SNPMiner Trials by Shray Alag

SNPMiner Trials: Clinical Trial Report

Report for Clinical Trial NCT03770273

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

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. 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.

NCT03770273 Indolent Systemic Mastocytosis
MeSH: Mastocytosis Mastocytosis, Systemic
HPO: Mastocytosis

2 Interventions

Name: Placebo

Description: Placebo

Type: Other


Name: Sarilumab

Description: Sarilumab is a fully human anti-IL-6R-alpha monoclonal antibody that binds membrane-bound and soluble human IL-6R and has been shown to inhibit IL-6 signaling

Type: Biological


Primary Outcomes

Measure: Frequency and severity of adverse events (AEs) during the randomized double-blinded placebo-controlled treatment period.

Time: day 0 through week 28

Measure: QoL at 16 weeks post-initiation of study drug/placebo using the Mastocytosis Quality of Life Questionnaire (MC-QoL).

Time: 16 weeks post study drug initiation

Secondary Outcomes

Measure: Decrease in the allelic frequency of D816V using PCR

Time: Day 0, week 16 and week 28 for D816V allelic frequency by PCR

Measure: Percent improvement in QoL using MC-QoL, scoring of mastocytosis index (SCORMA), and Memorial Symptom Assessment Scale (MSAS)

Time: Day 0 through Week 28

Measure: Reduction in use of medicines for symptomatic relief, reduction in serum levels of tryptase

Time: Day 0 through Week 28

Measure: Reduction of percentage infiltrating mast cells in bone marrow

Time: Day 0 and Week 16 only for bone marrow

Purpose: Treatment

Allocation: Randomized

Parallel Assignment

There is one SNP


1 D816V

Decrease in the allelic frequency of D816V using PCR. --- D816V ---

HPO Nodes

Genes 1