Efficacy, safety and tolerability of the CCR1 antagonist BAY 86-5047 for the treatment of endometriosis-associated pelvic pain: a randomized controlled trial.
BACKGROUND
Antagonism of CC chemokine receptor type 1 (CCR1) may provide a novel treatment approach for women with symptomatic endometriosis. Studies of CCR1 antagonists in these patients have not been reported.


METHODS
Women (n = 110; 18-45 years) with symptomatic endometriosis were randomized to BAY 86-5047 or placebo for 12 weeks. Pelvic pain was assessed using the visual analogue scale (VAS) and women recorded the intake of pain medication in a diary. The primary efficacy outcome was a composite of the absolute change in VAS score and the cumulative change in consumption of analgesics between baseline and the end of treatment. Safety assessments included adverse events, blood and urine evaluation and electrocardiography.


RESULTS
Mean VAS scores decreased from 64.8 mm at baseline to 49.2 mm at week 12 in the BAY 86-5047 group and from 67.2 mm to 47.8 mm in the placebo group. The proportion of women using analgesics decreased from 33.9% to 11.5% or from 44.4% to 15.4% for patients who received BAY 86-5047 or placebo, respectively. There was no significant difference between the two treatment groups in terms of change in VAS scores (p = 0.45) or intake of analgesics (p = 0.82). A three-step sensitivity analysis failed to show superiority of BAY 86-5047 over placebo (p = 0.67). BAY 86-5047 was well tolerated and no significant safety concerns arose during the study.


CONCLUSIONS
Based on these results, BAY 86-5047 is unlikely to be useful in the treatment of women with endometriosis-associated pelvic pain.
R&D Statistics, Bayer Pharma AG, Global Development, Berlin, Germany.
Dietmar
http://orcid.org/0000-0001-9643-0109
D
Trummer
Global Clinical Development, Bayer Pharma AG, Global Development, Berlin, Germany.
Anja
A
Walzer
Global Pharmacovigilance, Bayer Pharma AG, Global Development, Berlin, Germany.
Esther
E
Groettrup-Wolfers
Global Clinical Development, Bayer Pharma AG, Global Development, Berlin, Germany.
Heinz
H
Schmitz
D000328:Adult; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D004715:Endometriosis / Q000150:complications* / Q000188:drug therapy; D005260:Female; D006801:Humans; D010147:Pain Measurement; D017699:Pelvic Pain / Q000188:drug therapy* / Q000209:etiology; D054389:Receptors, CCR1 / Q000008:administration & dosage / Q000037:antagonists & inhibitors*; D016896:Treatment Outcome; D055815:Young Adult
C515300:CCR1 protein, human; D054389:Receptors, CCR1
BAY 86-5047; CCR1 antagonist; Endometriosis; Placebo; pelvic pain