Trial · NCT03770273
Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis
NCT03770273, Phase 2, Completed, National Institute of Allergy and Infectious Diseases (NIAID); results first posted 2024-12-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis. Trials, record NCT03770273. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03770273
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis
- NCT number
- NCT03770273
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 21 participants, actual
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- Conditions
- Indolent Systemic Mastocytosis
- Interventions
- sarilumab, Placebo
- First posted
- 2018-12-10
- Last updated
- 2024-12-17
- Results first posted
- 2024-12-17
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL) | Baseline and 16-week post-initiation of study | The Mastocytosis Quality of Life Questionnaire (MC-QoL) is a validated health-related quality of life (QoL) survey for patients with mastocytosis that consists of 27 items and is divided into four domains: symptoms, social life/functioning, emotions, and skin. Each item has five options scored from 0-4 for a total minimum score of zero and maximum score of 108. The raw scores were transformed to a 0 to 100 scale. The overall disease impairment on QoL is measured by assessing both the total score and the scores of each domain with higher scores of 25% or greater indicating a higher QoL impairment. The percent change in QoL from baseline to 16 weeks post-initiation of study for participants was computed as \[(Baseline QoL - 16-week QoL)/Baseline QoL\] × 100\]. Positive value indicates improvement. |
| Primary | Number of Participants With Adverse Events by Grade | Up to 32 weeks post initiation of study | Severity of adverse event by grade using Terminology Criteria for Adverse Events (CTCAE) version 5.0: Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental activity of daily living (ADL). Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activity of daily living (ADL). Grade 4 Life-threatening consequences; urgent intervention indicated. Grade 5 Death related to adverse event |
| Secondary | Percent Change in Quality of Life (QoL) Using the Memorial Symptom Assessment Scale (MSAS) | Baseline and 16-week post-initiation of study | The Memorial Symptom Assessment Scale (MSAS) is used to evaluate a patient's experience with 32 symptoms over the course of the previous week. Symptoms are evaluated by severity (0 = not at all to 4 = very severe) and distress (0 = not at all to 4 = very much\] for eight of the symptoms, and for severity, distress, and frequency (0 = not at all to 4 = almost constantly) for the remaining 24 symptoms for a total minimum score of zero and maximum score of 352. Higher score indicates worsening condition. The result is calculated by taking the average of the scores for the 32 questions for baseline and week 16 post initiation of study. The percent change in quality of life (QoL) from baseline to 16 weeks post-initiation of study for participants was computed as \[(Baseline QoL - 16-week QoL)/Baseline QoL\] × 100\]. Positive value indicates improvement. |
| Secondary | Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MQLQ) | Baseline and 16-week post-initiation of study | The Mastocytosis Quality of Life Questionnaire (MQLQ) is disease specific quality of life questionnaire consisting of 49 questions to assess the effect of mastocytosis on daily life. It includes the role of mastocytosis in 8 domains: fatigue and mental health, anaphylaxis, bone symptoms, unfamiliarity (low awareness), flushing, general symptoms, skin symptoms, and triggers. Each item in the 49 questions has seven options scored from 0-6 with a minimum total score of zero and a maximum total score of 294. Higher score indicates worsening condition. The result is calculated by summing scores from all 49 items for baseline and week 16 post initiation of study. The percent change in QoL from baseline to 16 weeks post-initiation of study for participants was computed as \[(Baseline QoL - 16-week QoL)/Baseline QoL\] × 100\]. Positive value indicates improvement. |
| Secondary | Percent Change in Quality of Life (QoL) Using Mastocytosis Symptoms Assessment Form (MSAF) | Baseline and 16-week post-initiation of study | The Mastocytosis Symptoms Assessment Form (MSAF) is a 22-item questionnaire that assesses the severity of symptoms with an 11-point numeric rating scale, where "0 = No symptom" and "10 = Worst imaginable symptom" for a total minimum score of zero and total maximum score of 220. Higher score indicates worsening condition. The result is calculated by summing scores from all 22 items for baseline and week 16 post initiation of study. The percent change in QoL from baseline to 16 weeks post-initiation of study for participants was computed as \[(Baseline QoL - 16-week QoL)/Baseline QoL\] × 100\]. Positive value indicates improvement. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL) | Baseline and 16-week post-initiation of study | Modified Intent-to-Treat (mITT) participants who completed questionnaire during the blinded phase of the study. One participant did not complete study during the blinded phase so was excluded from analysis. | percentage of change in quality of life | LEAST_SQUARES_MEAN |
| PRIMARY | Number of Participants With Adverse Events by Grade | Up to 32 weeks post initiation of study | Modified Intent-to-Treat (mITT) participants who completed questionnaire during the blinded phase of the study. One participant did not complete study during the blinded phase so was excluded from analysis. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percent Change in Quality of Life (QoL) Using the Memorial Symptom Assessment Scale (MSAS) | Baseline and 16-week post-initiation of study | Modified Intent-to-Treat (mITT) participants who completed questionnaire during the blinded phase of the study. One participant did not complete study during the blinded phase so was excluded from analysis. | percentage of change in quality of life | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MQLQ) | Baseline and 16-week post-initiation of study | Modified Intent-to-Treat (mITT) participants who completed questionnaire during the blinded phase of the study. One participant did not complete study during the blinded phase so was excluded from analysis. | percentage of change in quality of life | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change in Quality of Life (QoL) Using Mastocytosis Symptoms Assessment Form (MSAF) | Baseline and 16-week post-initiation of study | Modified Intent-to-Treat (mITT) participants who completed questionnaire during the blinded phase of the study. One participant did not complete study during the blinded phase so was excluded from analysis. | percentage of change in quality of life | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL) | Drug: Sarilumab | 38.98 | 95% Confidence Interval 28.83 to 49.13 | percentage of change in quality of life | |
| Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL) | Placebo | 33.02 | 95% Confidence Interval 22.17 to 43.88 | percentage of change in quality of life | |
| Number of Participants With Adverse Events by Grade | Drug: Sarilumab | Grade 1 | 7 | Participants | |
| Number of Participants With Adverse Events by Grade | Placebo | Grade 1 | 6 | Participants | |
| Number of Participants With Adverse Events by Grade | Drug: Sarilumab | Grade 2 | 6 | Participants | |
| Number of Participants With Adverse Events by Grade | Placebo | Grade 2 | 3 | Participants | |
| Number of Participants With Adverse Events by Grade | Drug: Sarilumab | Grade 3 | 2 | Participants | |
| Number of Participants With Adverse Events by Grade | Placebo | Grade 3 | 2 | Participants | |
| Number of Participants With Adverse Events by Grade | Drug: Sarilumab | Grade 4 | 0 | Participants | |
| Number of Participants With Adverse Events by Grade | Placebo | Grade 4 | 0 | Participants |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Drug: Sarilumab | 8 | |
| Overall Study | STARTED | Placebo | 8 | |
| Overall Study | COMPLETED | Drug: Sarilumab | 5 | |
| Overall Study | COMPLETED | Placebo | 7 | |
| Overall Study | NOT COMPLETED | Drug: Sarilumab | 3 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events112
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 | Eosinophilia | Blood and lymphatic system disorders | Drug: Sarilumab | 0 | 8 | |
| other | Eosinophilia | Blood and lymphatic system disorders | Placebo | 1 | 8 | |
| other | Dizziness | Cardiac disorders | Drug: Sarilumab | 1 | 8 | |
| other | Dizziness | Cardiac disorders | Placebo | 0 | 8 | |
| other | Palpitations | Cardiac disorders | Drug: Sarilumab | 0 | 8 | |
| other | Palpitations | Cardiac disorders | Placebo | 1 | 8 | |
| other | Ear pain | Ear and labyrinth disorders | Drug: Sarilumab | 1 | 8 | |
| other | Ear pain | Ear and labyrinth disorders | Placebo | 0 | 8 | |
| other | Eye pain | Eye disorders | Drug: Sarilumab | 1 | 8 | |
| other | Eye pain | Eye disorders | Placebo | 0 | 8 |
1 to 10 of 112Source ClinicalTrials.gov
See all 112 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Drug: Sarilumab | 0 | 8 |
| Total, all-cause mortality | Placebo | 0 | 8 |
| Total, other adverse events | Drug: Sarilumab | 7 | 8 |
| Total, other adverse events | Placebo | 7 | 8 |
| Total, serious adverse events | Drug: Sarilumab | 1 | 8 |
| Total, serious adverse events | Placebo | 0 | 8 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40529483 | DERIVED |
Source PubMed
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Drug: Sarilumab vs Placebo: deaths from any cause | Trial comparisons |
| Drug: Sarilumab vs Placebo: serious adverse events | Trial comparisons |
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/NCT03770273
GET https://conductscience.org/api/trials/v1/NCT03770273/events?page=2