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Systemic SclerosisPhase 2NCT01166139

Nilotinib in the Treatment of Systemic Sclerosis

A Phase 2 study testing Nilotinib (Tasigna) in people with Systemic Sclerosis, run by Hospital for Special Surgery, New York at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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

Who is it for?
Ages 18 years and older
Conditions the study is for
Systemic Sclerosis
What is being tested?
Nilotinib (Tasigna)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
Jul 2010to Jan 2015
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age greater than or equal to eighteen years.
  • 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.
  • Disease duration of less than or equal to 3 years as defined by the date of onset of the first non-Raynaud's symptom.
  • Estimated ejection fraction of greater than 50% by echocardiography

Who cannot join

  • Inability to render informed consent in accordance with institutional guidelines.
  • Disease duration of greater than 3 years.
  • Patients with mixed connective tissue disease or "overlap" (i.e. those who satisfy more than one set of ACR criteria for a rheumatic disease.)
  • Limited scleroderma.
  • Systemic sclerosis-like illness associated with environmental or ingested agents such as toxic rapeseed oil, vinyl chloride, or bleomycin.
  • Ongoing treatment with immunosuppressive therapies including cyclophosphamide, azathioprine, mycophenolic acid, methotrexate, or cyclosporine, or use of those medications within 1 month of trial entry.
Show all 16Show fewer
  • The use of other anti-fibrotic agents including colchicine, D-penicillamine, minocycline, or Type 1 oral Collagen in the month prior to enrollment.
  • 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.
  • 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.
  • History of pancreatitis.
  • Prolonged QTc interval defined as a QTc > 450 msec
  • 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.
  • Patients requiring the ongoing use of medications that are potent inhibitors or inducers of CYP3A4.
  • 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.
  • Participation in another clinical research study involving the evaluation of another investigational drug within ninety days of entry into this study.
  • The presence of severe lung disease as defined by a diffusion capacity of less than 30% of predicted.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Hospital for Special Surgery

    New York, New York, 10021

Other trials in United States

About this study

Official title: Phase IIA Study of the Safety and Tolerability of the Use of 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.

Read the 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.

Being tested
Nilotinib (Tasigna)
Keywords
Systemic Sclerosis
Medical subject headings
Scleroderma, Systemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Nilotinib (Tasigna)Drug

    Patients will be treated with Nilotinib 400 mg two times a day for 6 months.

Study groups in NCT01166139
Study groupRoleWhat they receive
Nilotinib 400 mg twice dailyExperimentalNot described
Receives: Drug: Nilotinib (Tasigna)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Adverse Events as a Measure of Safety and Tolerability

    Measured: 6 Months and 12 months treatment

Secondary outcomes (2)

  1. Improvement of Modified Rodnan Skin Score Reported as a Mean (Units Equals Number of Points)

    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.

    Measured: 6 Months of treatment

  2. Efficacy of Nilotinib in Patients With Systemic Sclerosis, as Defined by an Improvement in the Modified Rodnan Skin Score

    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.

    Measured: 12 months treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Jul 2010 (estimated)
  2. Study startJul 2010
  3. Primary completionJul 2014
  4. Study completionJan 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01166139
Sponsor's protocol ID
10041
Status last verified
Oct 2017
First submitted
1 Jul 2010
Last updated
4 Oct 2017
Data monitoring committee
Yes

Publications

  • Haddon DJ, Wand HE, Jarrell JA, Spiera RF, Utz PJ, Gordon JK, Chung LS. Proteomic Analysis of Sera from Individuals with Diffuse Cutaneous Systemic Sclerosis Reveals a Multianalyte Signature Associated with Clinical Improvement during Imatinib Mesylate Treatment. J Rheumatol. 2017 May;44(5):631-638. doi: 10.3899/jrheum.160833. Epub 2017 Mar 15.

    PubMed 28298564

  • Gordon JK, Martyanov V, Magro C, Wildman HF, Wood TA, Huang WT, Crow MK, Whitfield ML, Spiera RF. Nilotinib (Tasigna) in the treatment of early diffuse systemic sclerosis: an open-label, pilot clinical trial. Arthritis Res Ther. 2015 Aug 18;17(1):213. doi: 10.1186/s13075-015-0721-3.

    PubMed 26283632

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Nilotinib (Tasigna) in people with Systemic Sclerosis, run by Hospital for Special Surgery, New York at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Oct 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events as a Measure of Safety and Tolerability (time frame: 6 Months and 12 months treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01166139, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2017.

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