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Phase IIA Study of the Safety and Tolerability of the Use of Imatinib Mesylate (Gleevec) in the Treatment of Systemic Sclerosis

Phase 2
18 Years
Open (Enrolling)
Systemic Sclerosis

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Trial Information

Phase IIA Study of the Safety and Tolerability of the Use of Imatinib Mesylate (Gleevec) in the Treatment of Systemic Sclerosis

Further description of extension phase:

Patients will first be evaluated for inclusion in the extension phase of the study at either
the follow-up visit following three month withdrawal from Gleevec treatment or at the visit
after the follow-up visit. Patients can undergo evaluation for inclusion in the extension
phase up to six months following their completion of the initial one year trial. All
patients evaluated for inclusion in the extension phase will sign a new informed consent
form detailing the purpose and procedures associated with the extension phase at the initial
visit. After the initial visit, patients who meet inclusion criteria will be required to
undergo evaluation every three months for the 27 months following initiation of treatment,
or more frequently if deemed clinically necessary. Treatment will consist of Gleevec, at
doses ranging from 100 to 400 mg daily (100 mg pills will be distributed for oral
administration). At each study visit, a history and physical exam will be performed and
urine and blood tests will be conducted for disease activity and organ function. Additional
blood for research may also be collected at study visits. The Modified Rodnan Skin Score
will be measured to assess the degree of skin involvement associated with the patient's
disease. Patients will also continue to complete questionnaires about their ability to
function and quality of life.

Patients will be financially responsible for all professional and clinical services, as well
as all laboratory and diagnostic tests, associated with the extension phase of the study.
All co-pays, deductibles and co-insurances will be paid by the participants. Any additional
costs for parking and travel the patients incur as a result of participating in the
extension phase will not be reimbursed by the study. Novartis Pharmaceuticals will donate
drug supply.

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 Gleevec
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 10 years.

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 10 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. Ongoing treatment with other immunosuppressive therapies including cyclophosphamide,
azathioprine, mycophenolic acid, methotrexate, or cyclosporine, or use of those
medications within 3 months of trial entry.

5. 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, serum creatinine of
greater than 2.0, active infection, severe diabetes, unstable atherosclerotic
cardiovascular disease, malignancy, HIV, or severe peripheral vascular disease.

6. The use of other anti-fibrotic agents including colchicine, D-penicillamine,
minocycline, or Type 1 oral Collagen in the three months prior to enrollment.

7. Limited scleroderma.

8. Systemic sclerosis-like illness associated with environmental or ingested agents such
as toxic rapeseed oil, vinyl chloride, or bleomycin.

9. A positive pregnancy 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.

10. 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
but not be increased during the course of the study.

11. Participation in another clinical research study involving the evaluation of another
investigational drug within ninety days of entry into this study.

12. The presence of severe lung disease as defined by a diffusion capacity of less than
30% of predicted.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety and tolerability of Gleevec in systemic sclerosis, as defined by an improvement in the Modified Rodnan skin score and indices of pulmonary function.

Outcome Time Frame:

16 months for initial phase, 27 months for extension

Safety Issue:


Principal Investigator

Robert Spiera, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Hospital for Special Surgery, New York


United States: Institutional Review Board

Study ID:




Start Date:

August 2007

Completion Date:

December 2013

Related Keywords:

  • Systemic Sclerosis
  • Systemic Sclerosis
  • Scleroderma, Systemic
  • Scleroderma, Diffuse
  • Sclerosis



Hospital for Special Surgery New York, New York  10021