A population based cohort study to monitor the safety and effectiveness of sirolimus in patients with sporadic lymphangioleiomyomatosis (S-LAM)

Study identification

• EU PAS number:
  EUPAS30455
• Study ID:
  47297
• Official title and acronym:
  A population based cohort study to monitor the safety and effectiveness of sirolimus in patients with sporadic lymphangioleiomyomatosis (S-LAM)
• DARWIN EU® study:
  No
• Study countries:
  United Kingdom

  United States
• Study description:
  This will be a descriptive cohort study using existing LAM data sources in the UK and the USA to evaluate S-LAM patients treated with sirolimus for long-term safety and effectiveness endpoints. Data within each of the UK and USA data sources will be used to estimate the incidence proportion of adverse events among S-LAM patients treated with sirolimus and to evaluate the selected effectiveness endpoints among S-LAM patients treated with sirolimus. In both countries, this descriptive study will include data collected in the respective data sources until 02 August 2020 which would be two years post EU S-LAM approval date of 02 August 2018. Therefore, the study observation period in the UK is from 2011 to 02 August 2020 and in the USA the study observation period is from 2015 to 02 August 2020.
• Study status:
  Finalised

Research institutions and networks

Institutions

Pfizer

Study timelines

Date when funding contract was signed

Actual: 21/06/2019

Study start date

Planned: 03/08/2019

Actual: 03/08/2019

Date of interim report, if expected

Planned: 03/02/2020

Actual: 03/02/2020

Date of final study report

Planned: 03/02/2022

Actual: 31/01/2022

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Pfizer

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)

Other study registration identification numbers and links

B1741224