A study to see if pomalidomide is safe and works to treat patients with sclerosis affecting the skin on the whole body and that also have lung disease

Phase 1

• Conditions: Diffuse cutaneous systemic sclerosis with interstitial lung disease; MedDRA version: 14.1Level: LLTClassification code 10025109Term: Lung involvement in systemic sclerosisSystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders; MedDRA version: 14.1Level: SOCClassification code 10038738Term: Respiratory, thoracic and mediastinal disordersSystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2010-023047-15-IT
• Lead Sponsor: CELGENE CORPORATIO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-12-28
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 88

Inclusion Criteria

1.Male or female subjects between 18 and 80 years of age (inclusive) at the time of signing the ICD 2.Body weight = 80 pounds (36.3 kilograms) at Screening and Baseline 3.Understand and voluntarily sign ICDs prior to the initiation of any study-related assessments / procedures 4.Able to adhere to the study visit schedule and other protocol requirements5.Diagnosis of dc-SSc as defined by ACR criteria 6.Onset of the first non-Raynaud’s manifestation of SSc within 5 years of Screening 7.FVC = 45% and=80% predicted at Screening and Baseline. Source documentation confirming a = 5% decrease in FVC based on 3 or more assessments within 18 months of Baseline is required.Two assessments may be done during the Screening Phase provided the assessments are completed at least 2 weeks apart.8.Repeat FVC at Baseline within 5% of the FVC measured at Screening (most recent assessment if multiple assessments are performed during Screening)9.DLco = 40% and=80% of predicted value at Screening 10.Abnormalities on HRCT consistent with sclerodermatous involvement of the lung 11•Hemoglobin =10.0g/dL •White blood cell count = 3000/µL(= 3.0x 109/L)e=14.000/µL(14x 109/L •Absolute neutrophil count = 2x 109/L •Platelet count = 150,000 /µL (= 150 X 109/L)•Serum creatinine = 1.5 mg/dL (= 132.6 µmol/L)•Total bilirubin = 2.0 mg/dL•Albumin = 3.0 g/dL•Aspartate transaminase AST and alanine transaminase ALT = 1.5 X upper limit of normal (ULN.)12.Females of childbearing potential (FCBP)must undergo pregnancy testing based on the frequency outlined in the protocol and pregnancy results must be negative.13.Unless practicing complete abstinence from heterosexual intercourse,sexually active FCBP must agree to use adequate contraceptive methods as specified in the protocol. 14.Males (including those who have had a vasectomy)must use barrier contraception (condoms) when engaging in sexual activity with FCBP as specified in the protocol.15.Males must agree not to donate semen or sperm during the duration specified in the protocol.16.All subjects must: •Understand that the IP could have a potential teratogenic risk.•Agree to abstain from donating blood while taking IP and following discontinuation of investigational product.•Agree not to share IP with another person.•Other than the subject, FCBP and males able to father a child should not handle the IP or touch the capsules, unless gloves are worn.•Be counseled about pregnancy precautions and risks of fetal exposure.17.Use of concomitant medications for the treatment of SSc-related symptoms is permitted.These include:•Proton pump inhibitors or other gastroesophageal reflux disease (GERD)therapies •Angiotensin receptor blocker (ARB)therapies •Angiotensin-converting enzyme inhibitor (ACEI) therapies•Selective serum reuptake inhibitors (SSRIs)•Calcium channel blockers •Non-sedating oral or intranasal antihistamine agents (intermittent usage)•PDE5 inhibitors (if not being taken for pulmonary arterial hypertension [PAH])•= 10 mg/day of oral prednisone (or equivalent)•Oral statin agents for hyperlipidemia (if the medication has been taken for=84 days [12 weeks] prior to Screening) •Analgesic or opioid pain medications •Subjects are required to follow a low-dose aspirin regimen (=100 mg per day) unless contraindicated.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of sub

Exclusion Criteria

1.Oxygen saturation (SpO2)< 92% at Screening or Baseline 2.Known diagnosis of obstructive lung disease as defined by FEV1/FVC ratio <0.65 3.Diagnosis of pulmonary arterial hypertension (PAH) requiring treatment 4.Known diagnosis of other significant respiratory disorders.5. Any significant medical condition, laboratory abnormality or psychiatric illness that would prevent the subject from participating in the study 6.Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study 7.Any condition that confounds the ability to interpret data from the study 8.Pregnant or lactating females 9.Current clinical diagnosis of another inflammatory connective tissue disease 10.History of a thromboembolic event or hypercoagulable state 11.Family history of genetic disease associated with deep vein thrombosis or thromboembolism 12.History of clinically significant endocrinologic, pulmonary (other than SSc-related), GI (other than SSc-related), neurologic, psychiatric, hepatic, renal, hematologic, cardiovascular, immunologic or other major uncontrolled disease 13. History or current diagnosis of peripheral neuropathy 14.History of alcohol or drug abuse 15.History of any of the following cardiac conditions within 6 months of Screening: acute myocardial infarction, acute coronary syndrome, new onset atrial fibrillation, new onset atrial flutter, second- or third-degree atrioventricular block, ventricular fibrillation, ventricular tachycardia, heart failure, cardiac surgery, interventional cardiac catheterization (with or without a stent placement), interventional electrophysiology procedure, presence of implanted permanent pacemaker or presence of implanted defibrillator 16.History of additional risk factors for torsade de pointes (TdP) (eg, heart failure, hypokalemia, family history of Long QT syndrome) 17.Corrected QTcF > 440 msec at Screening or Baseline (pre-dose)18.Presence of any of the following on 2 of the 3 Screening or Baseline ECGs (pre-dose) at rest: •heart rate < 45 beats per minute•heart rate > 110 beats per minute•PR interval > 220 ms •QRS duration > 110 ms 19.Clinically significant abnormality on any Screening or Baseline ECG 20.History of tuberculosis (TB) (Subjects with a history of TB who have completed a standard course of treatment [documented] are eligible for study entry).21.History of human immunodeficiency virus (HIV) infection 22.History of congenital and acquired immunodeficiencies 23.Hepatitis B surface antigen positive or hepatitis B core antibody positive at screening 24Antibodies to hepatitis C at scerrening 25History of malignancy 26.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 27.Use of melphalan within 52 weeks of Screening 28.Use of concomitant medications which prolong the QT/QTc interval 29.Use of any anti-coagulant or anti-thrombotic medications 30.Use of any cytotoxic/immunosuppressive agent within 84 days of Screening 31.Use of prostaglandin analogues within 28 days of Screening 32.Use of any biologic agent within 84 days or 5 half-lives of Screening.33.Use of endothelin-1 inhibitors within 84 days of Screening 34.Use of medications with putative scleroderma disease-modifying properties within 28 days of Screening 35.Smoking of cigars, pipes or cig

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified