A Phase 2, Proof-Of-Concept, Multicenter, Randomized, Double-Blind, Placebo- Controlled, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Pomalidomide (CC-4047) in Subjects With Systemic Sclerosis With Interstitial Lung Disease

CC-4047-SSC-001

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.

2012-08-09

2016-11-03

2016-11-03

Terminated

Early phase I

23

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.

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2023-12-01