Ixazomib for Scleroderma, Diffuse

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Scleroderma, Diffuse+18 More
Ixazomib - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new medication for scleroderma/systemic sclerosis to see if it is safe and effective.

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

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Scleroderma, Diffuse

Study Objectives

4 Primary · 10 Secondary · Reporting Duration: 7 months

12 weeks, 24 weeks
Dyspnea Status Rating (Mahler Transitional Dyspnea Index)
7 months
Number of participants with at least one treatment-emergent adverse event (AE)
Number of participants with treatment-emergent AEs leading to ixazomib dose modifications.
Number of participants with treatment-emergent AEs leading to ixazomib early discontinuation.
Baseline
Severity of dyspnea at baseline (Mahler Baseline Dyspnea Index)
Week 24
Change from baseline in Modified Rodnan Skin Score (MRSS)
Diffuse Scleroderma
Change in Patient Global Assessment of Disease Activity (PtGA)
Change in Physician Global Assessment of Disease Activity (MDGA)
Change in Scleroderma Health Assessment Questionnaire (SHAQ) score
Change in total lung capacity (TLC) % predicted
Baseline, 24 weeks
Change from baseline in high resolution chest CT scan Goh score
Month 7
Change in forced vital capacity (FVC) and diffusion capacity (DLCO) % predicted.
Month 7
Change in the UCLA Scleroderma Clinical Trials Consortium Gastrointestinal 2.0 (UCLA SCTC GIT 2.0) questionnaire score

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Scleroderma, Diffuse

Side Effects for

[VRd]; Bortezomib + Lenalidomide + Dexamethasone Therapy
67%Rash
50%Diarrhoea
50%Decreased appetite
33%Nasopharyngitis
33%Taste disorder
33%White blood cell count decreased
17%Compression fracture
17%Bone pain
17%Tibia fracture
17%Constipation
17%Neutrophil count decreased
17%Anaemia
17%Pyrexia
17%Malaise
17%Platelet count decreased
17%Spinal compression fracture
This histogram enumerates side effects from a completed 2021 Phase 4 trial (NCT03416374) in the [VRd]; Bortezomib + Lenalidomide + Dexamethasone Therapy ARM group. Side effects include: Rash with 67%, Diarrhoea with 50%, Decreased appetite with 50%, Nasopharyngitis with 33%, Taste disorder with 33%.

Trial Design

1 Treatment Group

Ixazomib in patients with scleroderma-interstitial lung disease (ILD)
1 of 1
Experimental Treatment

12 Total Participants · 1 Treatment Group

Primary Treatment: Ixazomib · No Placebo Group · Phase 2

Ixazomib in patients with scleroderma-interstitial lung disease (ILD)
Drug
Experimental Group · 1 Intervention: Ixazomib · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ixazomib
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 7 months

Who is running the clinical trial?

W. Leroy GriffingLead Sponsor
Leroy Griffing, MD5.011 ReviewsPrincipal Investigator - Mayo Clinic
Mayo Clinic
5Patient Review
I felt extremely confident in this doctor's ability after our consultation. They have excellent bedside manner and made sure I was comfortable with the information they were sharing with me. I would definitely recommend them to anyone seeking a great physician.

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have a confirmed diagnosis of diffuse cutaneous systemic sclerosis/scleroderma.
You are willing to undergo supervised withdrawal during the first 90 days of the study of any other medication used specifically as treatment of scleroderma-related interstitial lung disease with confirmed stable pulmonary status.
You have a skin thickness score between 15 and 45.
You are willing to undergo supervised withdrawal during the first 90 days of the study of any other prohibited medications with confirmed stable status.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 1st, 2021

Last Reviewed: October 1st, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.