Trial Outcomes & Findings for Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia (NCT NCT04045145)

Last Updated: 2024-07-18

Results Overview

Percent changes in 17-OHP were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The 2 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

Baseline, Day 14

Results posted on

2024-07-18

Participant Flow

Participant milestones

Participant milestones
Measure	Crinecerfont 50 mg BID Participants received crinecerfont 50 milligrams (mg) twice daily (BID) orally for 14 consecutive days.
Overall Study STARTED	8
Overall Study Received at Least 1 Dose of Study Drug	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Testosterone (morning window average) is presented by female and male participants separately.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Crinecerfont 50 BID n=8 Participants Participants received crinecerfont 50 mg BID orally for 14 consecutive days.
Age, Continuous	15.0 years STANDARD_DEVIATION 0.9 • n=8 Participants
Sex: Female, Male Female	5 Participants n=8 Participants
Sex: Female, Male Male	3 Participants n=8 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=8 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=8 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=8 Participants
Race/Ethnicity, Customized Asian	1 Participants n=8 Participants
Race/Ethnicity, Customized White	7 Participants n=8 Participants
17-hydroxyprogesterone (OHP) (Morning Window Average)	7703.74 nanograms/deciliter (ng/dL) n=8 Participants
17-OHP (24-Hour Average)	2739.74 ng/dL n=8 Participants
Androstenedione Morning Window Average)	367.88 ng/dL n=8 Participants
Adrenocorticotropic hormone (ACTH) (Morning Window Average)	226.23 picograms/milliliter (pg/mL) n=8 Participants
Testosterone (Morning Window Average) Female	63.50 ng/dL n=5 Participants • Testosterone (morning window average) is presented by female and male participants separately.
Testosterone (Morning Window Average) Male	222.00 ng/dL n=3 Participants • Testosterone (morning window average) is presented by female and male participants separately.

PRIMARY outcome

Timeframe: Baseline, Day 14

Population: Pharmacodynamic (PD) analysis set included all enrolled participants who received at least one dose of study drug and had at least one PD parameter measurement at baseline and at least one PD parameter measurement at the Day 14 visit.

Outcome measures

Outcome measures
Measure	Crinecerfont 50 mg n=8 Participants Participants received crinecerfont 50 mg BID orally for 14 consecutive days.
Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average)	-69.48 percent change Interval -89.2 to -23.05

SECONDARY outcome

Timeframe: Baseline, Day 14

Population: PD analysis set included all enrolled participants who received at least one dose of study drug and had at least one PD parameter measurement at baseline and at least one PD parameter measurement at the Day 14 visit.

Percent changes in 17-OHP were assessed through the collection of samples for a 24-hour period prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The average of each participant's serial sampling values at each visit where serial sampling was performed was calculated and used to determine the percent change from baseline.

Outcome measures

Outcome measures
Measure	Crinecerfont 50 mg n=8 Participants Participants received crinecerfont 50 mg BID orally for 14 consecutive days.
Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period)	-76.0 percent change Interval -82.34 to -29.74

SECONDARY outcome

Timeframe: Baseline, Day 14

Percent changes in ACTH were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.

Outcome measures

Outcome measures
Measure	Crinecerfont 50 mg n=8 Participants Participants received crinecerfont 50 mg BID orally for 14 consecutive days.
Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages)	-57.12 percent change Interval -70.08 to -28.45

SECONDARY outcome

Timeframe: Baseline, Day 14

Percent changes in androstenedione were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.

Outcome measures

Outcome measures
Measure	Crinecerfont 50 mg n=8 Participants Participants received crinecerfont 50 mg BID orally for 14 consecutive days.
Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages)	-58.27 percent change Interval -82.22 to -11.99

SECONDARY outcome

Timeframe: Baseline, Day 14

Percent change in testosterone were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.

Outcome measures

Outcome measures
Measure	Crinecerfont 50 mg n=8 Participants Participants received crinecerfont 50 mg BID orally for 14 consecutive days.
Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages) Female	-76.18 percent change Interval -83.53 to -28.57
Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages) Male	1.39 percent change Interval -2.67 to 49.55

Adverse Events

Crinecerfont 50 mg

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Crinecerfont 50 mg n=8 participants at risk Participants received crinecerfont 50 mg BID orally for 14 consecutive days.
Nervous system disorders Headache	25.0% 2/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Injury, poisoning and procedural complications Arthropod sting	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Eye disorders Blepharospasm	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Skin and subcutaneous tissue disorders Dermatitis contact	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Nervous system disorders Dizziness	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Gastrointestinal disorders Frequent bowel movements	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Gastrointestinal disorders Gastritis	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Musculoskeletal and connective tissue disorders Myalgia	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Infections and infestations Nasopharyngitis	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
General disorders Pyrexia	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
Gastrointestinal disorders Vomiting	12.5% 1/8 • Day 1 (after dosing) up to Day 35 Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data. Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.

Additional Information

Neurocrine Medical Information Call Center

Neurocrine Biosciences

Phone: 877-641-3461

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place