NCT04837131

A Study to Evaluate the Safety and Tolerability of Oral Ixazomib in Scleroderma-related Lung Disease Patients

A Phase 2 Open-Label Pilot Study of the Safety and Tolerability of Ixazomib Administered Orally to Patients With Scleroderma-Related Interstitial Lung Disease

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Michael M. Pham · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The primary objective of this study is to assess the safety and tolerability of oral ixazomib taken on days 1, 8, and 15 of each 28-day treatment cycle for 6 cycles in 12 participants having diffuse systemic sclerosis/scleroderma of less than 5 years duration with non-severe interstitial lung involvement as identified from chest CT scan completed within the preceding 3 months or at study screening visit. All 12 participants will receive oral ixazomib. Reflecting the common use of mycophenolate in the management of scleroderma with complicating interstitial lung disease, 6 of 12 participants who will be enrolled will be already taking mycophenolate at a stable dose for the preceding 3 months and will be allowed to continue taking mycophenolate throughout the entire study prescribed as routine care. Ixazomib will be added to their medications. The remaining 6 of 12 participants meeting the same eligibility criteria will not be using mycophenolate or any other treatment for scleroderma interstitial lung disease at the time of enrollment and will subsequently take ixazomib study medication during participation in this study. Ixazomib dose modification or interruption is allowed for safety and tolerability reasons at any time during the study. The secondary objective of this study is to assess the effect of ixazomib on scleroderma skin tightness/thickening and its effect on scleroderma interstitial lung disease. The study includes approximately 13 clinic visits over up to approximately 10 months. Study procedures include medical examinations, blood tests, chest CT scans, pulmonary function tests, echocardiogram, EKG, skin biopsies, and questionnaires. There is no cost for participation in this study.

Conditions

• Systemic Sclerosis
• Scleroderma
• Diffuse Systemic Sclerosis
• Diffuse Scleroderma
• Diffuse Cutaneous Systemic Sclerosis
• Diffuse Cutaneous Scleroderma
• Progressive Systemic Sclerosis
• Progressive Scleroderma
• Scleroderma, Systemic
• Scleroderma, Diffuse
• Scleroderma;Progressive
• Systemic Sclerosis, Diffuse
• Systemic; Sclerosis, Progressive
• Scleroderma of Lung
• Scleroderma With Pulmonary Involvement
• Systemic Sclerosis Pulmonary
• Systemic Sclerosis With Lung Involvement
• Interstitial Lung Disease
• Pulmonary Fibrosis Interstitial

Interventions

Type	Name	Description
DRUG	Ixazomib	Ixazomib 4 mg capsule taken orally on days 1, 8, and 15 of a 28-day treatment cycle repeated for 6 cycles

Timeline

• Start date: 2021-04-28
• Primary completion: 2024-02-23
• Completion: 2024-02-23
• First posted: 2021-04-08
• Last updated: 2025-04-06
• Results posted: 2025-04-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study