Trial Outcomes & Findings for A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS (NCT NCT04517253)

Last Updated: 2025-07-10

Results Overview

Diaries were specific to individual indications or conditions (i.e., NNS/CANDLE, SAVI, or AGS). For NNS/CANDLE, participant or caregiver was instructed to rate each symptom (fever, rash, musculoskeletal pain, headache and fatigue in the diary on a scale from 0 to 4 (where a score of 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms, 3 = more severe symptoms, and 4 = severe symptoms \[equivalent to "worst" symptoms\]. The mean daily score range was 0-4 with the higher score indicating a more severe symptom. Total score was not utilized.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

10 participants

Primary outcome timeframe

Baseline, 20 weeks

Results posted on

2025-07-10

Participant Flow

Per analysis plan,only for participants with chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE),a pretreatment period lasted for 12 weeks, during which natural history data were collected.This data serves as baseline to assess changes during baricitinib treatment for evaluating key secondary outcomes: Percentage of days with a mean daily symptom score below 0.5,Average daily symptom score,and the Physician's Global Assessment of Disease Activity scores.

Participants who met all eligibility criteria underwent an 8-week dose adjustment period and received an optimized dosage of baricitinib during the primary treatment period (12 weeks \[week 8 to week 20\] for CANDLE and 24 weeks \[week 8 to week 32\] for SAVI or AGS participants), followed by a maintenance period of up to 191.1 weeks for CANDLE participants, and up to 202.9 weeks for SAVI participants, and up to 206.1 weeks for AGS participants.

Participant milestones

Participant milestones
Measure	CANDLE Participants with chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE) were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and estimated glomerular filtration rate (eGFR). Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with stimulator of interferon genes (STING)-associated vasculopathy with onset during infancy (SAVI) were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with Aicardi-Goutières Syndrome (AGS) were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Primary Treatment Period STARTED	5	3	2
Primary Treatment Period Received at Least One Dose of Study Drug	5	3	2
Primary Treatment Period COMPLETED	4	2	2
Primary Treatment Period NOT COMPLETED	1	1	0
Maintenance Period STARTED	4	2	2
Maintenance Period COMPLETED	0	0	0
Maintenance Period NOT COMPLETED	4	2	2

Reasons for withdrawal

Reasons for withdrawal
Measure	CANDLE Participants with chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE) were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and estimated glomerular filtration rate (eGFR). Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with stimulator of interferon genes (STING)-associated vasculopathy with onset during infancy (SAVI) were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with Aicardi-Goutières Syndrome (AGS) were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Primary Treatment Period Adverse Event	1	0	0
Primary Treatment Period Death	0	1	0
Maintenance Period Withdrawal by Subject	1	0	0
Maintenance Period Study Terminated by Sponsor	3	2	2

Baseline Characteristics

The questionnaire in the daily diary score differs among diseases (CANDLE, SAVI, and AGS), and we cannot calculate a total score. Hence, data are reported separately, and 0 is entered for the Numbers Analyzed for the Arms/Groups to which a particular row does not apply for each Baseline Measure.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI n=3 Participants Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) n=2 Participants Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.	Total n=10 Participants Total of all reporting groups
Age, Continuous	39.20 years STANDARD_DEVIATION 13.79 • n=5 Participants	9.70 years STANDARD_DEVIATION 9.81 • n=3 Participants	7.50 years STANDARD_DEVIATION 2.12 • n=2 Participants	24.00 years STANDARD_DEVIATION 19.07 • n=10 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	3 Participants n=3 Participants	1 Participants n=2 Participants	5 Participants n=10 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	0 Participants n=3 Participants	1 Participants n=2 Participants	5 Participants n=10 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants	3 Participants n=3 Participants	2 Participants n=2 Participants	10 Participants n=10 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants	3 Participants n=3 Participants	2 Participants n=2 Participants	10 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	0 Participants n=10 Participants
Region of Enrollment Japan	5 participants n=5 Participants	3 participants n=3 Participants	2 participants n=2 Participants	10 participants n=10 Participants
Weight >=10 -<20	0 Participants n=5 Participants	2 Participants n=3 Participants	2 Participants n=2 Participants	4 Participants n=10 Participants
Weight >=40 -<50	4 Participants n=5 Participants	1 Participants n=3 Participants	0 Participants n=2 Participants	5 Participants n=10 Participants
Weight >=50 -<60	1 Participants n=5 Participants	0 Participants n=3 Participants	0 Participants n=2 Participants	1 Participants n=10 Participants
Baseline estimated glomerular filtration rate (eGFR)	124.69 mL/min/1.73m^2 STANDARD_DEVIATION 19.32 • n=5 Participants	109.37 mL/min/1.73m^2 STANDARD_DEVIATION 22.50 • n=3 Participants	99.80 mL/min/1.73m^2 STANDARD_DEVIATION 9.77 • n=2 Participants	115.11 mL/min/1.73m^2 STANDARD_DEVIATION 20.08 • n=10 Participants
Mean Daily Diary Score CANDLE	0.92 score on a scale STANDARD_DEVIATION 0.345 • n=5 Participants • The questionnaire in the daily diary score differs among diseases (CANDLE, SAVI, and AGS), and we cannot calculate a total score. Hence, data are reported separately, and 0 is entered for the Numbers Analyzed for the Arms/Groups to which a particular row does not apply for each Baseline Measure.	—	—	0.92 score on a scale STANDARD_DEVIATION 0.345 • n=5 Participants • The questionnaire in the daily diary score differs among diseases (CANDLE, SAVI, and AGS), and we cannot calculate a total score. Hence, data are reported separately, and 0 is entered for the Numbers Analyzed for the Arms/Groups to which a particular row does not apply for each Baseline Measure.
Mean Daily Diary Score SAVI	—	0.786 score on a scale STANDARD_DEVIATION 0.275 • n=3 Participants • The questionnaire in the daily diary score differs among diseases (CANDLE, SAVI, and AGS), and we cannot calculate a total score. Hence, data are reported separately, and 0 is entered for the Numbers Analyzed for the Arms/Groups to which a particular row does not apply for each Baseline Measure.	—	0.786 score on a scale STANDARD_DEVIATION 0.275 • n=3 Participants • The questionnaire in the daily diary score differs among diseases (CANDLE, SAVI, and AGS), and we cannot calculate a total score. Hence, data are reported separately, and 0 is entered for the Numbers Analyzed for the Arms/Groups to which a particular row does not apply for each Baseline Measure.
Mean Daily Diary Score AGS	—	—	0.705 score on a scale STANDARD_DEVIATION 0.77 • n=2 Participants • The questionnaire in the daily diary score differs among diseases (CANDLE, SAVI, and AGS), and we cannot calculate a total score. Hence, data are reported separately, and 0 is entered for the Numbers Analyzed for the Arms/Groups to which a particular row does not apply for each Baseline Measure.	0.705 score on a scale STANDARD_DEVIATION 0.77 • n=2 Participants • The questionnaire in the daily diary score differs among diseases (CANDLE, SAVI, and AGS), and we cannot calculate a total score. Hence, data are reported separately, and 0 is entered for the Numbers Analyzed for the Arms/Groups to which a particular row does not apply for each Baseline Measure.
Pre- Treatment Period:Baseline (Average) Mean Daily Diary Score	0.642 score on a scale STANDARD_DEVIATION 0.322 • n=5 Participants • SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.	—	—	0.642 score on a scale STANDARD_DEVIATION 0.322 • n=5 Participants • SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.
Pre-Treatment Period:Baseline (Average) Physician's Global Assessment of Disease Activity Scores	5.45 score on a scale STANDARD_DEVIATION 1.33 • n=5 Participants • SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.	—	—	5.45 score on a scale STANDARD_DEVIATION 1.33 • n=5 Participants • SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.
Pre-Treatment Period: Baseline (Average) % of days where participants' daily diary score was < 0.5.	35.6 Percentage (%) of days STANDARD_DEVIATION 24.4 • n=5 Participants • SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.	—	—	35.6 Percentage (%) of days STANDARD_DEVIATION 24.4 • n=5 Participants • SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.

PRIMARY outcome

Timeframe: Baseline, 20 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period)	-0.217 score on a scale Standard Deviation 0.586	—	—

PRIMARY outcome

Timeframe: Baseline, 32 weeks

Population: SAVI: All enrolled participants who received at least one dose of study drug.

Diaries were specific to individual indications or conditions (i.e. NNS/CANDLE, SAVI, or AGS). For SAVI, participant or caregiver was instructed to rate each symptom (fever, rash, musculoskeletal pain, fatigue, respiratory/breathing problems, and ulcers/ischemic lesions in the diary on a scale from 0 to 4 (where a score of 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms,3 = more severe symptoms, and 4 = severe symptoms \[equivalent to "worst" symptoms\].The mean daily score range was 0-4 with the higher score indicating a more severe symptom. Total score was not utilized.

Outcome measures

Outcome measures
Measure	CANDLE n=3 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period)	-0.23 score on a scale Standard Deviation 0.238	—	—

PRIMARY outcome

Timeframe: Baseline, 32 weeks

Population: AGS: All enrolled participants who received at least one dose of study drug.

Diaries were specific to individual indications or conditions (i.e. NNS/CANDLE, SAVI, or AGS). For AGS, participant or caregiver was instructed to rate each symptom (rating) (neurologic disability (0, 5, 7,10) crying (0, 1, 2, 3), length of uninterrupted sleep (0, 1, 2, 3), generalized seizure (0, 8), fever (0,1), excessive irritability (0, 1, 2, 3), skin findings(body) (0, 1, 2, 3), and skin findings (hands, feet, and ears) (0, 1, 2, 3) with a higher score for each symptom indicating a more severe symptom. The mean daily diary score was the average of all symptom scores and the range was 0 - 4.25 with the higher score indicating a more severe symptom. Total score was not utilized.

Outcome measures

Outcome measures
Measure	CANDLE n=2 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period)	-0.045 score on a scale Standard Deviation 0.164	—	—

SECONDARY outcome

Timeframe: Baseline, 191.1 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)	-0.24 score on a scale Standard Deviation 0.613	—	—

SECONDARY outcome

Timeframe: Baseline, 202.9 weeks

Population: SAVI: All enrolled participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	CANDLE n=3 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period)	-0.286 score on a scale Standard Deviation 0.333	—	—

SECONDARY outcome

Timeframe: Baseline, 206.1 weeks

Population: AGS: All enrolled participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	CANDLE n=2 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period)	-0.08 score on a scale Standard Deviation 0.114	—	—

SECONDARY outcome

Timeframe: CANDLE: Week 20, SAVI and AGS: Week 32

Population: CANDLE, SAVI and AGS: All enrolled participants who received at least one dose of study drug and took corticosteroids at baseline with evaluable data for this outcome.

Decrease was defined as total steroid dose at the visit \<0.15 mg/kg/day (prednisone-equivalent) or \>=50% decrease from baseline.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI n=1 Participants Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) n=1 Participants Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period)	4 participants	1 participants	1 participants

SECONDARY outcome

Timeframe: CANDLE: Week 191.1; SAVI: 202.9 and AGS: Week 206.1

Population: CANDLE, SAVI and AGS: All enrolled participants who received at least one dose of study drug and took corticosteroids at baseline with evaluable data for this outcome.

Decrease was defined as total steroid dose \<0.15 mg/kg/day (prednisone-equivalent) or \>=50% decrease from baseline.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI n=1 Participants Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) n=1 Participants Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period)	3 participants	1 participants	1 participants

SECONDARY outcome

Timeframe: Baseline, 20 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug.

Diaries were specific to individual indications or conditions (i.e, NNS/CANDLE,SAVI, or AGS). For NNS/CANDLE, participant or caregiver was instructed to rate each symptom (fatigue, fever, headache, musculoskeletal pain and rash in the diary on a scale from 0 to 4, where a score of 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms, 3 = more severe symptoms, and 4 = severe symptoms \[equivalent to "worst" symptoms\]). The mean daily score range was 0-4 with the higher score indicating a more severe symptom.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) Fatigue	-0.029 score on a scale Standard Deviation 0.997	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) Fever	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) Headache	0.057 score on a scale Standard Deviation 0.128	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) Musculo-skeletal Pain	-0.514 score on a scale Standard Deviation 1.128	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) Rash	-0.6 score on a scale Standard Deviation 0.894	—	—

SECONDARY outcome

Timeframe: Baseline, 32 weeks

Population: SAVI: All enrolled participants who received at least one dose of study drug.

Diaries were specific to individual indications or conditions (i.e. NNS/CANDLE, SAVI, or AGS). For SAVI, participant or caregiver was instructed to rate each symptom (fatigue, fever, musculoskeletal pain, rash, respiratory/breathing problems, and ulcers/ischemic lesions in the diary on a scale from 0 to 4, where a score of 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms, 3 = more severe symptoms, and 4 = severe symptoms \[equivalent to "worst" symptoms\]). The mean daily score range was 0-4 with the higher score indicating a more severe symptom.

Outcome measures

Outcome measures
Measure	CANDLE n=3 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) Fatigue	-0.381 score on a scale Standard Deviation 0.541	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) Fever	-0.095 score on a scale Standard Deviation 0.165	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) Musculo-skeletal Pain	-0.238 score on a scale Standard Deviation 0.412	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) Rash	-0.333 score on a scale Standard Deviation 0.577	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) Respiratory / Breathing Symptoms	-0.333 score on a scale Standard Deviation 0.577	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) Ulcers / Ischemic Lesions	0 score on a scale Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: Baseline, 32 weeks

Population: AGS: All enrolled participants who received at least one dose of study drug.

Diaries were specific to individual indications or conditions (i.e. NNS/CANDLE, SAVI, or AGS). For AGS, participant or caregiver was instructed to rate each symptom (rating) (crying (0, 1, 2, 3), excessive irritability (0, 1, 2, 3), fever (0,1), generalized seizure (0, 8), length of uninterrupted sleep (0, 1, 2, 3), neurologic disability (0, 5, 7,10), skin findings(body) (0, 1, 2, 3), and skin findings (hands, feet, and ears) (0, 1, 2, 3) with a higher score for each symptom indicating a more severe symptom.

Outcome measures

Outcome measures
Measure	CANDLE n=2 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Crying	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Excessive Irritability	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Fever	-0.071 score on a scale Standard Deviation 0.101	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Generalized Seizure	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Length of Uninterrupted Sleep	0.286 score on a scale Standard Deviation 0.404	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Neurologic Disability	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Skin Findings (Body)	-0.5 score on a scale Standard Deviation 0.707	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) Skin Findings (Hands, Feet, Ears)	-0.071 score on a scale Standard Deviation 0.101	—	—

SECONDARY outcome

Timeframe: Baseline, 191.1 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) Fatigue	-0.057 score on a scale Standard Deviation 1.172	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) Fever	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) Headache	0.057 score on a scale Standard Deviation 0.128	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) Musculo-skeletal Pain	-0.514 score on a scale Standard Deviation 1.128	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) Rash	-0.686 score on a scale Standard Deviation 0.842	—	—

SECONDARY outcome

Timeframe: Baseline, 202.9 weeks

Population: SAVI: All enrolled participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	CANDLE n=3 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period) Fatigue	-0.381 score on a scale Standard Deviation 0.541	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period) Fever	-0.095 score on a scale Standard Deviation 0.165	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period) Musculo-skeletal Pain	-0.238 score on a scale Standard Deviation 0.412	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period) Rash	-0.333 score on a scale Standard Deviation 0.577	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period) Respiratory / Breathing Symptoms	-0.667 score on a scale Standard Deviation 1.155	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period) Ulcers / Ischemic Lesions	0 score on a scale Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: Baseline, 206.1 weeks

Population: AGS: All enrolled participants who received at least one dose of study drug.

Diaries were specific to individual indications or conditions (i.e. NNS/CANDLE, SAVI, or AGS). For AGS, participant or caregiver was instructed to rate each symptom (rating) (crying (0, 1, 2, 3), excessive irritability (0, 1, 2, 3), fever (0,1), generalized seizure (0, 8), length of uninterrupted sleep (0, 1, 2, 3), neurologic disability (0, 5, 7,10), skin findings(body) (0, 1, 2, 3), and skin findings (hands, feet, and ears) (0, 1, 2, 3) with a higher score for each symptom indicating a more severe symptom.

Outcome measures

Outcome measures
Measure	CANDLE n=2 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Generalized Seizure	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Crying	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Excessive Irritability	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Fever	-0.071 score on a scale Standard Deviation 0.101	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Length of Uninterrupted Sleep	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Neurologic Disability	0 score on a scale Standard Deviation 0	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Skin Findings (Body)	-0.5 score on a scale Standard Deviation 0.707	—	—
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) Skin Findings on (Hands, Feet, Ears)	-0.071 score on a scale Standard Deviation 0.101	—	—

SECONDARY outcome

Timeframe: Baseline, 20 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug.

The Physician's Global Assessment of Disease Activity is used to assess the patient's current disease activity, as it relates to their signs and symptoms. The instrument uses a 21-circle Visual Analog Scale (VAS) ranging from 0 to 10 (using 0.5 increments) where 0 = "no activity" and 10 = "maximum activity" (Filocamo et al. 2010). Higher scores indicate greater disease severity.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment Period)	-2.1 score on a scale Standard Deviation 0.82	—	—

SECONDARY outcome

Timeframe: Baseline, 32 weeks

Population: SAVI and AGS: All enrolled participants who received at least one dose of study drug.

The Physician's Global Assessment of Disease Activity is used to assess the patient's current disease activity, as it relates to their signs and symptoms. The instrument uses a 21-circle VAS ranging from 0 to 10 (using 0.5 increments) where 0 = "no activity" and 10 = "maximum activity" (Filocamo et al. 2010). Higher scores indicate greater disease severity.

Outcome measures

Outcome measures
Measure	CANDLE n=3 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI n=2 Participants Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment Period)	-1.33 score on a scale Standard Deviation 1.76	-3 score on a scale Standard Deviation 3.54	—

SECONDARY outcome

Timeframe: Baseline, 191.1 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)	-2.6 score on a scale Standard Deviation 2.07	—	—

SECONDARY outcome

Timeframe: SAVI: Baseline, 202.9 weeks; AGS: Baseline, 206.1 weeks

Population: SAVI and AGS: All enrolled participants who received at least one dose of study drug.

The Physician's Global Assessment of Disease Activity is used to assess the patient's current disease activity, as it relates to their signs and symptoms. The instrument uses a 21-circle VAS ranging from 0 to 10 (using 0.5 increments) where 0 = "no activity" and 10 = "maximum activity" (Filocamo et al. 2010). Higher scores indicate greater disease severity.

Outcome measures

Outcome measures
Measure	CANDLE n=3 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI n=2 Participants Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Maintenance Period)	-1.5 score on a scale Standard Deviation 1.73	-3.75 score on a scale Standard Deviation 3.89	—

SECONDARY outcome

Timeframe: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug. SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.

Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score \<0.5 Compared to that in Pre-treatment period in Participants with CANDLE was evaluated

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 Compared to That in Pre-treatment Period in Participants With CANDLE (Primary Treatment Period)	4.4 Percentage of Days Standard Deviation 48.7	—	—

SECONDARY outcome

Timeframe: Pre-treatment period (average of 12-week pre-treatment data), up to 191.1 weeks

Population: CANDLE: All enrolled participants who received at least one dose of study drug. SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.

Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score \<0.5 Compared to That in Pre-treatment Period in Participants With CANDLE was evaluated.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 Compared to That in Pre-treatment Period in Participants With CANDLE (Primary Treatment and Maintenance Period)	2.0 Percentage of Days Standard Deviation 47.1	—	—

SECONDARY outcome

Timeframe: CANDLE: Baseline, 191.1 weeks; SAVI: Baseline, 202.9 weeks and AGS: Baseline, 206.1 weeks

Population: CANDLE, SAVI and AGS: All enrolled participants who received at least one dose of study drug and had evaluable data for this outcome.

The change from baseline in normalized scores for body weight or height is measured using Z-scores. A Z-score indicates how many standard deviations a person's height or body weight is above or below the average for their age and gender. Z-score which was calculated by (value - \[mean of the population\]) / \[SD of the population\] at a given age/sex. Interpretation: Z-score of 0: The measurement is equal to the population mean. Z-score less than 0: The measurement is below the population mean. Z-score greater than 0: The measurement is above the population mean. An increase in the Z-score for weight or height means that the weight or height of the participants has increased more than the standard population during the study. For study participants with Z-scores less than 0 at baseline, an increase in Z-score is considered a positive outcome. A Z-score within the range of -2 to +2 indicates that the height or weight is within the normal range.

Outcome measures

Outcome measures
Measure	CANDLE n=1 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI n=2 Participants Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) n=2 Participants Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Baseline in Growth Velocity (Height and Weight Z Score) (Primary Treatment and Maintenance Period) Height	3.03 Z-score Standard Deviation NA Standard Deviation is not calculable as number of participants analyzed (n) =1	0.75 Z-score Standard Deviation 0.88	0.13 Z-score Standard Deviation 1.17
Change From Baseline in Growth Velocity (Height and Weight Z Score) (Primary Treatment and Maintenance Period) Weight	0.92 Z-score Standard Deviation NA Standard Deviation is not calculable as n=1	1.17 Z-score Standard Deviation 0.03	0.46 Z-score Standard Deviation 1.16

SECONDARY outcome

Timeframe: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks

Population: CANDLE: All participants who received at least one dose of study drug. SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.

Diaries were specific to individual indications or conditions (ie, NNS/CANDLE,SAVI, or AGS). For NNS/CANDLE, participant or caregiver was instructed to rate each symptom (fever, rash, musculoskeletal pain, headache and fatigue in the diary on a scale from 0 to 4 (where a score of 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms, 3 = more severe symptoms, and 4 = severe symptoms \[equivalent to "worst" symptoms\]. The mean daily score range was 0-4 with the higher score indicating a more severe symptom.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment Period)	0.061 score on a scale Standard Deviation 0.432	—	—

SECONDARY outcome

Timeframe: Pre-treatment period (average of 12-week pre-treatment data), up to 191.1 weeks

Population: CANDLE: All participants who received at least one dose of study drug. SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)	0.038 score on a scale Standard Deviation 0.464	—	—

SECONDARY outcome

Timeframe: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks

Population: CANDLE: All participants who received at least one dose of study drug. SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment Period)	-1.75 score on a scale Standard Deviation 0.83	—	—

SECONDARY outcome

Timeframe: Pre-treatment period (average of 12-week pre-treatment data), up to 191.1 weeks

Population: CANDLE: All participants who received at least one dose of study drug. SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.

Outcome measures

Outcome measures
Measure	CANDLE n=5 Participants Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)	-2.25 score on a scale Standard Deviation 1.79	—	—

Adverse Events

CANDLE

Serious events: 3 serious events

Other events: 5 other events

Deaths: 0 deaths

SAVI

Serious events: 1 serious events

Other events: 3 other events

Deaths: 1 deaths

Aicardi-Goutières Syndrome (AGS)

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	CANDLE n=5 participants at risk Participants with CANDLE were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.	SAVI n=3 participants at risk Participants with SAVI were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.	Aicardi-Goutières Syndrome (AGS) n=2 participants at risk Participants with AGS were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.
Blood and lymphatic system disorders Pancytopenia	20.0% 1/5 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/3 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.
Cardiac disorders Acute coronary syndrome	20.0% 1/5 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/3 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.
Gastrointestinal disorders Nausea	20.0% 1/5 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/3 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.
Infections and infestations Atypical mycobacterial infection	0.00% 0/5 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	33.3% 1/3 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.
Infections and infestations Bronchopulmonary aspergillosis	0.00% 0/5 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	33.3% 1/3 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.
Infections and infestations Pneumonia	20.0% 1/5 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/3 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.
Nervous system disorders Haemorrhage intracranial	0.00% 0/5 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	33.3% 1/3 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.
Nervous system disorders Hypoaesthesia	20.0% 1/5 • Number of events 1 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/3 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.	0.00% 0/2 • CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks All enrolled participants who received at least one dose of study drug. Based on the planned safety analysis, adverse events were collected per the treatment regimen, irrespective of dose. Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.

Other adverse events

Additional Information

Chief Medical Officer

Eli Lilly and Company

Phone: 800-545-5979

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60