Research

Randomized, Double-Blind, Placebo-Controlled Trial of IL1-Trap, Rilonacept in Systemic Sclerosis-A Phase I/II Biomarker Trial (Old IRB #14115)

IRB Number: 2014-393

Institutional Review Board, Hospital for Special Surgery

The safety of study participants is our top priority. The trial is approved and periodically reviewed by an Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.

Before enrolling in a clinical trial, the investigator will explain the purpose of the trial, its expected benefits, any possible risks or side effects, and what your role will be. This is the time to ask questions! If you want to join the trial, you must sign the informed consent documents. You can leave a clinical trial at any time without penalty.

For further information, see Understanding Clinical Trials.

Principal Investigator

Jessica K. Gordon, MD

Co-Investigators

Robert F. Spiera, MD
Horatio Wildman, MD
Mylinh Duong
Annel Fernandez
Nicole Olivia
Karima Becetti
Emily Bakaj
Alexandra Morquette
 

Summary

This is a multi-center randomized, placebo-controlled, double-blind clinical trial that will recruit a total of 24 patients with early diffuse systemic sclerosis (scleroderma) to evaluate the safety and tolerability of rilonacept, a drug that has been FDA approved for the treatment of cryopin-associated periodic syndromes (CAPS), which is an inflammatory condition distinct from scleroderma. This study will be 12 weeks in duration. Two thirds of patients will be randomized to receive the study drug, rilonacept, and the other third will receive a placebo (normal saline solution). The rilonacept/placebo will be self-administered as a weekly injection for 6 weeks. The rilonacept/placebo will be supplied by the study sponsor. Participants will also have blood and skin samples taken for biopsies

Inclusion/Exclusion Criteria

Inclusion

Must meet the American College of Rheumatology criteria for systemic sclerosis with diffuse cutaneous involvement.
Onset of the first SSc manifestation other than Raynaud’s phenomenon must be <60 months.
Must have a MRSS of ≥ 15
Male or female patients ≥18 years of age.
Able and willing to give written informed consent and comply with the requirements of the study protocol

Exclusion:

•You are participating in another clinical research study involving the evaluation of another investigational drug within 4 weeks or 5 half-lives of the investigational drug of entry into this study.

•You are using corticosteroids at doses exceeding the equivalent of prednisone 10mg daily.

•You are receiving treatment with therapies that weaken your immune system including cyclophosphamide, azathioprine, methotrexate, or cyclosporine; or use of those medications within 1 month of trial entry.

•You are receiving (other than low dose steroids), cytotoxic or anti-fibrotic drug within 4 weeks of screening.

•You have tested positive for hepatitis B, hepatitis C or HIV on screening laboratory tests. If you tested positive for hepatitis B surface antibodies, you are still eligible if you have been immunized for hepatitis B and other tests are negative.

•You have a known, active bacterial, viral, fungal, mycobacterial, or other infection (including tuberculosis or atypical mycobacterial disease, but excluding fungal infections of nail beds) or any other episode of infection requiring hospitalization or treatment with i.v. antibiotics within 4 weeks of screening.

•You had a positive PPD or Quantiferon (tuberculosis test) and have not been treated with the appropriate antibiotics.

•You have a history of cancer within the past 5 years.

•You have moderate to severe liver impairment, (for example, Child-Pugh Class B or C).

•You have experienced scleroderma renal crisis (kidney failure due to high blood pressure) within 6 months or had a creatinine level greater than 2.0

•You are pregnant and/or nursing.

•You do not agree to use effective contraception while enrolled on study and receiving the experimental drug, and for at least 3 months after the last study treatment.

•You have had gastrointestinal problems requiring intravenous (IV) nutrition or hospitalization within the past 3 months for pseudo-obstruction.

•You have moderately severe lung disease that is characterized by a forced vital capacity of <60%, or a diffusing capacity of <50% predicted. These are values taken from a pulmonary function test that explain how much air your lungs can hold and how well they take up oxygen from the air.

•You have moderately severe heart disease with either a history of significant arrhythmia (not including conduction delays other than trifascicular block, or PVCs or PACs <5/minute), clinically significant heart failure, or unstable angina.

•You hemoglobin levels at screening were < 8.5 gm/dL. Hemoglobin is a protein in your blood that transports oxygen from your lungs to the rest of your body.

•Your white blood cell count was < 3,000/mm3 or your total neutrophil count was < 1,500 at screening. White blood cells and neutrophils are types of cells that part of your immune system.

•Your platelet count was < 100,000/mm3 at screening. Platelets help your blood clot and form scabs at injury sites.

Contact Information

Alexandra Morquette
212.774.7194
morquettea@hss.edu