TerminatedPhase 2

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

Stopped: Enrollment was stopped early (see limitations and caveats section).

United States, Australia, France23 participantsStarted 2012-08-09

Plain-language 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.

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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:. 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 * Oxygen saturation (SpO2) \< 92% (room air \[sea level\] at rest) at Screening or Baseline. * Known diagnosis …

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Timeframe: From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide.

Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52

Timeframe: Baseline (defined as the average of all values between Screening and Baseline) and Weeks 48 and 52

Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52/Early Termination

Timeframe: Baseline and Week 52 (or the Treatment Phase Early Termination visit)

Change From Baseline in University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) Total Score at Week 52/Early Termination

Timeframe: Baseline and Week 52 (or Treatment Phase Early Termination visit)

Trial details

NCT IDNCT01559129

SponsorCelgene

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2016-11-03

Results submitted2017-11-03

View on ClinicalTrials.gov More Scleroderma, Systemic trials