Nilotinib in the Treatment of Systemic Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01166139
• Date of registration: 01/07/2010
• Prospective Registration: Yes
• Primary sponsor: Hospital for Special Surgery, New York
• Public title: Nilotinib in the Treatment of Systemic Sclerosis
• Scientific title: Phase IIA Study of the Safety and Tolerability of the Use of Nilotinib in the Treatment of Systemic Sclerosis
• Date of first enrolment: July 2010
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01166139
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Robert Spiera, MD
• Affiliation: Hospital for Special Surgery, New York

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Systemic Sclerosis

Intervention(s)

Drug: Nilotinib (Tasigna)

Primary Outcome(s)

Number of Participants With Adverse Events as a Measure of Safety and Tolerability [Time Frame: 6 Months and 12 months treatment]

Secondary Outcome(s)

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 of Modified Rodnan Skin Score Reported as a Mean (Units Equals Number of Points) [Time Frame: 6 Months of treatment]

Secondary ID(s)

10041

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Rudolph Rupert Scleroderma Program

Novartis Pharmaceuticals

Results

• Results available: Yes
• Date Posted: 04/10/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01166139