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Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis

NCT03770273 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2024-12-17

Study results available
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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.

Conditions

  • Indolent Systemic Mastocytosis

Interventions

BIOLOGICAL

sarilumab

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.

OTHER

Placebo

Placebo

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Hirsh D Komarow, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-06-19
Primary Completion
2023-07-31
Completion
2023-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03770273 on ClinicalTrials.gov