Trial Outcomes & Findings for Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1 (NCT NCT02574910)
NCT ID: NCT02574910
Last Updated: 2025-11-04
Results Overview
The original primary endpoint was the dose of abiraterone acetate that normalized androstenedione to age-appropriate levels in 7/8 subjects after 7 days of treatment. However, an insufficient number of participants was enrolled to evaluate the primary endpoint. Therefore, we have instead presented the number of participants at each dose level who did normalize androstenedione to age-appropriate levels.
Recruitment status
TERMINATED
Study phase
PHASE1
Target enrollment
4 participants
Primary outcome timeframe
7 days
Results posted on
2025-11-04
Participant Flow
Participant milestones
| Measure |
Abiraterone acetate 1 mg/kg/d
Abiraterone acetate will be administered orally at a daily dose of 1 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 2 mg/kg/d
If the 1 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 2 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 4 mg/kg/d
If the 2 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 4 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
|---|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
0
|
|
Overall Study
COMPLETED
|
2
|
2
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Baseline characteristics by cohort
| Measure |
Abiraterone acetate 1 mg/kg/d
n=2 Participants
Abiraterone acetate will be administered orally at a daily dose of 1 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 2 mg/kg/d
n=2 Participants
If the 1 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 2 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 4 mg/kg/d
If the 2 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 4 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=15 Participants
|
2 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
4 Participants
n=3 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
0 Participants
n=3 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
0 Participants
n=3 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=15 Participants
|
2 participants
n=161 Participants
|
—
|
4 participants
n=3 Participants
|
|
Age, Continuous
|
5 years
n=15 Participants
|
7 years
n=161 Participants
|
—
|
6 years
n=3 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=15 Participants
|
1 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
2 Participants
n=3 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=15 Participants
|
1 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
2 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=15 Participants
|
2 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
2 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
2 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
—
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
—
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
—
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
—
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=15 Participants
|
2 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
4 Participants
n=3 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
—
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
—
|
0 Participants
n=3 Participants
|
PRIMARY outcome
Timeframe: 7 daysPopulation: No subjects enrolled at 4 mg/kg/d
The original primary endpoint was the dose of abiraterone acetate that normalized androstenedione to age-appropriate levels in 7/8 subjects after 7 days of treatment. However, an insufficient number of participants was enrolled to evaluate the primary endpoint. Therefore, we have instead presented the number of participants at each dose level who did normalize androstenedione to age-appropriate levels.
Outcome measures
| Measure |
Abiraterone acetate 1 mg/kg/d
n=2 Participants
Abiraterone acetate will be administered orally at a daily dose of 1 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 2 mg/kg/d
n=2 Participants
If the 1 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 2 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 4 mg/kg/d
If the 2 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 4 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
|---|---|---|---|
|
Normalization of Serum Androstenedione Level
|
0 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 7 daysPopulation: Due to early termination of this study and incomplete enrollment, the collected data are incomplete and limited. These collected samples will never be analyzed in the future. Due to quality issues with the resulting of 17-hydroxyprogestoerone, there is one per-protocol patient to report this result on.
Final lab results at Screening and V6. This outcome is not pre-specified in the protocol and was inadvertently added at the time of registration.
Outcome measures
| Measure |
Abiraterone acetate 1 mg/kg/d
n=1 Participants
Abiraterone acetate will be administered orally at a daily dose of 1 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 2 mg/kg/d
If the 1 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 2 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 4 mg/kg/d
If the 2 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 4 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
|---|---|---|---|
|
17-hydroxyprogestoerone Levels
|
-2790 ng/dL
|
—
|
—
|
SECONDARY outcome
Timeframe: 7 daysPopulation: The values for this outcome measurement were not collected and we have no data relating to PK to report because the collected samples were not processed for this outcome measure and will never be processed in the future as well.
Sparse pharmacokinetic (PK) will be derived from peak and trough abiraterone levels.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 7 daysPopulation: The values for this outcome measurement were not collected and we have no data relating to PK to report because the collected samples were not processed for this outcome measure and will never be processed in the future as well.
Sparse pharmacokinetic (PK) will be derived from peak and trough abiraterone levels.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: 7 daysPopulation: Due to early termination of this study and incomplete enrollment, the collected data are incomplete and limited, it is not considered valid to perform this outcome measure assessment and therefore unable to analyze and report it.
Final lab results at Screening and V6. This was erroneously listed as a secondary outcome in PRS. We have changed the outcome measure type to "Other pre-specified." .
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: 7 daysPopulation: No participants enrolled at the 4 mg/kg/d level.
Safety monitoring in Phase 1 will include liver function tests (AST, Alanine Aminotransferease (ALT), bilirubin) and possible mineralocorticoid effects (blood pressure, plasma renin).
Outcome measures
| Measure |
Abiraterone acetate 1 mg/kg/d
n=2 Participants
Abiraterone acetate will be administered orally at a daily dose of 1 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 2 mg/kg/d
n=2 Participants
If the 1 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 2 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 4 mg/kg/d
If the 2 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 4 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
|---|---|---|---|
|
Number of Adverse Events
|
6 events
|
1 events
|
—
|
Adverse Events
Abiraterone acetate 1 mg/kg/d
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Abiraterone acetate 2 mg/kg/d
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Abiraterone acetate 4 mg/kg/d
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Abiraterone acetate 1 mg/kg/d
n=2 participants at risk
Abiraterone acetate will be administered orally at a daily dose of 1 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 2 mg/kg/d
n=2 participants at risk
If the 1 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 2 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
Abiraterone acetate 4 mg/kg/d
If the 2 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 4 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Abiraterone acetate: This is a dose escalation study. Up to 3 successive cohorts of 8 subjects each will receive 7 days of 1, 2 or 4 mg/kg/d of abiraterone acetate, until 7/8 subjects have met the desired endpoint.
|
|---|---|---|---|
|
Nervous system disorders
Headache
|
50.0%
1/2 • Number of events 2 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
0.00%
0/2 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
—
0/0 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
|
Gastrointestinal disorders
vomiting
|
50.0%
1/2 • Number of events 2 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
50.0%
1/2 • Number of events 1 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
—
0/0 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
|
General disorders
Fever
|
50.0%
1/2 • Number of events 1 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
0.00%
0/2 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
—
0/0 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
|
Skin and subcutaneous tissue disorders
abrasion
|
50.0%
1/2 • Number of events 1 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
0.00%
0/2 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
—
0/0 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
0.00%
0/2 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
50.0%
1/2 • Number of events 1 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
—
0/0 • 60 days
No subjects were enrolled at the 4 mg/kg/d dose level.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place