Phase 2
N=20
A Study Using The Experimental Drug Called Imatinib (Gleevec) in Subjects With Systemic Sclerosis
Alveolitis · Systemic Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT00512902 ↗Enrolled (actual)
20
Serious AEs
15.0%
Results posted
Oct 2014
Primary outcome: Primary: Treatment-related Adverse Events — 5 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Imatinib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- University of California, Los Angeles
- Primary completion
- Nov 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Treatment-related Adverse Events |
5 | — |
| SECONDARY Change in FVC (Forced Vital Capacity) |
1.74 | — |
| SECONDARY Change in TLC (Total Lung Capacity) |
4.17 | — |
| SECONDARY Change in DLco |
1.46 | — |
| SECONDARY Change in Modified Rodnan Skin Score (MRSS) |
3.9 | — |
Summary
The purpose of this study is to assess the safety and tolerability of imatinib (gleevec) in subjects who have systemic sclerosis. Imatinib has been approved by the FDA for the treatment of newly diagnosed adult patients with CML (newly diagnosed adult patients and for the treatment of patients with an accelerated phase. Imatinib is also approved for the treatment of patients with a certain type of gastrointestinal cancer (called stromal tumors) but it has not been approved to treat systemic sclerosis. Imatinib works by interfering with an enzyme called tyrosine phosphatase resulting in suppression of the immune system. It als interferes with a protein called platelet derived growth factor receptor (PDGFr) that has been linked to increased fibrosis.
Eligibility Criteria
Inclusion Criteria
- All patients must fulfill the criteria for SSc by ACR criteria
- Age of entry into the study ≥ 18 yrs
- FVC 50 mm Hg and/or mean PAP ≥30 mm Hg) pulmonary pressure or echocardiographic evidence of PAH (if echo cardiographic systolic pressure ≥ 55 mmHg) or FVC/DLCO ratio >1.6 on pulmonary function testing
- Persistent unexplained hematuria (>10 RBCs/hpf).
- History of persistent leukopenia (white blood cell count ULN.
- Pregnancy (documented by urine pregnancy test), breast feeding
- If of child-bearing potential, failure regularly to employ a reliable means of contraception
- Active infection of the lung or elsewhere, whose management would be compromised by Imatinib
- Unreliability, drug abuse (including active alcoholism)
- Any chronic, debilitating illness (other than SSc)
- Smoking of cigars, pipes or cigarettes during the past 6 months
- Baseline liver function tests (ALT or AST or bilirubin >1.5 x upper limit of normal
- Previous use of prednisone > 10 mg per day. If on prednisone ≤10 mg/d, dose must have been stable for > 1 month.
- All other medication with putative disease-modifying properties (e.g., D-penicillamine, cyclophosphamide, azathioprine, methotrexate, colchicine, Potaba) must be discontinued 1 month prior to beginning study medication.
- Patient is < 5 years since she/he had a primary malignancy except: if the other primary malignancy is not currently clinically significant nor requiring active intervention, or if other primary malignancy is a basal cell skin cancer or a cervical carcinoma in situ. Existence of any other malignant disease is not allowed except after consultation with the PI.
- Patient with Grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
- Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
- Patient has known chronic liver disease (i.e., chronic active hepatitis and cirrhosis).
- Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.
- Use of contraindicated medications at baseline.
Data sourced from ClinicalTrials.gov (NCT00512902). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.