Nilotinib in the Treatment of Systemic Sclerosis
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|ClinicalTrials.gov Identifier: NCT01166139|
Recruitment Status : Completed
First Posted : July 20, 2010
Results First Posted : October 4, 2017
Last Update Posted : October 4, 2017
|Condition or disease||Intervention/treatment||Phase|
|Systemic Sclerosis||Drug: Nilotinib (Tasigna)||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase IIA Study of the Safety and Tolerability of the Use of Nilotinib in the Treatment of Systemic Sclerosis|
|Study Start Date :||July 2010|
|Actual Primary Completion Date :||July 2014|
|Actual Study Completion Date :||January 2015|
|Experimental: Nilotinib 400 mg twice daily||
Drug: Nilotinib (Tasigna)
Patients will be treated with Nilotinib 400 mg two times a day for 6 months.
- Number of Participants With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 6 Months and 12 months treatment ]
- Improvement of Modified Rodnan Skin Score Reported as a Mean (Units Equals Number of Points) [ Time Frame: 6 Months of treatment ]Efficacy of Nilotinib in patients with systemic sclerosis, as defined by an improvement in the Modified Rodnan Skin Score (MRSS) The Modified Rodnan Skin Score (MRSS) measures dermal skin thickness through the examination of 17 body areas: fingers, hands, forearms, arms, feet, legs, and thighs (in pairs), and face, chest, and abdomen. The skin score is evaluated by manual palpation in each of these areas. The skin score is 0 for uninvolved skin, 1 for mild thickening, 2 for moderate thickening, and 3 for severe thickening (hidebound skin). The total skin score is the sum of the skin scores of the individual areas where the minimum score is 0 and the maximum score is 51. A higher score indicates greater severity of disease.
- Efficacy of Nilotinib in Patients With Systemic Sclerosis, as Defined by an Improvement in the Modified Rodnan Skin Score [ Time Frame: 12 months treatment ]
Improvement in Modified Rodnan skin score reported as a mean (units equals number of points).
The Modified Rodnan Skin Score (MRSS) measures dermal skin thickness through the examination of 17 body areas: fingers, hands, forearms, arms, feet, legs, and thighs (in pairs), and face, chest, and abdomen. In each of these areas, the skin score is evaluated by manual palpation. The skin score is 0 for uninvolved skin, 1 for mild thickening, 2 for moderate thickening, and 3 for severe thickening (hidebound skin). The total skin score is the sum of the skin scores of the individual areas. The minimum score is 0 and the maximum score is 51. A higher score indicates greater severity of disease.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|