Efficacy and tolerability of CDB-2914 treatment for symptomatic uterine fibroids: a randomized, double-blind, placebo-controlled, phase IIb study.
OBJECTIVE
To evaluate the efficacy and tolerability of the P receptor modulator CDB-2914 (Ulipristal, CDB).


METHODS
Randomized, placebo-controlled double-blind clinical trial.


METHODS
Clinical research center.


METHODS
Premenopausal women with symptomatic uterine fibroids.


METHODS
Once-daily oral CDB (10 or 20 mg) or placebo (PLC) for 12 weeks (treatment 1). A second 3-month treatment with CDB (treatment 2) was offered. A computer-generated blocked randomization was used.


METHODS
Magnetic resonance imaging (MRI)-determined total fibroid volume (TFV) change was the primary outcome; amenorrhea and quality of life (QOL) were secondary end points.


RESULTS
Treatment 1 TFV increased 7% in the PLC group, but decreased 17% and 24% in the CDB10 and CDB20 groups. The TFV decreased further in treatment 2 (-11%). Amenorrhea occurred in 20/26 women taking CDB and none on PLC. Ovulation resumed after CDB. Hemoglobin improved only with CDB (11.9 ± 1.5 to 12.9 ± 1.0 g/dL) as did the Fibroid QOL Questionnaire symptom severity, energy/mood, and concern subscores, and overall QOL scores. The CDB was well tolerated, with no serious adverse events. Adverse events were unchanged during treatments.


CONCLUSIONS
Administration of CDB-2914 for 3-6 months controls bleeding, reduces fibroid size, and improves QOL.
Program in Reproductive and Adult Endocrinology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, Maryland, USA. NiemanL@nih.gov
Lynnette K
LK
Nieman
Wendy
W
Blocker
Tonja
T
Nansel
Sheila
S
Mahoney
James
J
Reynolds
Diana
D
Blithe
Robert
R
Wesley
Alicia
A
Armstrong
D000328:Adult; D000465:Algorithms; D018931:Antineoplastic Agents, Hormonal / Q000009:adverse effects / Q000627:therapeutic use; D004311:Double-Blind Method; D005260:Female; D006727:Hormone Antagonists / Q000009:adverse effects / Q000627:therapeutic use; D006801:Humans; D007889:Leiomyoma / Q000188:drug therapy*; D008875:Middle Aged; D009649:Norpregnadienes / Q000009:adverse effects* / Q000627:therapeutic use*; D010919:Placebos; D011374:Progesterone / Q000037:antagonists & inhibitors; D016896:Treatment Outcome; D014594:Uterine Neoplasms / Q000188:drug therapy*
D018931:Antineoplastic Agents, Hormonal; D006727:Hormone Antagonists; D009649:Norpregnadienes; D010919:Placebos; D011374:Progesterone; C094854:ulipristal