Vilaprisan for the treatment of symptomatic endometriosis: results from a terminated phase 2b randomized controlled trial.
F S Rep
; 5(2): 189-196, 2024 Jun.
Article
de En
| MEDLINE
| ID: mdl-38983729
ABSTRACT
Objective:
To evaluate the efficacy and safety of 2 doses of vilaprisan vs. placebo in participants with symptomatic endometriosis.Design:
Multicenter, randomized, double-blind, placebo-controlled, parallel-group phase 2b trial (NCT03573336). The initially planned sample size was 315 patients. Recruitment was paused to assess long-term toxicity findings in rodents; although the findings were assessed as likely to be of limited clinical relevance in humans, the study was closed by the sponsor. During the pause, enrolled patients completed 3 or 6 months of treatment per their assigned regimen.Setting:
University hospitals, a regional hospital, and a private clinic. Patients Premenopausal adults with confirmed endometriosis and moderate-to-severe pelvic pain (≥4/10 on a numerical rating scale) were enrolled. Inclusion required protocol adherence, including ≥24 diary entries, and an average pain score of ≥3.5. Intervention Participants were randomly assigned 111 to receive vilaprisan (2 mg), vilaprisan (4 mg), or placebo. Main OutcomeMeasures:
The primary outcome was a change in the 7-day mean "worst pain" (per the endometriosis symptom diary item 1) from baseline to month 3. All analyses were descriptive only.Results:
Eight participants were randomly assigned to treatment before the study pause 6 received vilaprisan (4 mg, n = 4 and 2 mg, n = 2), and 2 received placebo. The 6 vilaprisan recipients experienced an improvement in endometriosis-associated pelvic pain, whereas the 2 placebo recipients experienced no change or increased pain; all 8 participants had decreased use of pain medication. Bleeding intensity decreased from baseline in the vilaprisan group.Conclusion:
The study findings suggest that vilaprisan may improve outcomes in patients with endometriosis. Further studies in larger populations would be needed to accurately assess treatment effects. Clinical Trial Registration Number NCT03573336.
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Langue:
En
Journal:
F S Rep
Année:
2024
Type de document:
Article
Pays de publication:
États-Unis d'Amérique