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Protocol Details

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

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Institute of Allergy and Infectious Diseases (NIAID)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 18 Years
Max Age: 75 Years

Referral Letter Required


Population Exclusion(s)

Pregnant Women


Monoclonal Therapy;
Mast Cell;
Bone Marrow

Recruitment Keyword(s)



Indolent Systemic Mastocytosis

Investigational Drug(s)


Investigational Device(s)



Biological/Vaccine: sarilumab
Other: Placebo

Supporting Site

National Institute of Allergy and Infectious Diseases


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.


To see if sarilumab is a safe and effective treatment for people with ISM.


Adults ages 18-75 with ISM who are enrolled in NIH study 02-I-0277


Participants will be screened with:

-Physical exam

-Medical history

-Blood and urine tests


-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.

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Participants must meet all of the following criteria to be enrolled in this study:

1. Male or female participant greater than or equal to 18 and < 75 years of age at screening.

2. Enrolled on NIAID protocol 02-I-0277.

3. Documented pathologic diagnosis of ISM.

4. MC-QoL score of at least 25% (which suggests participant is at least somewhat affected by all McQoL questions).

5. Willing and able to undergo a bone marrow biopsy and aspirate.

6. Absolute neutrophil count (ANC) greater than or equal to 2000/mL.

7. Hemoglobin greater than or equal to 12.0 g/dL (males), greater than or equal to 11 g/dL (females).

8. Platelet count greater than or equal to 150,000/microliters.

9. Alanine transaminase (ALT) and aspartate transaminase (AST) < 1.5 times the upper limit of normal (ULN).

10. Willing to allow storage of blood and bone marrow for future use in medical research.

11. Willing to allow genetic testing on biospecimens.

12. Able to provide informed consent.

13. 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.


Individuals meeting any of the following criteria will be excluded from study participation:

1. 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

2. Infected with HIV or has other known immunodeficiency.

3. Has an active infection, including localized infection.

4. Active diverticulitis.

5. Active or chronic viral hepatitis.

6. Active or latent tuberculosis.

7. Use of any other anti-IL-6 or anti-IL-6R agent within 1 year prior to the date informed consent was obtained.

8. Use of cytoreductive therapy for mastocytosis within 1 year prior to the date informed consent was obtained.

9. Known lymphoma or advanced and metastatic solid tumors on active therapy (including chemotherapy) within 1 year prior to the date informed consent was obtained

10. Use of chemotherapy within 1 year prior to the date informed consent was obtained.

11. 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.

12. Receipt of intravenous (IV) immunoglobulin within 30 days prior to the date informed consent was obtained.

13. Receipt of live attenuated vaccines within 30 days prior to the date informed consent was obtained.

14. History of alcohol or drug/abuse within 12 months prior to date informed consent was obtained.

15. Is allergic to any component of the sarilumab formulation.

16. Pregnant or breastfeeding.

17. Any condition that, in the opinion of the investigator, contraindicates participation in this study.

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Not Provided

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Principal Investigator

Referral Contact

For more information:

Hirsh D. Komarow, M.D.
National Institute of Allergy and Infectious Diseases (NIAID)
(301) 594-2197

Robin R. Eisch, R.N.
National Institute of Allergy and Infectious Diseases (NIAID)
National Institutes of Health
Building 10
Room 1C129A
10 Center Drive
Bethesda, Maryland 20892
(301) 443-1720

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1

Clinical Trials Number:


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