Trial · NCT04837131
A Study to Evaluate the Safety and Tolerability of Oral Ixazomib in Scleroderma-related Lung Disease Patients
NCT04837131, Phase 2, Terminated, Michael M. Pham; results first posted 2025-04-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Safety and Tolerability of Oral Ixazomib in Scleroderma-related Lung Disease Patients. Trials, record NCT04837131. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04837131
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Safety and Tolerability of Oral Ixazomib in Scleroderma-related Lung Disease Patients
- NCT number
- NCT04837131
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 4 participants, actual
- Lead sponsor
- Michael M. Pham
- 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
- Ixazomib
- First posted
- 2021-04-08
- Last updated
- 2025-04-06
- Results first posted
- 2025-04-06
- Limitations and caveats
- The study was terminated due to low enrollment.
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Treatment-emergent Adverse Event (AE) | 7 months | 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. |
| Primary | Adverse Events Leading to Ixazomib Dose Modifications | 7 months | Number of participants with treatment-emergent AEs leading to ixazomib dose modifications. |
| Primary | Adverse Events Leading to Ixazomib Early Discontinuation | 7 months | Number of participants with treatment-emergent AEs leading to ixazomib early discontinuation. |
| Primary | Change in the UCLA Scleroderma Clinical Trials Consortium Gastrointestinal 2.0 (UCLA SCTC GIT 2.0) Questionnaire Score | Baseline, 7 months | 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 captures overall burden of severity of scleroderma-associated gastrointestinal involvement. |
| Secondary | Change From Baseline in Modified Rodnan Skin Score (MRSS) | Baseline, 24 weeks | 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. |
| Secondary | Change From Baseline in High Resolution Chest CT Scan Goh Score | Baseline, 24 weeks | Staging extent of scleroderma lung involvement on high resolution chest CT scan by the application of Goh criteria which identified a severity extent threshold of 20% lung involvement yields two cohorts with significantly different 10 year survivorship. Goh scores range 0-100% with a higher score indicating greater extent of lung involvement. |
| Secondary | Change in Forced Vital Capacity (FVC) % Predicted. | Baseline, 7 months | FVC is the maximum amount of air a person can forcefully exhale from their lungs after taking a deep breath. |
| Secondary | Change in Total Lung Capacity (TLC) % Predicted | Baseline, 24 weeks | TLC is the total maximum amount of air that lungs can hold. |
| Secondary | Severity of Dyspnea at Baseline (Mahler Baseline Dyspnea Index) | Baseline | Severity of dyspnea at baseline is measured with the Mahler Baseline Dyspnea Index (BDI). BDI includes 5 grades of severity from 0 (severe impairment) to 4 (no impairment ) for each of 3 categories (functional impairment; magnitude of task; magnitude of effort) yielding a summation focal score (0-12). Higher scores indicate lower impairment. |
| Secondary | Dyspnea Status Rating (Mahler Transitional Dyspnea Index) | 12 weeks, 24 weeks | Mahler Transitional Dyspnea Index (TDI) rates a participant's dyspnea status relative to baseline. TDI scores range from -3 (major deterioration) to +3 (major improvement) including 0 to indicate no change in status for each of the 3 BDI categories (functional impairment; magnitude of task; magnitude of effort) yielding a summation focal score (-9 to +9). Higher scores indicate major improvement. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Treatment-emergent Adverse Event (AE) | 7 months | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Adverse Events Leading to Ixazomib Dose Modifications | 7 months | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Adverse Events Leading to Ixazomib Early Discontinuation | 7 months | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Change in the UCLA Scleroderma Clinical Trials Consortium Gastrointestinal 2.0 (UCLA SCTC GIT 2.0) Questionnaire Score | Baseline, 7 months | score on a scale | MEAN | |
| SECONDARY | Change From Baseline in Modified Rodnan Skin Score (MRSS) | Baseline, 24 weeks | score on a scale | MEAN | |
| SECONDARY | Change From Baseline in High Resolution Chest CT Scan Goh Score | Baseline, 24 weeks | Chest CT scan not performed on any participants. Data not collected nor analyzed as study was terminated due to low enrollment. | ||
| SECONDARY | Change in Forced Vital Capacity (FVC) % Predicted. | Baseline, 7 months | percentage of air | MEAN | |
| SECONDARY | Change in Total Lung Capacity (TLC) % Predicted | Baseline, 24 weeks | percentage of air in lungs | MEAN | |
| SECONDARY | Severity of Dyspnea at Baseline (Mahler Baseline Dyspnea Index) | Baseline | Mahler Baseline Dyspnea Index was not completed by any subject. Data not collected nor analyzed as study was terminated due to low enrollment. | ||
| SECONDARY | Dyspnea Status Rating (Mahler Transitional Dyspnea Index) | 12 weeks, 24 weeks | Mahler Transitional Dyspnea Index was not completed by any subject. Data not collected nor analyzed as study was terminated due to low enrollment. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Treatment-emergent Adverse Event (AE) | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 3 | Participants | ||
| Adverse Events Leading to Ixazomib Dose Modifications | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 | Participants | ||
| Adverse Events Leading to Ixazomib Early Discontinuation | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 | Participants | ||
| Change in the UCLA Scleroderma Clinical Trials Consortium Gastrointestinal 2.0 (UCLA SCTC GIT 2.0) Questionnaire Score | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | -0.09 | Standard Deviation 0.156 | score on a scale | |
| Change From Baseline in Modified Rodnan Skin Score (MRSS) | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | -4.33 | Standard Deviation 11.02 | score on a scale | |
| Change in Forced Vital Capacity (FVC) % Predicted. | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | -0.33 | Standard Deviation 5.51 | percentage of air | |
| Change in Total Lung Capacity (TLC) % Predicted | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 5 | Standard Deviation 39.11 | percentage of air in lungs | |
| Change in Patient Global Assessment of Disease Activity (PtGA) | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 3.4 | Standard Deviation 3.3 | score on a scale | |
| Change in Physician Global Assessment of Disease Activity (MDGA) | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 1.7 | Standard Deviation 1.0 | score on a scale | |
| Change in Diffusion Capacity (DLCO) % Predicated | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 0 | Standard Deviation 5.30 | percentage of carbon monoxide |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 4 | |
| Overall Study | COMPLETED | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 0 | |
| Overall Study | NOT COMPLETED | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 4 |
Source ClinicalTrials.gov
Adverse events8
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pruritus | Nervous system disorders | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 | 4 | 3 |
| other | Rash | Skin and subcutaneous tissue disorders | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 | 4 | 2 |
| other | Vomiting | Gastrointestinal disorders | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 | 4 | 3 |
| other | Diarrhea | Gastrointestinal disorders | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 2 | 4 | 3 |
| other | Fever | Infections and infestations | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 1 | 4 | 2 |
| other | Hyponatremia | Nervous system disorders | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 1 | 4 | 2 |
| other | Hypokalemia | Nervous system disorders | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 1 | 4 | 1 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 1 | 4 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 1 | 4 |
| Total, other adverse events | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 3 | 4 |
| Total, serious adverse events | Ixazomib in Patients With Scleroderma-interstitial Lung Disease (ILD) | 0 | 4 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04837131
GET https://conductscience.org/api/trials/v1/NCT04837131/registered