Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Ind… (NCT03770273) | Clinical Trial Compass
CompletedPhase 2
Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis
United States21 participantsStarted 2019-06-19
Plain-language 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.
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.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
. 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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL)
Timeframe: Baseline and 16-week post-initiation of study
2
Number of Participants With Adverse Events by Grade
Timeframe: Up to 32 weeks post initiation of study
Trial details
NCT IDNCT03770273
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)
. Platelet count greater than or equal to 150,000/microliters.
Exclusion criteria
. 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.
. 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.