Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Patients With Systemic Sclerosis With Interstitial Lung Disease

• Status: Terminated
• Trial end date: 2016-11-03
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Celgene; Celgene Corporation
• Treatments: Pomalidomide; Thalidomide

Criteria

Key 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:

• 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] OR

• FVC readings ≥ 70% and ≤ 80% at Screening and Baseline (Visit 2) with a documented history of either or both of:

1. 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.

2. A high resolution computed tomography (HRCT) fibrosis score > 20%

• 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.

Key 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