CC-4047-MM-015-Pomalidomide for relapsed and refractory MM patients_Ob
Research type
Research Study
Full title
A non-interventional post authorisation registry of patients treated with pomalidomide for relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy.
IRAS ID
166544
Contact name
Graham Jackson
Contact email
Sponsor organisation
Celgene Europe Limited
Clinicaltrials.gov Identifier
EU/1/13/850/001-004, Product reference
Duration of Study in the UK
5 years, 2 months, 1 days
Research summary
Research Summary -
This study is a non-interventional, post-authorisation registry of patients being treated with pomalidomide for relapsed and refractory multiple myeloma (RRMM) to monitoring the incidence of adverse drug reactions (ADRs) in a real world setting.
It is not the purpose of this study to test a new experimental product.
This registry will collect post approval data on important identified and potential risks associated with pomalidomide administration, such as: teratogenicity, neutropenia, thromboembolic events, peripheral neuropathy, infection, thrombocytopenia and bleeding, tumour lysis syndrome, somnolence, second primary malignancies, thyroid disorders, renal failure, QT interactions (prolongations), severe skin reactions, cardiac failure, and cardiac arrhythmia.
Lay Summary of results -
The study database was initially locked on 22 December 2022. An inspection conducted by the Medicines & Healthcare product Regulatory Agency (MHRA) at an investigator site in 2022 identified data quality issues specific to the primary objective of the study. To assess the extent and impact of the data quality issues, BMS developed a formal, comprehensive plan to evaluate the overall quality of the study data and its potential impact on the study’s safety-specific outcomes. This risk-based approach included reopening the database, source data verification, as-needed data correction, statistical re-analysis, as indicated by the findings, and impact assessment analysis. The database was re-locked on 12 September 2024. To assess the extent and impact of the data quality issues, the Sponsor performed on-site quality review visits and targeted source data reviews (SDRs), including the site where the MHRA first identified an issue for which the data of 10 patients not assessed during the inspection were scrutinized. Additionally, the same activities were conducted at 15 other sites randomly selected. The targeted SDRs covered approximately 16% (122/761) of the safety population. Review of approximately 16% of all subjects in this PASS revealed previously unreported AEs across all sites and countries, without a particular pattern to recognize specific sites or countries at greater risk of safety underreporting. A detailed analysis of the commonly unreported AEs showed the largest discrepancy in the “infections and infestations” SOC. While this analysis was not designed to understand the causes of underreporting, it should be noted that infections are a known comorbid condition commonly associated with MM, as well as a known risk associated with pomalidomide use. Thus, it is plausible, despite investigator training and instruction, that some investigators considered these events to be part of the natural history and expected course of events for their MM patients. Importantly, even when this underreporting is extrapolated to the entire study population, it did not alter the established safety or benefit-risk profile of pomalidomide. Overall, the safety findings in patients treated with pomalidomide for RRMM in a real-world clinical setting did not identify any new safety concerns and reflected already demonstrated risks recognized through the clinical development of the drug for this indication.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
14/SC/1350
Date of REC Opinion
11 Nov 2014
REC opinion
Further Information Favourable Opinion