A Phase 2 Open-Label Pilot Study of the Safety and Tolerability of Ixazomib Administered Orally to Patients With Scleroderma-Related Interstitial Lung Disease
The purpose of this research study is to learn about the effects of the medication ixazomib in participants with scleroderma/systemic sclerosis including its safety and tolerability, its effects on skin, lungs and other organs, and its effects on overall health and quality of life.
• Male and female patients, age ≥18 years at time of signing informed written consent
• Confirmed diagnosis of diffuse cutaneous systemic sclerosis/scleroderma
• Disease duration not longer than 60 months defined as the time from the first non-Raynaud phenomenon manifestation
• Scleroderma skin thickness score (modified Rodnan skin score) between 15 and 45
• Evidence of scleroderma-related lung involvement on chest CT scan completed within the preceding 3 months or at screening study visit
• Pulmonary function testing demonstrating FVC ≥45% predicted and DLCO ≥40% predicted at screening study visit
• Resting transthoracic echocardiogram within the preceding 6 months or at screening study visit without evidence of pulmonary artery hypertension
• Stable mycophenolate dose during the preceding 3 months for those who are taking mycophenolate. Mycophenolate use is not an eligibility requirement to participate; but those participants already using mycophenolate at screening study visit will continue taking the medication throughout the entire study.
• Willingness to undergo supervised withdrawal during the first 90 days of the study of any other medication besides mycophenolate used specifically as treatment of scleroderma-related interstitial lung disease with confirmed stable pulmonary status.
• Willingness to undergo supervised withdrawal during the first 90 days of the study of any other prohibited medications with confirmed stable status.
• Able to understand and sign a written informed consent form
• Able to understand the importance of adhering to study treatment and the study protocol, and willing and able to follow all study requirements, including the concomitant medication restrictions, throughout the study
• Practice birth control requirements for sexually active female participants including option of abstinence for the entire study and for at least 90 days after the last dose of study medication
• Practice birth control requirements for sexually active male participants or partners including option of abstinence for the entire study and for at least 90 days after the last dose of study medication