Trial Outcomes & Findings for A Study of Safety and Efficacy of Tildacerfont in Females With Polycystic Ovary Syndrome and Elevated Adrenal Androgens (NCT NCT05370521)
NCT ID: NCT05370521
Last Updated: 2025-10-09
Results Overview
To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
27 participants
Primary outcome timeframe
12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Results posted on
2025-10-09
Participant Flow
Of the 82 subjects that were screened, a total of 27 subjects enrolled in the study (10 subjects in the placebo arm and 17 subjects in the tildacerfont arm).
Participant milestones
| Measure |
Treatment With Tildacerfont
Subjects randomized in this arm received 4 weeks of 50mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
Subjects randomized in this arm received 12 weeks of oral matched-placebo tablet
|
|---|---|---|
|
Overall Study
STARTED
|
17
|
10
|
|
Overall Study
COMPLETED
|
16
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of Safety and Efficacy of Tildacerfont in Females With Polycystic Ovary Syndrome and Elevated Adrenal Androgens
Baseline characteristics by cohort
| Measure |
Treatment With Tildacerfont
n=17 Participants
Subjects randomized in this arm will receive 4 weeks of 50mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
n=10 Participants
Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
17 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
27 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
27 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=93 Participants
|
9 Participants
n=4 Participants
|
24 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
13 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
14 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Dehydroepiandrosterone sulfate (DHEAS)
|
351.3 ug/dL
STANDARD_DEVIATION 90.53 • n=93 Participants
|
387.8 ug/dL
STANDARD_DEVIATION 107.16 • n=4 Participants
|
364.8 ug/dL
STANDARD_DEVIATION 96.65 • n=27 Participants
|
|
Adrenocorticotropic hormone (ACTH)
|
23.88 pg/dL
STANDARD_DEVIATION 11.927 • n=93 Participants
|
22.3 pg/dL
STANDARD_DEVIATION 12.013 • n=4 Participants
|
23.27 pg/dL
STANDARD_DEVIATION 11.744 • n=27 Participants
|
|
17-hydroxyprogesterone (17-OHP)
|
83.2 ng/dL
STANDARD_DEVIATION 86.28 • n=93 Participants
|
62.1 ng/dL
STANDARD_DEVIATION 54.1 • n=4 Participants
|
75.4 ng/dL
STANDARD_DEVIATION 75.51 • n=27 Participants
|
|
Androstenedione (A4)
|
185.2 ng/dL
STANDARD_DEVIATION 75.19 • n=93 Participants
|
130.0 ng/dL
STANDARD_DEVIATION 65.96 • n=4 Participants
|
166.8 ng/dL
STANDARD_DEVIATION 75.61 • n=27 Participants
|
|
Testosterone
|
61.0 ng/dL
STANDARD_DEVIATION 21.97 • n=93 Participants
|
61.1 ng/dL
STANDARD_DEVIATION 26.97 • n=4 Participants
|
61.0 ng/dL
STANDARD_DEVIATION 23.43 • n=27 Participants
|
|
Cortisol
|
13.44 ug/dL
STANDARD_DEVIATION 4.847 • n=93 Participants
|
16.41 ug/dL
STANDARD_DEVIATION 7.206 • n=4 Participants
|
14.54 ug/dL
STANDARD_DEVIATION 5.879 • n=27 Participants
|
PRIMARY outcome
Timeframe: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)Population: Analysis of Change from Baseline in DHEAS (μg/dL) at Week 12, Modified Intent-to-Treat Analysis Set.
To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens
Outcome measures
| Measure |
Treatment With Tildacerfont
n=16 Participants
Subjects randomized in this arm will receive 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
n=10 Participants
Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
|
|---|---|---|
|
Change in DHEAS
|
-22.93 ug/dL
Standard Deviation 67.600
|
-7.56 ug/dL
Standard Deviation 16.00
|
SECONDARY outcome
Timeframe: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)Population: Number of subjects with a \>/=30% change from Baseline in DHEAS (μg/dL) at Week 12, Modified Intent-to-Treat Analysis Set.
Number of subjects with \>/=30% change from baseline in DHEAS
Outcome measures
| Measure |
Treatment With Tildacerfont
n=16 Participants
Subjects randomized in this arm will receive 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
n=10 Participants
Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
|
|---|---|---|
|
Reduction in DHEAS
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)Population: Number of subjects with DHEAS =/\< upper limit of normal for DHEAS at Week 12, Modified Intent-to-treat Analysis Set.
Number of subjects with DHEAS =/\< upper limit of normal for DHEAS
Outcome measures
| Measure |
Treatment With Tildacerfont
n=16 Participants
Subjects randomized in this arm will receive 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
n=10 Participants
Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
|
|---|---|---|
|
Normalization of DHEAS
|
8 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)Population: All subjects who received at least 1 dose of placebo or tildacerfont; Safety Analysis Set.
Evaluation of safety of tildacerfont with PCOS as measured by number of subjects with adverse events following dosing by CTCAE Version 5
Outcome measures
| Measure |
Treatment With Tildacerfont
n=17 Participants
Subjects randomized in this arm will receive 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
n=10 Participants
Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
|
|---|---|---|
|
Number of Subjects With TEAE as Assessed by CTCAE Version 5
|
13 Participants
|
9 Participants
|
Adverse Events
Treatment With Tildacerfont
Serious events: 1 serious events
Other events: 13 other events
Deaths: 0 deaths
Placebo Control Arm
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment With Tildacerfont
n=17 participants at risk
Subjects randomized in this arm will receive 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
n=10 participants at risk
Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
|
|---|---|---|
|
Gastrointestinal disorders
Obstructive pancreatitis
|
5.9%
1/17 • Number of events 1 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
Other adverse events
| Measure |
Treatment With Tildacerfont
n=17 participants at risk
Subjects randomized in this arm will receive 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment.
|
Placebo Control Arm
n=10 participants at risk
Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
|
|---|---|---|
|
Ear and labyrinth disorders
Vertigo positional
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Gastrointestinal disorders
Diarrhea
|
47.1%
8/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Gastrointestinal disorders
Abdominal Pain
|
11.8%
2/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Gastrointestinal disorders
Abdominal pain lower
|
11.8%
2/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Gastrointestinal disorders
Nausea
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Gastrointestinal disorders
Haematochezia
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Gastrointestinal disorders
Vomiting
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
General disorders
Fatigue
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
20.0%
2/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
General disorders
Pyrexia
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Nasopharyngitis
|
11.8%
2/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
COVID-19
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Vulvovaginal mycotic infection
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Gastroenteritis viral
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Oral herpes
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Otitis media
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Urinary tract infection
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Infections and infestations
Viral rhinitis
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Investigations
Blood potassium increased
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Investigations
Total bile acids increased
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Investigations
Weight increased
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Fibrous histiocytoma
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Nervous system disorders
Headache
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Nervous system disorders
Tension headache
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Psychiatric disorders
Anxiety
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Psychiatric disorders
Insomnia
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Psychiatric disorders
Mood swings
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
20.0%
2/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Skin and subcutaneous tissue disorders
Pruitis
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
10.0%
1/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
|
Gastrointestinal disorders
Gastrointestinal Pain
|
5.9%
1/17 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
0.00%
0/10 • 12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place