Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Patients With Systemic Sclerosis With Interstitial Lung Disease
NCT ID: NCT01559129
Last Updated: 2023-12-01
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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TERMINATED
PHASE2
23 participants
INTERVENTIONAL
2012-08-09
2016-11-03
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Placebo
Placebo
Matching placebo capsules taken orally once a day
Pomalidomide
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Pomalidomide (CC-4047)
1 mg orally every day for 52 weeks
Interventions
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Pomalidomide (CC-4047)
1 mg orally every day for 52 weeks
Placebo
Matching placebo capsules taken orally once a day
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
18 Years
80 Years
ALL
No
Sponsors
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Celgene
INDUSTRY
Responsible Party
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Principal Investigators
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Shimon Korish, MD
Role: STUDY_DIRECTOR
Celgene Corporation
Locations
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UCLA Division of Rheumatology
Los Angeles, California, United States
Advances in Medicine
Rancho Mirage, California, United States
Delaware Medical Care Associates, LLC
Newark, Delaware, United States
Georgetown University School of Medicine
Washington D.C., District of Columbia, United States
USF Health Faculty Office Building-FOB
Tampa, Florida, United States
Arthritis Research and Treatment Center
Stockbridge, Georgia, United States
University of Illinois at Chicago
Chicago, Illinois, United States
LaPorte County Institute for Clinical Research, Inc
Michigan City, Indiana, United States
University of Kentucky
Lexington, Kentucky, United States
Louisiana State University
Shreveport, Louisiana, United States
Boston University of Medicine BUMC
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
UMDNJ-Robert Wood Johnson Medical School Clinical Research Center
New Brunswick, New Jersey, United States
North Shore-LIJ Health System-Division of Rheumatology
Great Neck, New York, United States
Cleveland Clinic
Cleveland, Ohio, United States
University of Toledo College of Medicine
Toledo, Ohio, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Texas Health Science Center at Houston
Houston, Texas, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Monash Medical Centre
Clayton, Victoria, Australia
The Prince Charles Hospital
Chermside, , Australia
The Queen Elizabeth Hospital
Woodville South, , Australia
CHRU de Lille FR
Lille, , France
Hopital Saint Louis
Paris, , France
Groupe Hospitalier Saint Vincent de Paul
Paris, , France
Kerckhoff-Klinik gGmbH
Bad Nauheim, , Germany
Charite - Universitätsmedizin Berlin
Berlin, , Germany
University of Erlangen-Nuremberg
Erlangen, , Germany
Klinikum der J.W. Gothe-Universitat Frankfurt
Frankfurt, , Germany
Rheumazentrum Ruhrgebiet
Herne, , Germany
University Hospital of Ulm
Ulm, , Germany
Azienda Ospedaliera Universitaria S. Martino di Genova
Genova, , Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, , Italy
Ospedale Luigi Sacco
Milan, , Italy
IRCCS Policlinico S. Matteo di Pavia
Pavia, , Italy
Azienda Ospedaliera Universitaria Pisana
Pisa, , Italy
Policlinico Umberto I
Roma, , Italy
Policlinico Universitario Agostino Gemelli
Roma, , Italy
Centrum Miriada Prywatny Gabinet Specjalistyczny Profesora Dra Stanislawa Sierakowskiego
Bialystok, , Poland
Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy
Bydgoszcz, , Poland
SPSK Nr 7 Slaskiego Uniwersytetu Medycznego, Oddzial Chorob Wewnetrznych i Reumatologii
Katowice, , Poland
Samodzielny Publiczny Szpital Kliniczny nr 1 im. prof.Tadeusza Sokolowskiego Pomorskiego UM w Szczec
Szczecin, , Poland
Instytut Reumatologii, Klinika i Poliklinika Ukladowych Chorób Tkanki Lacznej
Warsaw, , Poland
Akademicki Szpital Kliniczny Klinika Reumatologii i Chorob Wewnetrznych
Wroclaw, , Poland
Institution of the Russian Academy of Medical Sciences Research Institute of Rheumatology of the Ru
Moscow, , Russia
Penza Regional Clinical Hospital n.a. N.N. Burdenko
Penza, , Russia
St. Petersburg State Healthcare Institution "Clinical Rheumatology Hospital # 25"
Saint Petersburg, , Russia
Hospital Universitario Gregorio Maranon
Madrid, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Complejo Hospitalario de Santiago
Santiago de Compostela, , Spain
Hospital Universitario Dr. Peset
Valencia, , Spain
University Hospital Basel
Basel, , Switzerland
Chapel Allerton Hospital
Leeds, , United Kingdom
Royal Free Hospital
London, , United Kingdom
Royal Brompton Hospital - Interstitial Lung Disease Unit
London, , United Kingdom
Countries
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References
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Hsu VM, Denton CP, Domsic RT, Furst DE, Rischmueller M, Stanislav M, Steen VD, Distler JHW, Korish S, Cooper A, Choi S, Schafer PH, Horan G, Hough DR. Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study. J Rheumatol. 2018 Mar;45(3):405-410. doi: 10.3899/jrheum.161040. Epub 2017 Nov 1.
Hsu V, et al. A Phase 2 Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness in Subjects with Systemic Sclerosis with Interstitial Lung Disease. Presented at the 2016 ACR/ARHP Annual Meeting, November 11-16, 2016, Washington, DC. Abstract No. 823.
Other Identifiers
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2010-023047-15
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CC-4047-SSC-001
Identifier Type: -
Identifier Source: org_study_id