Trial Outcomes & Findings for Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study) (NCT NCT04806451)

Last Updated: 2025-02-05

Results Overview

Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

103 participants

Primary outcome timeframe

Baseline, Week 4

Results posted on

2025-02-05

Participant Flow

The study includes a double-blind (DB) placebo-controlled treatment period, an open-label (OL) treatment period, and an open-label extension (OLE) period. The OL treatment period and OLE period of the study is ongoing. Only the primary analysis results (as of 09 August 2023 data cutoff date) have been reported. The final results will be reported after completion of the study.

Participant milestones

Participant milestones
Measure	Placebo Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.	Crinecerfont Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.
DB Treatment Period (28 Weeks) STARTED	34	69
DB Treatment Period (28 Weeks) Received at Least 1 Dose of Study Drug	33	69
DB Treatment Period (28 Weeks) COMPLETED	31	66
DB Treatment Period (28 Weeks) NOT COMPLETED	3	3
OL Treatment Period (24 Weeks) STARTED	31	66
OL Treatment Period (24 Weeks) Received at Least 1 Dose of Study Drug	31	66
OL Treatment Period (24 Weeks) COMPLETED	15	29
OL Treatment Period (24 Weeks) NOT COMPLETED	16	37

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.	Crinecerfont Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.
DB Treatment Period (28 Weeks) Withdrawal by Subject	3	1
DB Treatment Period (28 Weeks) Adverse Event	0	2
OL Treatment Period (24 Weeks) Ongoing in the Study	16	37

Baseline Characteristics

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=34 Participants Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.	Crinecerfont n=69 Participants Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.	Total n=103 Participants Total of all reporting groups
Age, Continuous	12.142 years STANDARD_DEVIATION 3.690 • n=5 Participants	12.022 years STANDARD_DEVIATION 3.405 • n=7 Participants	12.061 years STANDARD_DEVIATION 3.484 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants	34 Participants n=7 Participants	50 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants	35 Participants n=7 Participants	53 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants	8 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	25 Participants n=5 Participants	52 Participants n=7 Participants	77 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	6 Participants n=5 Participants	9 Participants n=7 Participants	15 Participants n=5 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	2 Participants n=5 Participants	7 Participants n=7 Participants	9 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	0 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	23 Participants n=5 Participants	42 Participants n=7 Participants	65 Participants n=5 Participants
Race/Ethnicity, Customized Race · Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race/Ethnicity, Customized Race · Multiple	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized Race · Not Collected	6 Participants n=5 Participants	9 Participants n=7 Participants	15 Participants n=5 Participants
Serum Androstenedione Concentration	483.32 nanograms (ng)/deciliter (dL) STANDARD_DEVIATION 455.64 • n=5 Participants	404.96 nanograms (ng)/deciliter (dL) STANDARD_DEVIATION 464.10 • n=7 Participants	430.83 nanograms (ng)/deciliter (dL) STANDARD_DEVIATION 460.58 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 4

Population: Full Analysis Set included all randomized participants.

Outcome measures

Outcome measures
Measure	Placebo n=34 Participants Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.	Crinecerfont n=69 Participants Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.
Change From Baseline in Serum Androstenedione at Week 4	70.986 ng/dL Standard Error 56.198	-196.789 ng/dL Standard Error 39.369

SECONDARY outcome

Timeframe: Baseline, Week 4

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 28

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 28

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 28

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 28

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 28

Outcome measures

Outcome data not reported

Adverse Events

DB Period: Placebo

Serious events: 4 serious events

Other events: 22 other events

Deaths: 0 deaths

DB Period: Crinecerfont

Serious events: 1 serious events

Other events: 51 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	DB Period: Placebo n=33 participants at risk Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period.	DB Period: Crinecerfont n=69 participants at risk Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1.
General disorders Pyrexia	0.00% 0/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	1.4% 1/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Gastrointestinal disorders Vomiting	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	0.00% 0/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Pharyngitis	3.0% 1/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	0.00% 0/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
General disorders Chest pain	3.0% 1/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	0.00% 0/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Gastroenteritis norovirus	3.0% 1/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	0.00% 0/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Gastroenteritis	3.0% 1/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	0.00% 0/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Gastroenteritis viral	3.0% 1/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	0.00% 0/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.

Other adverse events

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

Measure	DB Period: Placebo n=33 participants at risk Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period.	DB Period: Crinecerfont n=69 participants at risk Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1.
Nervous system disorders Headache	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	24.6% 17/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
General disorders Pyrexia	24.2% 8/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	21.7% 15/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Gastrointestinal disorders Vomiting	24.2% 8/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	14.5% 10/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Upper respiratory tract infection	0.00% 0/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	11.6% 8/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Nasopharyngitis	18.2% 6/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	10.1% 7/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Influenza	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	8.7% 6/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Gastrointestinal disorders Abdominal pain	0.00% 0/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	7.2% 5/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Coronavirus infection	9.1% 3/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	7.2% 5/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
General disorders Fatigue	0.00% 0/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	7.2% 5/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Respiratory, thoracic and mediastinal disorders Nasal congestion	3.0% 1/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	7.2% 5/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Respiratory, thoracic and mediastinal disorders Cough	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	5.8% 4/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Nervous system disorders Dizziness	9.1% 3/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	5.8% 4/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Gastrointestinal disorders Nausea	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	5.8% 4/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Pharyngitis streptococcal	0.00% 0/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	5.8% 4/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Viral infection	3.0% 1/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	5.8% 4/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Investigations 17-hydroxyprogesterone increased	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	4.3% 3/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Investigations Blood androstenedione increased	9.1% 3/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	4.3% 3/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Investigations Blood corticotrophin increased	9.1% 3/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	2.9% 2/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Gastrointestinal disorders Diarrhoea	9.1% 3/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	2.9% 2/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Investigations Blood insulin increased	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	1.4% 1/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Gastrointestinal disorders Constipation	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	1.4% 1/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
Infections and infestations Tonsillitis	6.1% 2/33 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.	0.00% 0/69 • Baseline up to Week 28 Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.