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

• Registration Number: EUCTR2010-023047-15-ES
• Lead Sponsor: Celgene Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-01-19
• Last Update Posted: 23 January 2017

Recruitment & Eligibility

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

Inclusion Criteria

1.Male or female subjects between 18 and 80 (inclusive) at the time of signing the ICD
2.Body weight >=80pounds (36.3kg) 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 requirements
5.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 18months of Baseline (Visit 2) 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 (Visit 2) within 5% of the FVC measured at Screening
9.DLco >=40% and <=80% of predicted value at Screening
10.Abnormalities on HRCT consistent with sclerodermatous involvement of the lung
11.Meet the following laboratory criteria:
Hemoglobin >=10.0 g/dL
White blood cell (WBC) count >=3000/µL(3.0x10^9/L) and <=14,000/µL
(14x109/µL)
Absolute neutrophil count (ANC)>=2x10^9/L
Platelet count>=150,000/µL(>=150x10^9/L)
Serum creatinine<=1.5 mg/dL(<=132.6?mol/L)
Total bilirubin<=2.0 mg/dL
Albumin>=3.0g/dL
AST and ALT<=1.5X upper limit of normal
12.Females of childbearing potential (FCBP) must undergo pregnancy testing based and results must be negative
13.Unless practicing complete abstinence from heterosexual intercourse, sexually active FCBP must use adequate contraceptive methods
14.Males (including those who have had a vasectomy) must use barrier contraception when engaging in sexual activity with FCBP
15.Males must agree not to donate semen or sperm
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 IP
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 (Proton pump inhibitors or other gastroesophageal reflux disease therapies, Angiotensin receptor blocker, Angiotensin-converting enzyme inhibitor, Selective serum reuptake inhibitors, Calcium channel blockers, Non-sedating oral or intranasal antihistamine agents, PDE5 inhibitors, <=10mg/day of oral prednisone, Oral statin agents for hyperlipidemia, Analgesic or opioid pain medications, Subjects are required to follow a low-dose aspirin regimen (<=100mg/day) unless contraindicated
Are the trial subjects under 18? no
Number of subjects for this age range:
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 subjects for this age range 18

Exclusion Criteria

1.Oxygen saturation<92%(room air [sea level] at rest) 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
17.Corrected QTcF>440msec at Screening or Baseline
18. Presence of any of the following on 2 of the 3 Screening or Baseline ECGs (pre-dose) at rest: heart rate<45beats/min or >110beats/min, PR interval>220ms, QRS duration>110ms
19.Clinically significant abnormality on any Screening or Baseline ECG
20.History of tuberculosis
21.History of HIV infection
22.History of congenital and acquired immunodeficiencies
23.Hepatitis B surface antigen positive or hepatitis B core antibody positive at Screening
24.Antibodies to hepatitis C at Screening
25.History of malignancy
26.Use of concomitant medication(s) which could increase the risk for developing deep vein thrombosis
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
31.Use of prostaglandin analogues
32.Use of any biologic agent within 84 days or 5 half-lives of Screening
33.Use of endothelin-1 inhibitors
34.Use of medications with putative scleroderma disease-modifying properties within 28 days of Screening
35.Smoking within 168 days of Screening
36.Ingestion of grapefruit, grapefruit juice or grapefruit-containing products within 14 days of randomization
37.Use of any investigational drug within 28 days of Screening or 5 PD/PK
half-lives

Study & Design

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