Nilotinib in the Treatment of Systemic Sclerosis
A phase IIa open-label single center pilot study to assess the safety and efficacy of Nilotinib in patients with Scleroderma.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase IIA Study of the Safety and Tolerability of the Use of Nilotinib in the Treatment of Systemic Sclerosis|
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 6 Months ] [ Designated as safety issue: Yes ]
- Efficacy of Nilotinib in patients with systemic sclerosis, as defined by an improvement in the Modified Rodnan skin score and indices of pulmonary function. [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
|Study Start Date:||July 2010|
|Estimated Primary Completion Date:||July 2012 (Final data collection date for primary outcome measure)|
Drug: Nilotinib (Tasigna)
The purpose of this study is to learn how safe and tolerable a medication called Nilotinib (Tasigna) will be for patients diagnosed with Systemic Sclerosis. Systemic Sclerosis (scleroderma) is an autoimmune disease that can involve the skin, the blood vessels, the muscles and other connective tissues, and major organs including the lungs, kidneys, gastrointestinal tract, and heart. The exact cause of this disorder is not known at this time and no drug has been proven to cure scleroderma. Experiments done in animal models and "test-tube" models of fibrosis suggest that Nilotinib may be a useful therapy for scleroderma. Nilotinib is a medication on the market which has been FDA approved for the treatment of a type of leukemia called chronic myelogenous leukemia (CML). It is an oral medication, taken two times a day.
This is a 32 week, open-label, Phase IIa, single center clinical trial. The primary goal of the study is to assess the safety and tolerability of Nilotinib in patients with scleroderma. The secondary goal is to assess how effective Nilotinib is in treating patients with scleroderma. The clinical tests performed such as the Modified Rodnan Skin Score, Pulmonary Function Tests, Echocardiograms, Electrocardiograms, and the blood and skin collected in this study will help determine whether this therapy is safe and effective, and also improve our understanding of scleroderma.
|United States, New York|
|Hospital for Special Surgery|
|New York, New York, United States, 10021|
|Principal Investigator:||Robert Spiera, MD||Hospital for Special Surgery, New York|