Nilotinib in the Treatment of Systemic Sclerosis
|Study Design:||Intervention Model: Single Group Assignment
Masking: No masking
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 ]
- 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 ]
|Study Start Date:||July 2010|
|Study Completion Date:||January 2015|
|Primary Completion Date:||July 2014 (Final data collection date for primary outcome measure)|
|Experimental: Nilotinib 400 mg twice daily||
Drug: Nilotinib (Tasigna)
Patients will be treated with Nilotinib 400 mg two times a day for 6 months.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01166139
|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|