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Vilaprisan

Phase 2

Leiomyoma | Small molecule | Other |Bayer AG|Last Updated: Dec 3, 2019

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials2
Total Enrollment156

FDA Designations

No designations recorded

Clinical trial landscape

Vilaprisan · 6 trials · 5 indications

Phase 2 1Phase 1 5
NCT02465814Assess Safety and Efficacy of Vilaprisan in Patients With Uterine FibroidsLeiomyoma
COMPLETED120 Analytics
PHASE2COMPLETED
Assess Safety and Efficacy of Vilaprisan in Patients With Uterine Fibroids
LeiomyomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Amenorrhea (yes/no)
From day 7 to day 84 of treatment.

Defined as no scheduled or unscheduled bleeding/spotting after end of the initial bleeding episode until the end of the respective treatment period.

Area under the concentration versus time curve from zero to infinity after single (first) dose (AUC) of BAY1002670
-1hour (h), 30minutes (min), 1h, 1.5h, 2h, 2.5h, 3h, 4h, 6h, 8h, 12h ,16h, 1day (d), 2d, 3d, 4d, 7d, 10d, 14d

Area under the concentration versus time curve from zero to the last data point above the lower limit of quantitation \[AUC(0-tlast)\], if AUC cannot be estimated in all subjects. In subjects with normal and moderately reduced renal function.

Maximum observed drug concentration in measured matrix after single dose administration (Cmax) of BAY1002670
-1h, 30min, 1h, 1.5h, 2h, 2.5h, 3h, 4h, 6h, 8h, 12h ,16h, 1d, 2d, 3d, 4d, 7d, 10d, 14d

In subjects with normal and moderately reduced renal function.

Number of subjects with ovulation (i.e. Hoogland score = 6) or risk of ovulation (Hoogland score = 5)
treatment day 57 to day 84
Number of subjects with a Hoogland score = 4
treatment day 57 to day 84
Area under the concentration vs. time curve in plasma from zero to infinity (AUCu) (unbound)
At pre-dose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16 hours and at 1, 2, 3, 7, 10, 13, 16, 20 days

Exposure of Vilaprisan in plasma following a single dose administration

Maximum observed (unbound) drug concentration (Cmax,u)
At pre-dose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16 hours and at 1, 2, 3, 7, 10, 13, 16, 20 days

Maximum observed (unbound) drug concentration (Cmax,u) in measured matrix after a single dose administration

Area under the concentration time curve [AUC(0-11d)] after single oral dose of vilaprisan with and without ITZ.
up to 14 days
Maximum plasma concentration (Cmax) after single oral dose of vilaprisan with and without ITZ.
up to 14 days
Hoogland score during treatment, day 9 to day 28
Day 9 to 28

based on maximum size of follicle-like structures, estradiol and progesterone serum concentrations

Hoogland score during treatment, day 63 to day 84
Day 63 to 84

based on maximum size of follicle-like structures, estradiol and progesterone serum concentrations

Hoogland score during follow up cycle 1
4 weeks following treatment period

based on maximum size of follicle-like structures, estradiol and progesterone serum concentrations

Hoogland score during follow up cycle 2
4 weeks following follow up cycle 1

based on maximum size of follicle-like structures, estradiol and progesterone serum concentrations

Number of subjects without bleeding/spotting
After three month treatment

Secondary Endpoints

Number of bleeding days
Up to 32 weeks
Time to onset of controlled bleeding
Up to 28 weeks
Percent change in volume of largest fibroid from baseline to end of treatment.
Baseline and up to 28 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Arm 1 - BAY1002670 + BAY1002670EXPERIMENTALVilaprisan (BAY1002670) 2 mg once daily (12 weeks), Vilaprisan 2 mg once daily (12 weeks)
Arm 2 - Placebo + BAY1002670EXPERIMENTALPlacebo once daily (12 weeks), Vilaprisan 2 mg once daily (12 weeks)
Arm 3 - BAY1002670 + BAY1002670EXPERIMENTALVilaprisan 2 mg once daily (12 weeks), treatment break, Vilaprisan 2 mg once daily (12 weeks)
Arm 4 - Placebo+BAY1002670EXPERIMENTALPlacebo once daily (12 weeks), treatment break, Vilaprisan 2 mg once daily(12 weeks)
Arm 5 - Ulipristal + UlipristalACTIVE_COMPARATORUlipristal 5 mg once daily (12 weeks), treatment break, Ulipristal 5 mg once daily (12 weeks)
Arm 6- Placebo + UlipristalACTIVE_COMPARATORPlacebo once daily (12 weeks), treatment break, Ulipristal 5 mg once daily (12 weeks)
Arm 7- Ulipristal + PlaceboACTIVE_COMPARATORUlipristal 5 mg once daily (12 weeks), treatment break, Placebo once daily (12 weeks)
Subjects with moderately decreased renal functionEXPERIMENTALSubjects with moderate renal impairment with an estimated glomerular filtration rate (eGFR) of 30 to 59 mL/min/1.73 m\*2 according to the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
Subjects with severely decreased renal functionEXPERIMENTALSubjects with severe renal impairment not on dialysis with an eGFR \<30 mL/min/1.73 m\*2 (CKD-EPI formula).
Control subjects with normal renal functionEXPERIMENTALSubjects with an eGFR ≥90 mL/min/1.73 m\*2 (CKD-EPI formula) who are matched based on sex, age, race and weight.
COC + VilaprisanEXPERIMENTALCOC + Vilaprisan (BAY 1002670)
COC + PlaceboPLACEBO_COMPARATORCOC + Placebo
Healthy subjectsEXPERIMENTALhealthy subjects
Subjects with mild hepatic impairmentEXPERIMENTALhepatically impaired patients (classified as Child Pugh A)
Subjects with moderate hepatic impairmentEXPERIMENTALhepatically impaired patients (classified as Child Pugh B)
Vilaprisan + ItraconazoleEXPERIMENTALVilaprisan (BAY1002670)
Vilaprisan [0.5mg]EXPERIMENTAL0.5 mg BAY1002670, oral administration, one tablet to be taken daily, 84 consecutive days
Vilaprisan [1mg]EXPERIMENTAL1 mg BAY1002670, oral administration, one tablet to be taken daily, 84 consecutive days
Vilaprisan [2mg]EXPERIMENTAL2 mg BAY1002670, oral administration, one tablet to be taken daily, 84 consecutive days
Vilaprisan [4mg]EXPERIMENTAL4 mg BAY1002670, oral administration, one tablet to be taken daily, 84 consecutive days

Interventions

NameTypeDescription
Vilaprisan (BAY1002670)DRUGVilaprisan 2 mg (12 weeks), Vilaprisan 2 mg (12 weeks)
Vilaprisan (BAY1002670DRUGPlacebo (12 weeks),Vilaprisan (12 weeks)
UlipristalDRUGUlipristal 5 mg (12 weeks), treatment break, Ulipristal 5 mg (12 weeks)
Vilaprisan (BAY 1002670)DRUGMultiple doses of Vilaprisan for 3 cycles of 28 days each and 7 additional doses on the 7 days after cycle 3.
Vilaprisan PlaceboDRUGMultiple doses of placebo for 3 cycles of 28 days each and 7 additional doses on the 7 days after cycle 3.
MicrogynonDRUGMultiple doses of Microgynon for 3 cycles of 21 days each (day 1-21)
Microgynon PlaceboDRUGFor the treatment cycle 1-3, 7 doses of Microgynon placebo on Day 22-28 per cycle and after 3 treatment cycles 7 additional doses of Microgynon placebo on the 7 days after cycle 3 (day 84 - 91).
[14C] VilaprisanDRUGan intravenous microtracer dose of \[14C\]Vilaprisan administered together with the 1st single oral dose of Vilaprisan
Itraconazole(ITZ)OTHERItraconazole(ITZ) 200 mg as solution, once daily for 14 days
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Eligibility Criteria

Age Range18 Years to 50 Years
SexFEMALE
Healthy VolunteersNo
Study Sites64

Inclusion Criteria: * Women, 18 to 50 years of age at the time of screening * Diagnosis of uterine fibroid(s) documented by transvaginal or abdominal ultrasound at screening with at least 1 fibroid with largest diameter \>/=3.0 cm * Heavy menstrual bleeding (HMB) \>80 mL documented by menstrual pic...

Countries:AustriaBelgiumBulgariaCzechiaFinlandGermanyHungaryItalyLithuaniaNetherlandsNorwayPolandPortugalSpainSwedenUnited KingdomUnited States
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Frequently asked questions about Vilaprisan

What is Vilaprisan used for?

Vilaprisan is an investigational small molecule being studied for uterine fibroids, also known as leiomyoma, and endometriosis. It has been evaluated in clinical trials for these conditions, though it remains in clinical development and is not approved for any use.

Who makes Vilaprisan?

Vilaprisan is being developed by Bayer AG, a German pharmaceutical company traded on the OTC market under the ticker BAYRY. Bayer has sponsored multiple clinical trials of the drug across various countries.

What phase is Vilaprisan in?

Vilaprisan is in Phase 1 clinical development. While one completed trial was Phase 2, the most advanced ongoing stage is Phase 1, and the drug is still investigational. It has not been approved by regulatory authorities.

What clinical trials is Vilaprisan in?

Vilaprisan has been studied in several completed trials, including NCT02465814, a Phase 2 safety and efficacy study in patients with uterine fibroids, and NCT03092999, a Phase 1 pharmacokinetic study in hepatic impairment. Other trials include NCT03210246 and NCT03411980.

How does Vilaprisan work?

Vilaprisan is a small molecule that acts as a selective progesterone receptor modulator. It works by modulating the activity of progesterone receptors, which are involved in the growth of uterine fibroids and endometrial tissue, thereby potentially reducing symptoms of these conditions.

Is Vilaprisan the same as BAY1002670?

Yes, Vilaprisan is also known as BAY1002670. In clinical trial NCT03092999, the drug is referred to as BAY1002670 (Vilaprisan), confirming that these names refer to the same investigational compound.