PEARL IV (PGL4001 Efficacy Assessment in Reduction of symptoms due to

• Research type

  Research Study

• Full title

  A Phase III, multicentre, randomized, double-blind clinical study, investigating the efficacy and safety of repeated 12-week courses of daily 5mg or 10mg doses of PGL4001 for the long-term management of symptomatic uterine fibroids

• IRAS ID

  100337

• Contact name

  Proefessor Hilary OD Critchley

• Sponsor organisation

  PregLem S.A.

• Eudract number

  2012-000036-26

• Research summary

  This is a Phase III research study to assess the effectiveness and safety of long term repeated intermittent treatment with the investigational drug PGL4001. This study will examine the impact of 5 or 10mg doses on uterine bleeding, myoma volume, uterus volume, quality of life and pain in women with uterine fibroids. PGL4001 5mg is approved by Regulatory Authorities in Europe under the brand name Esmya©, however, the treatment duration is currently limited to 3 months. This study will provide data on longterm use. A total of up to 500 premenopausal women between 18 and 50 years inclusive will take part in this study in approximately 90 centers. To investigate the best ways of treating patients, there is a need to compare two doses. Each participant will be randomly assigned to one of 2 treatment groups (at a ratio of 1:1) and the results of the groups will be compared after the study. This randomisation will be double blind (neither the research team nor patient knows which treatment is being given). The two study groups are as follows: Group 1: Participants will receive PGL4001 5 mg active drug and placebo of 10 mg once a day Group 2: Participants will receive PGL4001 10 mg active drug and placebo of 5 mg once a day The entire study will last approximately 20 months and will be divided into Screening Periods, a Treatment Period and a Post-Treatment Period. During the study each participant will be asked to come to the clinic a total of 14 times for the following assessments and procedures: physical examinations, blood & urine tests, ultrasound, endometrium biopsies, PAP smears, ECGs and questionnaires. It is hoped the information gained from the study will help in the development of better treatment for women with uterine fibroids in the future.

• REC name

  Scotland A REC

• REC reference

  12/SS/0068

• Date of REC Opinion

  17 May 2012

• REC opinion

  Further Information Favourable Opinion