Trial record 3 of 77 for:    Open Studies | "Scleroderma, Systemic"

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

This study is currently recruiting participants.
Verified March 2014 by Celgene Corporation
Sponsor:
Information provided by (Responsible Party):
Celgene Corporation
ClinicalTrials.gov Identifier:
NCT01559129
First received: March 19, 2012
Last updated: March 28, 2014
Last verified: March 2014
  Purpose

The purpose of this first study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of pomalidomide (CC-4047) in the treatment of subjects with systemic sclerosis with interstitial lung disease.


Condition Intervention Phase
Scleroderma, Systemic
Sclerosis, Systemic
Systemic Scleroderma
Systemic Sclerosis
Drug: Pomalidomide (CC-4047)
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Celgene Corporation:

Primary Outcome Measures:
  • Evaluation of the number of Systemic Sclerosis subjects with pomalidomide-related adverse events [ Time Frame: Up to 56 weeks ] [ Designated as safety issue: Yes ]
    Evaluation of the number of Systemic Sclerosis subjects with pomalidomide-related adverse events

  • Change from Baseline (Week 0) of the forced vital capacity (FVC) at Week 52 in Systemic Sclerosis subjects [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
    Change from Baseline (Week 0) of the forced vital capacity (FVC) at Week 52 in Systemic Sclerosis subjects

  • Change from Baseline (Week 0) of the modified Rodnan Skin Score (mRSS)at Week 52 in Systemic Sclerosis subjects [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) of the modified Rodnan Skin Score (mRSS)at Week 52 in Systemic Sclerosis subjects

  • Change from Baseline of the UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) total score in Systemic Sclerosis subjects [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) of the UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) total score in Systemic Sclerosis subjects


Secondary Outcome Measures:
  • Estimation of pomalidomide pharmacokinetic (PK) parameters in plasma, AUCt in Systemic Sclerosis subjects [ Time Frame: Up to 8 weeks ] [ Designated as safety issue: No ]
    Estimation of pomalidomide pharmacokinetic (PK) parameters in plasma, AUCt in Systemic Sclerosis subjects

  • Estimation of pomalidomide PK parameters in plasma, Cmax in Systemic Sclerosis subjects [ Time Frame: Up to 8 weeks ] [ Designated as safety issue: No ]
    Estimation of pomalidomide PK parameters in plasma, Cmax in Systemic Sclerosis subjects

  • Estimation of pomalidomide PK parameters in plasma, Tmax in Systemic Sclerosis subjects [ Time Frame: Up to 8 weeks ] [ Designated as safety issue: No ]
    Estimation of pomalidomide PK parameters in plasma, Tmax in Systemic Sclerosis subjects

  • Change from Baseline (Week 0) of the FVC in Systemic Sclerosis subjects [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) of the FVC in Systemic Sclerosis subjects

  • Change from Baseline (Week 0) of the Modified Rodnan Skin Score in Systemic Sclerosis subjects [ Time Frame: Up to 24 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) of the Modified Rodnan Skin Score in Systemic Sclerosis subjects

  • Change from Baseline (Week 0) of the UCLA SCTC GIT 2.0 total score in Systemic Sclerosis subjects [ Time Frame: Up to 24 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) of the UCLA SCTC GIT 2.0 total score in Systemic Sclerosis subjects

  • Change from Baseline (Week 0) UCLA SCTC GIT 2.0 subscale scores (Reflux, Distention/Bloating, Fecal Soilage, Diarrhea, Social functioning, Emotional Well-being and Constipation) in Systemic Sclerosis subjects [ Time Frame: Up to 52 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) UCLA SCTC GIT 2.0 subscale scores (Reflux, Distention/Bloating, Fecal Soilage, Diarrhea, Social functioning, Emotional Well-being and Constipation) in Systemic Sclerosis subjects

  • Change from Baseline (Week 0) of the oxygen saturation (as measured by pulse oximetry) in Systemic Sclerosis subjects [ Time Frame: Up to 52 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) of the oxygen saturation (as measured by pulse oximetry) in Systemic Sclerosis subjects

  • Change from Baseline (Week 0) dyspnea (as measured by the Transition Dyspnea Index) in Systemic Sclerosis subjects [ Time Frame: Up to 52 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) dyspnea (as measured by the Transition Dyspnea Index) in Systemic Sclerosis subjects


Estimated Enrollment: 88
Study Start Date: July 2012
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Pomalidomide (1 mg once daily) Drug: Pomalidomide (CC-4047)
1 mg orally every day for 52 weeks
Placebo Comparator: Placebo Drug: Placebo
Placebo

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or females between 18 and 80 years of age (inclusive) at the time of consent
  • Diagnosis of SSC as defined by 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:
  • Repeat 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 computed tomography consistent with sclerodermatous involvement of the lung (eg, ground glass, honeycombing)

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. An HRCT fibrosis score > 20%

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 (e.g., systemic lupus erythematosus, rheumatoid arthritis, Sjogren's syndrome, etc.)
  • Use of melphalan within 52 weeks of Screening
  • 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 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 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01559129

Contacts
Contact: Lisa Serme 732-652-6307 lserme@celgene.com
Contact: Sigrid Böhme +41(0)32 729 8837 sboehme@celgene.com

  Show 66 Study Locations
Sponsors and Collaborators
Celgene Corporation
Investigators
Study Director: William Smith, MD Celgene Corporation
  More Information

No publications provided

Responsible Party: Celgene Corporation
ClinicalTrials.gov Identifier: NCT01559129     History of Changes
Other Study ID Numbers: CC-4047-SSC-001, 2010-023047-15
Study First Received: March 19, 2012
Last Updated: March 28, 2014
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
Australia: Human Research Ethics Committee
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
France: Committee for the Protection of Personnes
France: Conseil National de l'Ordre des Médecins
Germany: Federal Institute for Drugs and Medical Devices
Germany: Ethics Commission
Italy: Ethics Committee
Italy: National Monitoring Centre for Clinical Trials - Ministry of Health
Poland: Ethics Committee
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Poland: The Central Register of Clinical Trials
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Spain: Comité Ético de Investigación Clínica
Switzerland: Ethikkommission
Switzerland: Swissmedic
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: National Health Service
United Kingdom: National Institute for Health Research
United Kingdom: Research Ethics Committee
United States: Institutional Review Board
Russia: Ministry of Health of the Russian Federation

Additional relevant MeSH terms:
Scleroderma, Systemic
Scleroderma, Diffuse
Scleroderma, Localized
Lung Diseases
Sclerosis
Lung Diseases, Interstitial
Connective Tissue Diseases
Skin Diseases
Respiratory Tract Diseases
Pathologic Processes
Thalidomide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents

ClinicalTrials.gov processed this record on April 14, 2014