Table 3.

Summary of key findings of phase I and II clinical trials utilising vilaprisan.

Phase I clinical trials (Schütt et al. 2016, 2018)

Vilaprisan 0.5–5 mg/day for 12 weeks

1. Maximal non-bleeding rates achieved at dose of 2 mg or higher per day.

2. Doses >0.5 mg/day are associated with a decrease in FSH and LH.

3. Follicular growth mid-follicular oestradiol levels maintained.

4. Ovulation inhibited on >80% of participants at doses ≥1 mg/day.

5. Return of menstruation in ≤52 days after discontinuation.

6. No serious adverse events. A non-dose dependent transient rise in liver transaminases seen during treatment with vilaprisan which returned to baseline.

7. PAEC present in 10% of women pre-treatment and with 100% frequency in women on 5 mg/day (dose dependant). At doses of 1 mg/day, PAEC was observed in 70–90% of women. Regression was noted in majority of women at first bleed post treatment with regression in all participants at 4–6 months.

Phase II clinical trial (Bradley et al. 2016)

Vilaprisan 0.5–4 mg/day for 12 weeks

1. Amenorrhea (MBL <2 ml/28 days by alkali hematin) seen in 87–92% of participants at doses of 1 mg/day or higher.

2. Median time of amenorrhoea – 3 days.

3. Dose dependant reduction in fibroid size; up to 40% at 4 mg dose.

4. Improvements in HRQoL.

5. No serious adverse events.

6. PAEC seen in up to 40% of women on completing treatment with vilaprisan. Complete regression of PAEC to baseline levels was observed during the follow up period (24 weeks).