Efficacy and tolerability of CDB-2914 treatment for symptomatic uterine fibroids: a randomized, double-blind, placebo-controlled, phase IIb study

Fertil Steril. 2011 Feb;95(2):767-72.e1-2. doi: 10.1016/j.fertnstert.2010.09.059. Epub 2010 Nov 5.


Objective: To evaluate the efficacy and tolerability of the P receptor modulator CDB-2914 (Ulipristal, CDB).

Design: Randomized, placebo-controlled double-blind clinical trial.

Setting: Clinical research center.

Patient(s): Premenopausal women with symptomatic uterine fibroids.

Intervention(s): 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.

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

Result(s): 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.

Conclusion(s): Administration of CDB-2914 for 3-6 months controls bleeding, reduces fibroid size, and improves QOL.

Trial registration: ClinicalTrials.gov NCT00290251.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural

MeSH terms

  • Adult
  • Algorithms
  • Antineoplastic Agents, Hormonal / adverse effects
  • Antineoplastic Agents, Hormonal / therapeutic use
  • Double-Blind Method
  • Female
  • Hormone Antagonists / adverse effects
  • Hormone Antagonists / therapeutic use
  • Humans
  • Leiomyoma / drug therapy*
  • Middle Aged
  • Norpregnadienes / adverse effects*
  • Norpregnadienes / therapeutic use*
  • Placebos
  • Progesterone / antagonists & inhibitors
  • Treatment Outcome
  • Uterine Neoplasms / drug therapy*


  • Antineoplastic Agents, Hormonal
  • Hormone Antagonists
  • Norpregnadienes
  • Placebos
  • Progesterone
  • ulipristal

Associated data

  • ClinicalTrials.gov/NCT00290251