Indolent Systemic MastocytosisPhase 2NCT03770273
Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis
A Phase 2 study testing sarilumab in people with Indolent Systemic Mastocytosis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
Background: Mast cells help the body fight disease and heal wounds. People with indolent systemic mastocytosis (ISM) make too many mast cells. This causes pain, tiredness, digestive problems, and other symptoms. Researchers think the drug sarilumab could help. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Indolent Systemic Mastocytosis
- What is being tested?
- sarilumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Jun 2019to 31 Jul 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must meet all of the following criteria to be enrolled in this study:
- Male or female participant greater than or equal to 18 and < 75 years of age at screening.
- Enrolled on NIAID protocol 02-I-0277.
- Documented pathologic diagnosis of ISM.
- Mc-QoL score of at least 25% (which suggests participant is at least somewhat affected by all Mc-QoL questions).
- Willing and able to undergo a bone marrow biopsy and aspirate.
Show all 18Show fewer
- Absolute neutrophil count (ANC) greater than or equal to 2000/mL.
- Hemoglobin greater than or equal to 12.0 g/dL (males), greater than or equal to 11 g/dL (females).
- Platelet count greater than or equal to 150,000/microliters.
- Alanine transaminase (ALT) and aspartate transaminase (AST) < 1.5 times the upper limit of normal (ULN).
- Willing to allow storage of blood and bone marrow for future use in medical research.
- Willing to allow genetic testing on biospecimens.
- Able to provide informed consent.
- Participants who can become pregnant must agree to use adequate contraception when engaging in sexual activities that can result in pregnancy. Adequate contraception must be used consistently, beginning at least 1 month before the beginning of dosing and lasting until 3 months after the final dose of study drug. Acceptable methods of contraception include the following:
- Hormonal contraception (non-oral only).
- Male or female condom with spermicide.
- Diaphragm or cervical cap with a spermicide.
- Intrauterine device.
Who cannot join
- Individuals meeting any of the following criteria will be excluded from study participation:
- Any abnormality that would be scored as a Grade 4 toxicity on the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Only clinically significant lab results will deem the subject ineligible
- Infected with HIV or has other known immunodeficiency.
- Has an active infection, including localized infection.
- Active diverticulitis.
- Active or chronic viral hepatitis.
Show all 18Show fewer
- Active or latent tuberculosis.
- Use of any other anti-IL-6 or anti-IL-6R agent within 1 year prior to the date informed consent was obtained.
- Use of cytoreductive therapy for mastocytosis within 1 year prior to the date informed consent was obtained.
- Known lymphoma or advanced and metastatic solid tumors on active therapy (including chemotherapy) within 1 year prior to the date informed consent was obtained
- Use of chemotherapy within 1 year prior to the date informed consent was obtained.
- Receipt of any marketed (eg, omalizumab) or investigational biologic or monoclonal antibody reported to affect mast cell activation within 5 half-lives prior to date informed consent was obtained.
- Receipt of intravenous (IV) immunoglobulin within 30 days prior to the date informed consent was obtained.
- Receipt of live attenuated vaccines within 30 days prior to the date informed consent was obtained.
- History of alcohol or drug/abuse within 12 months prior to date informed consent was obtained.
- Is allergic to any component of the sarilumab formulation.
- Pregnant or breastfeeding.
- Any condition that, in the opinion of the investigator, contraindicates participation in this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase 2 Randomized Double-Blinded Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in Subjects With Indolent Systemic Mastocytosis
Background:
Mast cells help the body fight disease and heal wounds. People with indolent systemic mastocytosis (ISM) make too many mast cells. This causes pain, tiredness, digestive problems, and other symptoms. Researchers think the drug sarilumab could help.
Read more
Objective:
To see if sarilumab is a safe and effective treatment for people with ISM.
Eligibility:
Adults ages 18-75 with ISM who are enrolled in NIH study 02-I-0277
Design:
Participants will be screened with:
- Physical exam
- Medical history
- Blood and urine tests
- Questionnaires
- Bone marrow removed by a needle inserted into the hip bone
- Ultrasound of the abdomen
- Photographs of the skin
Participants will repeat some screening tests at study visits.
Participants will have a baseline visit in the hospital for 3 days. They will:
- Be assigned to get either the study drug or a placebo. They will not know which one they get.
- Have a skin punch biopsy: An instrument will remove a small piece of skin.
- Get their first drug dose injected under their skin
Participants will keep a side effect and medication diary during the study.
Participants will visit the clinic to get a drug dose every 2 weeks, for a total of 8 doses.
Participants will have a visit 2 weeks after their final dose. It will last up to 2 days.
Participants will have another visit 12 weeks later.
Participants may then continue this study for 1 more year. Those who continue will get sarilumab, even if they previously got the placebo, every 2 weeks. They will have visits every 6 weeks, and then every 3 months.
Read the detailed description
Systemic mastocytosis is a disorder caused by clonal mast cell proliferation and release of mast cell mediators including tryptase. As a result, mast cell numbers may increase and affect target organs including the dermis (maculopapular cutaneous mastocytosis/urticaria pigmentosa, flushing), gastrointestinal tract (abdominal pain, diarrhea), skeletal system (osteoporosis), hematological system (anemia, thrombocytopenia), and spleen and liver (organomegaly). Patients with indolent (non-aggressive) systemic mastocytosis (ISM) are not candidates for cytoreductive therapy and are generally treated with symptomatic therapy that only partly decreases symptoms. There is, however, a documented association between severity of mastocytosis and elevated serum levels of interleukin (IL)-6. Furthermore, mast cells have been shown to double their rate of division and exhibit increased reactivity and release of mediators when cultured in the presence of IL-6. In addition, in an animal model of mastocytosis, anti-IL-6 has been shown to slow disease progression. In this study, adults with ISM will thus be randomized and treated with sarilumab, a recombinant monoclonal antibody directed against the IL-6 receptor, or receive placebo. Sarilumab is marketed in the United States as Kevzara (Sanofi/Genzyme [Cambridge, Massachusetts, USA ]) and is approved by the Food and Drug Administration for the treatment of rheumatoid arthritis. Binding of sarilumab to the IL-6 receptor inhibits IL-6-associated human mast cell signaling and proliferation with a resultant decrease in proliferation and reactivity (decreased mediator release), and therefore is a rational choice for the treatment of ISM.
In this study, participants will be randomized with approximately half of the participants receiving study drug, which will be administered at 200 mg via subcutaneous (SC) injection once every 2 weeks (Q2W) for a total of 16 weeks. The other participants will receive a placebo administered via SC injection Q2W for 16 weeks. Participants will return for a follow-up visit 2 weeks after the final dose (treatment peak), and then again 12 weeks later. Evaluations at study visits will include quality of life and symptom assessments and measurement of serum tryptase levels. Bone marrow examination will be performed at the onset and conclusion of the study. After the week 28 visit, all participants will have the option to continue sarilumab for 52 more weeks, at 200 mg administered via SC injections. Participants will continue to be monitored on a regular basis for safety concerns, as instructed in the study drug s package insert.
- Conditions
- Indolent Systemic Mastocytosis
- Being tested
- sarilumab, Placebo
- Keywords
- Cytokine, Monoclonal Therapy, Interleukin-6, Mast Cell, Bone Marrow
- Medical subject headings
- Mastocytosis, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sarilumabBiological
Sarilumab is a fully human anti-IL-6Rα monoclonal antibody that binds membrane-bound and soluble human IL-6R and has been shown to inhibit IL-6 signaling.
PlaceboOther
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Drug: sarilumab | Active comparator | Participants with indolent systemic mastocytosis receive sarilumab 200 mg (1.14 ml) subcutaneous injection every two weeks for 16 weeks (8 doses total) Receives: Biological: sarilumab |
| Placebo | Active comparator | Participants with indolent systemic mastocytosis receive placebo subcutaneous injection every two weeks for 16 week (8 doses total) Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL)
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.
Measured: Baseline and 16-week post-initiation of study
Number of Participants With Adverse Events by Grade
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
Measured: Up to 32 weeks post initiation of study
Secondary outcomes (3)
Percent Change in Quality of Life (QoL) Using the Memorial Symptom Assessment Scale (MSAS)
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.
Measured: Baseline and 16-week post-initiation of study
Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MQLQ)
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.
Measured: Baseline and 16-week post-initiation of study
Percent Change in Quality of Life (QoL) Using Mastocytosis Symptoms Assessment Form (MSAF)
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.
Measured: Baseline and 16-week post-initiation of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2018
- Study start19 Jun 2019
- Primary completion31 Jul 2023
- Study completion31 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Indolent Systemic MastocytosisStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Hirsh D Komarow, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03770273
- Sponsor's protocol ID
- 190027
- Other IDs
- 19-I-0027
- Status last verified
- 24 Aug 2023
- First submitted
- 7 Dec 2018
- Last updated
- 17 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: .Human data generated in this study will be shared for future research as follows: * De-identified data in an NIH-funded or approved public repository. * De-identified data in another public repository. * Identified data in the Biomedical Translational Research Information System (BTRIS, automatic for activities in the CC) and the Genomic Research Integration System (GRIS). * De-identified or identified data with approved outside collaborators under appropriate agreements.
Publications
Komarow HD, Wang J, Eisch R, Scott L, Brittain EH, Metcalfe DD. Treatment of indolent systemic mastocytosis with sarilumab is not supported in a randomized trial. J Allergy Clin Immunol Glob. 2025 May 21;4(3):100498. doi: 10.1016/j.jacig.2025.100498. eCollection 2025 Aug.
PubMed 40529483
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized Double-Blinded Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in Subjects With Indolent Systemic Mastocytosis
- ClinicalTrials.gov ID
- NCT03770273
- Acronym / sponsor ID
- 190027
- Conditions
- Indolent Systemic Mastocytosis
- Keywords
- Cytokine, Monoclonal Therapy, Interleukin-6, Mast Cell, Bone Marrow
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 19 Jun 2019 (actual)
- Primary completion
- 31 Jul 2023 (actual)
- Study completion
- 31 Jul 2023 (actual)
- First posted
- 10 Dec 2018 (actual)
- Results
- Posted 17 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
Mast cells help the body fight disease and heal wounds. People with indolent systemic mastocytosis (ISM) make too many mast cells. This causes pain, tiredness, digestive problems, and other symptoms. Researchers think the drug sarilumab could help.
Objective:
To see if sarilumab is a safe and effective treatment for people with ISM.
Eligibility:
Adults ages 18-75 with ISM who are enrolled in NIH study 02-I-0277
Design:
Participants will be screened with:
- Physical exam
- Medical history
- Blood and urine tests
- Questionnaires
- Bone marrow removed by a needle inserted into the hip bone
- Ultrasound of the abdomen
- Photographs of the skin
Participants will repeat some screening tests at study visits.
Participants will have a baseline visit in the hospital for 3 days. They will:
- Be assigned to get either the study drug or a placebo. They will not know which one they get.
- Have a skin punch biopsy: An instrument will remove a small piece of skin.
- Get their first drug dose injected under their skin
Participants will keep a side effect and medication diary during the study.
Participants will visit the clinic to get a drug dose every 2 weeks, for a total of 8 doses.
Participants will have a visit 2 weeks after their final dose. It will last up to 2 days.
Participants will have another visit 12 weeks later.
Participants may then continue this study for 1 more year. Those who continue will get sarilumab, even if they previously got the placebo, every 2 weeks. They will have visits every 6 weeks, and then every 3 months.
Detailed description
Systemic mastocytosis is a disorder caused by clonal mast cell proliferation and release of mast cell mediators including tryptase. As a result, mast cell numbers may increase and affect target organs including the dermis (maculopapular cutaneous mastocytosis/urticaria pigmentosa, flushing), gastrointestinal tract (abdominal pain, diarrhea), skeletal system (osteoporosis), hematological system (anemia, thrombocytopenia), and spleen and liver (organomegaly). Patients with indolent (non-aggressive) systemic mastocytosis (ISM) are not candidates for cytoreductive therapy and are generally treated with symptomatic therapy that only partly decreases symptoms. There is, however, a documented association between severity of mastocytosis and elevated serum levels of interleukin (IL)-6. Furthermore, mast cells have been shown to double their rate of division and exhibit increased reactivity and release of mediators when cultured in the presence of IL-6. In addition, in an animal model of mastocytosis, anti-IL-6 has been shown to slow disease progression. In this study, adults with ISM will thus be randomized and treated with sarilumab, a recombinant monoclonal antibody directed against the IL-6 receptor, or receive placebo. Sarilumab is marketed in the United States as Kevzara (Sanofi/Genzyme [Cambridge, Massachusetts, USA ]) and is approved by the Food and Drug Administration for the treatment of rheumatoid arthritis. Binding of sarilumab to the IL-6 receptor inhibits IL-6-associated human mast cell signaling and proliferation with a resultant decrease in proliferation and reactivity (decreased mediator release), and therefore is a rational choice for the treatment of ISM.
In this study, participants will be randomized with approximately half of the participants receiving study drug, which will be administered at 200 mg via subcutaneous (SC) injection once every 2 weeks (Q2W) for a total of 16 weeks. The other participants will receive a placebo administered via SC injection Q2W for 16 weeks. Participants will return for a follow-up visit 2 weeks after the final dose (treatment peak), and then again 12 weeks later. Evaluations at study visits will include quality of life and symptom assessments and measurement of serum tryptase levels. Bone marrow examination will be performed at the onset and conclusion of the study. After the week 28 visit, all participants will have the option to continue sarilumab for 52 more weeks, at 200 mg administered via SC injections. Participants will continue to be monitored on a regular basis for safety concerns, as instructed in the study drug s package insert.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Drug: sarilumab | Active comparator | Biological: sarilumabParticipants with indolent systemic mastocytosis receive sarilumab 200 mg (1.14 ml) subcutaneous injection every two weeks for 16 weeks (8 doses total) |
| Placebo | Active comparator | Other: PlaceboParticipants with indolent systemic mastocytosis receive placebo subcutaneous injection every two weeks for 16 week (8 doses total) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Male or female participant greater than or equal to 18 and < 75 years of age at screening.
- Enrolled on NIAID protocol 02-I-0277.
- Documented pathologic diagnosis of ISM.
- Mc-QoL score of at least 25% (which suggests participant is at least somewhat affected by all Mc-QoL questions).
- Willing and able to undergo a bone marrow biopsy and aspirate.
Key exclusion criteria (18 listed)
- Individuals meeting any of the following criteria will be excluded from study participation:
- Any abnormality that would be scored as a Grade 4 toxicity on the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Only clinically significant lab results will deem the subject ineligible
- Infected with HIV or has other known immunodeficiency.
- Has an active infection, including localized infection.
- Active diverticulitis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| sarilumabSarilumab is a fully human anti-IL-6Rα monoclonal antibody that binds membrane-bound and soluble human IL-6R and has been shown to inhibit IL-6 signaling. | Biological | Drug: sarilumab |
| PlaceboPlacebo | Other | Placebo |
Primary outcome
Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL)
Time frame: 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.
Number of Participants With Adverse Events by Grade
Time frame: 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
3 secondary outcomes
Percent Change in Quality of Life (QoL) Using the Memorial Symptom Assessment Scale (MSAS)
Time frame: Baseline and 16-week post-initiation of study
Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MQLQ)
Time frame: Baseline and 16-week post-initiation of study
Percent Change in Quality of Life (QoL) Using Mastocytosis Symptoms Assessment Form (MSAF)
Time frame: Baseline and 16-week post-initiation of study
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40529483Komarow HD, Wang J, Eisch R, Scott L, Brittain EH, Metcalfe DD. Treatment of indolent systemic mastocytosis with sarilumab is not supported in a randomized trial. J Allergy Clin Immunol Glob. 2025 May 21;4(3):100498. doi: 10.1016/j.jacig.2025.100498. eCollection 2025 Aug.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. .Human data generated in this study will be shared for future research as follows: * De-identified data in an NIH-funded or approved public repository. * De-identified data in another public repository. * Identified data in the Biomedical Translational Research Information System (BTRIS, automatic for activities in the CC) and the Genomic Research Integration System (GRIS). * De-identified or identified data with approved outside collaborators under appropriate agreements.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hirsh D Komarow, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing sarilumab in people with Indolent Systemic Mastocytosis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL) (time frame: Baseline and 16-week post-initiation of study). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03770273, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2024; AzTrials last refreshed its copy on 8 Nov 2026.