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A Study to Evaluate the Safety and Tolerability of Oral Ixazomib in Scleroderma-related Lung Disease Patients
Phase 2 trial testing Ixazomib in Systemic Sclerosis in 4 participants. Terminated before completion.
| Lead sponsor | Michael M. Pham |
|---|---|
| Phase | Phase 2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 4 |
| Start date | 28 April 2021 |
| Primary completion | 23 February 2024 |
| Estimated completion | 23 February 2024 |
| Sites | 1 location across United States |
Michael M. Pham
18 and older, any sex, with Systemic Sclerosis or Scleroderma. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
The number of participants with at least one treatment-emergent adverse event. Defined as any of the following: Treatment-emergent AEs; Treatment-emergent serious adverse events (SAEs); Treatment-emergent treatment-related AEs; or Treatment-emergent treatment-related SAEs.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 3 |
Number of participants with treatment-emergent AEs leading to ixazomib dose modifications.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 |
Number of participants with treatment-emergent AEs leading to ixazomib early discontinuation.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 |
The UCLA SCTC GIT 2.0 is a self-administered survey consisting of 34 questions (Reflux 1 to 8, Distention/Bloating 9 to 12, Fecal Soilage 13, Diarrhea 14 to 15, Social functioning 16 to 21, Emotional well-being 22 to 30, Constipation 31 to 34). The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health, except for questions 15 and 31, which are scored as 0 (better health) and 1 (worse health). Scores from all scales except the constipation scale are averaged to form a total GIT score from 0 (no gastrointestinal problems) to 3 (most severe) that ca
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | -0.09 | ± 0.156 |
MRSS is a validated clinical semiquantitative assessment of scleroderma skin thickness at 17 prespecified sites on the body, with thickness at each site scored from 0 (uninvolved) to 3 (severe thickening) that are tabulated for a total numerical skin score ranging 0-51. Lower scores indicate less involvement, and higher scores indicate more severe thickening.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | -4.33 | ± 11.02 |
FVC is the maximum amount of air a person can forcefully exhale from their lungs after taking a deep breath.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | -0.33 | ± 5.51 |
TLC is the total maximum amount of air that lungs can hold.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 5 | ± 39.11 |
Patient-reported outcome that represents the participant's assessment of his or her current overall health (Question: How was your overall health in the last week?) rated on an 11-point numeric scale anchored at 0 (excellent) to 10 (extremely poor) with higher scores indicating worse disease in terms of severity and damage.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 3.4 | ± 3.3 |
Physician-reported outcome that represents an assessment of the participant's current overall health rated on an 11-point numeric scale anchored at 0 (excellent) to 10 (extremely poor) with higher scores indicating worse disease in terms of severity and damage.
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 1.7 | ± 1.0 |
DLCO is the amount of carbon monoxide (CO) that moves from the lungs to the blood
| Group | Value | 95% CI |
|---|---|---|
| Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 0 | ± 5.30 |
Time frame: Adverse events were collected from baseline to completion of the post-treatment follow-up visit, approximately 16 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Ixazomib in Patients With … |
|---|---|---|
| Pruritus | Nervous system disorders | — |
| Rash | Skin and subcutaneous tissue disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Diarrhea | Gastrointestinal disorders | — |
| Fever | Infections and infestations | — |
| Hyponatremia | Nervous system disorders | — |
| Hypokalemia | Nervous system disorders | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — |
Data from ClinicalTrials.gov NCT04837131 adverse events section.
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.
7 peer-reviewed publications reference this trial (live from Europe PMC):
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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04837131.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing