticoid (prednisone or prednisolone)	≥ 30 mg/day prednisone or prednisolone taken orally as per local clinical practice of sarcoidosis initial induction therapy

Timeline

• Start date: 2017-12-08
• Primary completion: 2019-11-15
• Completion: 2019-11-15
• First posted: 2017-10-27
• Last updated: 2020-02-26

Locations

20 sites across 3 countries: United States, Netherlands, United Kingdom