CDB-2914 for uterine leiomyomata treatment: a randomized controlled trial.
OBJECTIVE
To evaluate whether 3-month administration of CDB-2914, a selective progesterone receptor modulator, reduces leiomyoma size and symptoms.


METHODS
Premenopausal women with symptomatic uterine leiomyomata were randomly assigned to CDB-2914 at 10 mg (T1) or 20 mg (T2) daily or to placebo (PLC) for 3 cycles or 90-102 days if no menses occurred. The primary outcome was leiomyoma volume change determined by magnetic resonance imaging at study entry and within 2 weeks of hysterectomy. Secondary outcomes included the proportion of amenorrhea, change in hemoglobin and hematocrit, ovulation inhibition, and quality-of-life assessment.


RESULTS
Twenty-two patients were allocated, and 18 completed the trial. Age and body mass index were similar among groups. Leiomyoma volume was significantly reduced with CDB-2914 administration (PLC 6%; CDB-2914 -29%; P=.01), decreasing 36% and 21% in the T1 and T2 groups, respectively. During treatment, hemoglobin was unchanged, and the median estradiol was greater than 50 pg/mL in all groups. CDB-2914 eliminated menstrual bleeding and inhibited ovulation (% ovulatory cycles: CDB-2914, 20%; PLC, 83%; P=.001). CDB-2914 improved the concern scores of the uterine leiomyoma symptom quality-of-life subscale (P=.04). One CDB-2914 woman developed endometrial cystic hyperplasia without evidence of atypia. No serious adverse events were reported.


CONCLUSIONS
Compared with PLC, CDB-2914 significantly reduced leiomyoma volume after three cycles, or 90-102 days. CDB-2914 treatment resulted in improvements in the concern subscale of the Uterine Fibroid Symptom Quality of Life assessment. In this small study, CDB-2914 was well-tolerated without serious adverse events. Thus, there may be a role for CDB-2914 in the treatment of leiomyomata.


BACKGROUND
ClinicalTrials.gov,www.clinicaltrials.gov, NCT00290251


METHODS
I.
Levens
Eric D
ED
Reproductive Biology and Medicine Branch, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland 20892, USA.
Potlog-Nahari
Clariss
C
Armstrong
Alicia Y
AY
Wesley
Robert
R
Premkumar
Ahalya
A
Blithe
Diana L
DL
Blocker
Wendy
W
Nieman
Lynnette K
LK
D000328:Adult; D005260:Female; D006305:Health Status Indicators; D006400:Hematocrit; D006801:Humans; D007889:Leiomyoma / Q000188:drug therapy*; D008279:Magnetic Resonance Imaging; D008875:Middle Aged; D009649:Norpregnadienes / Q000008:administration & dosage / Q000627:therapeutic use*; D011788:Quality of Life; D016896:Treatment Outcome; D014594:Uterine Neoplasms / Q000188:drug therapy*
D009649:Norpregnadienes; C094854:ulipristal