Phase III long-term study to evaluate the efficacy and safety of ulipristal acetate in Japanese patients with uterine fibroids.

AIM: To assess the efficacy and safety of long-term intermittent administration of 10-mg ulipristal acetate (UPA) for symptomatic uterine fibroids in Japanese women. METHODS: Open-label, noncomparative study (Japan Primary Registries Network identifier: JapicCTI-173737) conducted at 32 gynecological centers (November 2017-December 2019). Premenopausal women diagnosed with uterine fibroids associated with heavy menstrual bleeding received three 12-week courses of 10-mg UPA once daily. Amenorrhea, fibroid volume, endometrial histology, and safety were assessed. RESULTS: Of 155 patients enrolled, 140 received ≥1 dose of UPA and were analyzed. Across all courses, the rates of patients with amenorrhea for 35 days were >90%, and >99% of patients achieved uterine bleeding normalization. Median time to amenorrhea after each course started was 4-5 days; menstruation returned after treatment within a median of 25-27 days. Mean changes in fibroid volume from baseline were -21.5%, -31.4%, and -35.0% for Courses 1, 2, and 3, respectively. Patients experienced sustained improvements in anemia, pain, and quality of life during treatment. Most adverse events were mild/moderate in severity and decreased in frequency with each course. Seven serious adverse events (six patients) were reported; anemia, embolic cerebral infarction, and pituitary apoplexy (one patient each) were considered UPA-related. Nonphysiological changes in endometrial histology were transient and benign. No safety concerns were detected in hormone concentrations or liver function tests. CONCLUSIONS: Long-term administration of 10-mg UPA is effective for reducing symptoms associated with uterine fibroids in Japanese women. UPA was well tolerated and few safety concerns were reported.

Ulipristal acetate compared with leuprorelin acetate for Japanese women with symptomatic uterine fibroids: a phase III randomized controlled trial.

OBJECTIVE: To evaluate the efficacy and safety of ulipristal acetate (UPA) for uterine fibroids (UFs), a phase III study was conducted with leuprorelin (LEU) as a comparator. This is the first confirmatory trial of UPA for UFs among Asians. DESIGN: Multicenter, randomized, double-blind, double-dummy, parallel-group study. SETTING: Thirty-two sites in Japan. PATIENT(S): Patients were assigned to 2 arms, with 82 patients in the UPA group and 79 patients in the LEU group. INTERVENTION(S): In the UPA group, 10 mg of UPA was orally administered once a day for 12 weeks. In the LEU group, 1.88 mg or 3.75 mg of LEU was subcutaneously administered at weeks 0, 4, and 8. MAIN OUTCOME MEASURE(S): The primary endpoint was the percentage of patients with amenorrhea for 35 days. For safety evaluation, adverse events (AEs) were recorded. RESULT(S): The percentage of patients with amenorrhea for 35 days was 87.0% in the UPA group and 81.8% in the LEU group, and the efficacy of UPA for causing amenorrhea for 35 days was confirmed to be noninferior to that of LEU. AEs occurred in 78.0% of the patients in the UPA group and 88.8% of the patients in the LEU group. CONCLUSION(S): The effect of UPA on heavy menstrual bleeding was shown to be comparable with that of LEU in Japanese patients with symptomatic UFs. No notable AEs occurred because of the UPA treatment, and the incidence of AEs in the UPA group was comparable with that of AEs in the LEU group. This result demonstrates the clinical utility of UPA for Asians.