study of pomalidomide (cc-4047) to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics and effectiveness for patients with systemic sclerosis with interstitial lung disease

Conditions: Scleroderma, Systemic; Sclerosis, Systemic; Systemic Scleroderma; Systemic Sclerosis; Interstitial Lung Disease

Summary: the primary objective of this study is to evaluate the safety, tolerability, and efficacy of pomalidomide in the treatment of patients with systemic sclerosis with interstitial lung disease.


Eligibility criteria:
inclusion criteria * male or females between 18 and 80 years of age (inclusive) at the time of consent. * diagnosis of systemic sclerosis (ssc) as defined by american college of rheumatology (acr) criteria. * onset of the first non-raynaud's manifestation of ssc within 7 years of screening. * subjects are required to meet at least one of the following 2 pulmonary-related criteria to be eligible for the study:. i) fvc readings ≥ 70% and ≤ 80% at screening and baseline (visit 2) with a documented history of either or both of:. a. a ≥ 5% decrease (expressed as percent predicted or in liters) in fvc in the 24-month period prior to baseline (visit 2) based on 3 or more assessments. two assessments may be done during the screening phase provided the assessments are completed at least 2 weeks apart. b. a high resolution computed tomography (hrct) fibrosis score \> 20%. ii) forced vital capacity (fvc) ≥ 45% and \<70% at screening and baseline (visit 2) \[with or without a documented pre-specified fvc decline or fibrosis score\]. * fvc at baseline (visit 2) within 5% of the fvc measured at screening. * carbon monoxide diffusing capacity (dlco) ≥ 35% and ≤ 80% of predicted value at screening. * abnormalities on high-resolution ct consistent with parenchymal changes encountered in ssc: honeycombing or reticular changes with or without ground glass. exclusion criteria * oxygen saturation (spo2) \< 92% (room air \[sea level\] at rest) at screening or baseline. * known diagnosis of obstructive lung disease as defined by forced expiratory volume (fev1)/fvc ratio \< 0.7. * diagnosis of pulmonary arterial hypertension (pah) requiring treatment. * known diagnosis of other significant respiratory disorders (e.g., asthma, tuberculosis, sarcoidosis, aspergillosis, chronic bronchitis, neoplastic disease, cystic fibrosis, etc.). * current clinical diagnosis of another inflammatory connective tissue disease (eg, systemic lupus erythematosus, rheumatoid arthritis, primary sjogren's syndrome, etc.). subjects having sjogren's syndrome secondary to ssc are eligible. * pregnant or lactating females. * history of a thromboembolic event (eg, deep vein thrombosis, thrombotic cerebrovascular or cardiovascular events). * history or current diagnosis of peripheral neuropathy. * use of concomitant medication(s) which could increase the risk for developing deep vein thrombosis, including sex steroid-based contraceptives (oral, injectable or implanted) and hormone replacement therapies, if use of a low-dose aspirin regimen is contraindicated. * additional concomitant medications which prolong the qt/qtc interval (measure of heart's electrical cycle) during the course of the study. * use of any anti-coagulant or anti-thrombotic medications (other than low dose-aspirin \[≤ 100 mg/day\]). * use of any cytotoxic/immunosuppressive agent (other than prednisone ≤ 10 mg/day \[mean dose\] or equivalent), including but not limited to azathioprine, cyclophosphamide, methotrexate, mycophenolate and cyclosporine within 28 days (4 weeks) of screening. * use of any biologic agent within 84 days (12 weeks) or 5 half-lives of screening. in the case of rituximab, use within 168 days (24 weeks) of screening or no recovery of cd20-positive b lymphocytes if the last dose of rituximab has been more than 24 weeks prior to screening. * use of bosentan, ambrisentan, sildenafil, tadalafil and macitentan for pah within 28 days (4 weeks) of screening. * use of medications (e.g., d-penicillamine, potaba) with putative scleroderma disease-modifying properties within 4 weeks of screening. * use of melphalan within 52 weeks of screening. * use of any investigational drug within 4 weeks of screening or 5 pharmacodynamic/pharmacokinetic half-lives if known (whichever is longer). * smoking of cigars, pipes or cigarettes within 24 weeks of screening. * other protocol-defined inclusion/exclusion criteria apply.