Vilaprisan in women with uterine fibroids: the randomized phase 2b ASTEROID 1 study.

Abstract

OBJECTIVES:

To assess the safety and efficacy of four vilaprisan doses (0.5-4.0 mg) in women with uterine fibroids.

DESIGN:

Randomized, double-blind, placebo-controlled, multicenter trial.

SETTING:

Ninety-eight centers in 12 countries.

PATIENT(S):

Women aged 18-50 years with uterine fibroids and heavy menstrual bleeding were randomized equally to oral vilaprisan at 0.5, 1.0, 2.0, or 4.0 mg or placebo once daily.

INTERVENTION(S):

Treatment for 12 weeks, 24-week follow-up.

MAIN OUTCOME MEASURE(S):

Primary end point was absence of scheduled or unscheduled bleeding/spotting. Key secondary efficacy end points included volume of menstrual blood loss and change in fibroid volume.

RESULT(S):

A total of 309 patients were randomized, and 300 received treatment. Complete absence of bleeding/spotting was observed in 30%, 56%, 54%, and 60% of patients in the vilaprisan 0.5, 1.0, 2.0, and 4.0 mg groups, respectively, versus 1.7% with placebo. After 12 weeks, >83% of women achieved amenorrhea (<2 mL/28 days) with ≥1.0 mg vilaprisan versus 9% with placebo. Heavy menstrual bleeding stopped (but returned at a lower volume after treatment cessation) with ≥1.0 mg vilaprisan treatment. Reductions in fibroid volume of up to 41% were observed. Most patients receiving vilaprisan reported improvements in symptom severity. No safety concerns were identified in general safety, endometrial safety (by biopsy), laboratory values, and ultrasound examinations.

CONCLUSION(S):

ASTEROID 1 supports the efficacy of vilaprisan for the treatment of heavy menstrual bleeding associated with uterine fibroids. Daily oral treatment with vilaprisan 0.5-4.0 mg was well tolerated, and vilaprisan 2.0 mg once daily has been selected for further investigation.

CLINICAL TRIAL REGISTRATION NUMBER:

NCT02131662.

• Authors: Linda D Bradley, Sukhbir S Singh, James Simon, Kristina Gemzell-Danielsson, Kathrin Petersdorf, Esther Groettrup-Wolfers, Xiaowei Ren, Michal Zvolanek, Christian Seitz
• Journal: Fertility and sterility (Fertil Steril) Vol. 111 Issue 2 Pg. 240-248 (02 2019) ISSN: 1556-5653 [Electronic] United States
• PMID: 30527839 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2018 The Authors. Published by Elsevier Inc. All rights reserved.

Chemical References

• Steroids
• vilaprisan

Topics

• Administration, Oral
• Adult
• Double-Blind Method
• Drug Administration Schedule
• Europe
• Female
• Humans
• Japan
• Leiomyoma (complications, diagnostic imaging, drug therapy, physiopathology)
• Menorrhagia (diagnosis, etiology, physiopathology, prevention & control)
• Menstruation (drug effects)
• Middle Aged
• North America
• Steroids (administration & dosage, adverse effects)
• Time Factors
• Treatment Outcome
• Tumor Burden
• Uterine Neoplasms (complications, diagnostic imaging, drug therapy, physiopathology)