Nilotinib in the Treatment of Systemic Sclerosis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2012 by Hospital for Special Surgery, New York.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborators:
Rudolph Rupert Scleroderma Program
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Hospital for Special Surgery, New York
ClinicalTrials.gov Identifier:
NCT01166139
First received: July 1, 2010
Last updated: March 1, 2012
Last verified: March 2012
  Purpose

A phase IIa open-label single center pilot study to assess the safety and efficacy of Nilotinib in patients with Scleroderma.


Condition Intervention Phase
Systemic Sclerosis
Drug: Nilotinib (Tasigna)
Phase 2

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Hospital for Special Surgery, New York:

Primary Outcome Measures:
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 6 Months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 10
Study Start Date: July 2010
Estimated Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Nilotinib (Tasigna)
    Patients will be treated with Nilotinib 400 mg two times a day for 6 months.
Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age greater than or equal to eighteen years.
  2. Clinical diagnosis of diffuse systemic sclerosis by ACR criteria, with a stable modified Rodnan skin score in the one month preceding introduction of oral nilotinib therapy. The modified Rodnan skin score must be greater than or equal to sixteen at screening and initiation of therapy.
  3. Disease duration of less than or equal to 3 years as defined by the date of onset of the first non-Raynaud's symptom.
  4. Estimated ejection fraction of greater than 50% by echocardiography

Exclusion Criteria:

  1. Inability to render informed consent in accordance with institutional guidelines.
  2. Disease duration of greater than 3 years.
  3. Patients with mixed connective tissue disease or "overlap" (i.e. those who satisfy more than one set of ACR criteria for a rheumatic disease.)
  4. Limited scleroderma.
  5. Systemic sclerosis-like illness associated with environmental or ingested agents such as toxic rapeseed oil, vinyl chloride, or bleomycin.
  6. Ongoing treatment with immunosuppressive therapies including cyclophosphamide, azathioprine, mycophenolic acid, methotrexate, or cyclosporine, or use of those medications within 1 month of trial entry.
  7. The use of other anti-fibrotic agents including colchicine, D-penicillamine, minocycline, or Type 1 oral Collagen in the month prior to enrollment.
  8. Use in the prior month of corticosteroids at doses exceeding the equivalent of prednisone 10 mg daily. Use of corticosteroid at < 10 mg of prednisone can continue during the course of the study.
  9. Concurrent serious medical condition which in the opinion of the investigator makes the patient inappropriate for this study such as uncontrollable CHF, arrhythmia, severe pulmonary or systemic hypertension, severe GI involvement, hepatic impairment, serum creatinine of greater than 2.0, active infection, severe diabetes, unstable atherosclerotic cardiovascular disease, malignancy, HIV, or severe peripheral vascular disease.
  10. History of pancreatitis.
  11. Prolonged QTc interval defined as a QTc > 450 msec
  12. Patients requiring the ongoing use of medications that are antiarrhythmics (including, but not limited to amiodarone, disopyramide, procainamide, quinidine and sotalol) or that prolong the QTc interval (including, but not limited to chloroquine, halofantrine, clarithromycin, haloperidol, methadone, moxifloxacin, bepridil and pimozide) will be excluded.
  13. Patients requiring the ongoing use of medications that are potent inhibitors or inducers of CYP3A4.
  14. A positive pregnancy test at entry into this study. Men and women with reproductive potential will be required to use effective means of contraception through the course of the study.
  15. Participation in another clinical research study involving the evaluation of another investigational drug within ninety days of entry into this study.
  16. The presence of severe lung disease as defined by a diffusion capacity of less than 30% of predicted.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01166139

Locations
United States, New York
Hospital for Special Surgery
New York, New York, United States, 10021
Sponsors and Collaborators
Hospital for Special Surgery, New York
Rudolph Rupert Scleroderma Program
Novartis Pharmaceuticals
Investigators
Principal Investigator: Robert Spiera, MD Hospital for Special Surgery, New York
  More Information

No publications provided

Responsible Party: Hospital for Special Surgery, New York
ClinicalTrials.gov Identifier: NCT01166139     History of Changes
Other Study ID Numbers: 10041
Study First Received: July 1, 2010
Last Updated: March 1, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by Hospital for Special Surgery, New York:
Systemic Sclerosis

Additional relevant MeSH terms:
Scleroderma, Systemic
Scleroderma, Diffuse
Sclerosis
Connective Tissue Diseases
Skin Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on August 27, 2014