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Clinical Trial Results:
A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients with NNS/CANDLE, SAVI, and AGS

Summary
EudraCT number	2025-000001-16
Trial protocol	Outside EU/EEA
Global end of trial date	28 Nov 2024

Results information
Results version number	v1(current)
This version publication date	12 Jun 2025
First version publication date	12 Jun 2025
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

I4V-JE-JAJE

ISRCTN number

US NCT number

NCT04517253

WHO universal trial number (UTN)

Other trial identifiers

Trial Number: 17571

Sponsor organisation name

Eli Lilly and Company

Sponsor organisation address

Lilly Corporate Center, Indianapolis, United States, 46285

Public contact

Available Mon ‐ Fri 9 AM ‐ 5 PM EST, Eli Lilly and Company, 1 877-CTLilly,

Scientific contact

Available Mon ‐ Fri 9 AM ‐ 5 PM EST, Eli Lilly and Company, 1 877-285-455,

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

28 Nov 2024

Is this the analysis of the primary completion data?

Yes

Primary completion date

04 Apr 2023

Global end of trial reached?

Yes

Global end of trial date

28 Nov 2024

Was the trial ended prematurely?

Main objective of the trial

The main purpose of this study is to evaluate the efficacy and safety of baricitinib in adult and pediatric Japanese participants with Nakajo-Nishimura Syndrome/chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (NNS/CANDLE), STING-associated vasculopathy with onset during infancy (SAVI), and Aicardi-Goutières Syndrome (AGS).

Protection of trial subjects

This study was conducted in accordance with International Conference on Harmonization (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.

Background therapy

Evidence for comparator

Actual start date of recruitment

27 Oct 2020

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Japan: 10

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

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 some key secondary outcomes

Screening details

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

Period 1 title

Primary Treatment Period

Is this the baseline period?

Yes

Allocation method

Not applicable

Blinding used

Not blinded

Are arms mutually exclusive

Yes

CANDLE

Arm description

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.

Arm type

Experimental

Investigational medicinal product name

LY3009104

Investigational medicinal product code

Other name

Pharmaceutical forms

Concentrate for oral solution

Routes of administration

Oral use

Dosage and administration details

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

Arm description

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.

Arm type

Experimental

Investigational medicinal product name

LY3009104

Investigational medicinal product code

Other name

Pharmaceutical forms

Concentrate for oral suspension

Routes of administration

Oral use

Dosage and administration details

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)

Arm description

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.

Arm type

Experimental

Investigational medicinal product name

LY3009104

Investigational medicinal product code

Other name

Pharmaceutical forms

Concentrate for oral suspension

Routes of administration

Oral use

Dosage and administration details

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 subjects in period 1	CANDLE	SAVI	Aicardi-Goutières Syndrome (AGS)
Started	5	3	2
Received at least one dose of study drug	5	3	2
Completed	4	2	2
Not completed	1	1	0
Adverse event, non-fatal	1	-	-
Death	-	1	-

Period 2 title

Maintenance Period

Is this the baseline period?

Allocation method

Not applicable

Blinding used

Not blinded

Are arms mutually exclusive

Yes

CANDLE

Arm description

Arm type

Experimental

Investigational medicinal product name

LY3009104

Investigational medicinal product code

Other name

Pharmaceutical forms

Concentrate for oral solution

Routes of administration

Oral use

Dosage and administration details

SAVI

Arm description

Arm type

Experimental

Investigational medicinal product name

LY3009104

Investigational medicinal product code

Other name

Pharmaceutical forms

Concentrate for oral suspension

Routes of administration

Oral use

Dosage and administration details

Aicardi-Goutières Syndrome (AGS)

Arm description

Arm type

Experimental

Investigational medicinal product name

LY3009104

Investigational medicinal product code

Other name

Pharmaceutical forms

Concentrate for oral suspension

Routes of administration

Oral use

Dosage and administration details

Number of subjects in period 2	CANDLE	SAVI	Aicardi-Goutières Syndrome (AGS)
Started	4	2	2
Completed	0	0	0
Not completed	4	2	2
Consent withdrawn by subject	1	-	-
Study Terminated by Sponsor	3	2	2

Baseline characteristics

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Reporting group title

CANDLE

Reporting group description

Reporting group title

SAVI

Reporting group description

Reporting group title

Aicardi-Goutières Syndrome (AGS)

Reporting group description

Reporting group values	CANDLE	SAVI	Aicardi-Goutières Syndrome (AGS)	Total
Number of subjects	5	3	2	10
Age categorical
Units: Subjects
Age continuous
All enrolled participants who received at least one dose of study drug.
Units: years
arithmetic mean (standard deviation)	39.20 ( 13.79 )	9.70 ( 9.81 )	7.50 ( 2.12 )	-
Gender categorical
All enrolled participants who received at least one dose of study drug.
Units: Subjects
Female	1	3	1	5
Male	4	0	1	5
Ethnicity (NIH/OMB)
All enrolled participants who received at least one dose of study drug.
Units: Subjects
Hispanic or Latino	0	0	0	0
Not Hispanic or Latino	5	3	2	10
Unknown or Not Reported	0	0	0	0
Race (NIH/OMB)
All enrolled participants who received at least one dose of study drug.
Units: Subjects
American Indian or Alaska Native	0	0	0	0
Asian	5	3	2	10
Native Hawaiian or Other Pacific Islander	0	0	0	0
Black or African American	0	0	0	0
White	0	0	0	0
More than one race	0	0	0	0
Unknown or Not Reported	0	0	0	0
Region of Enrollment
All enrolled participants who received at least one dose of study drug.
Units: Subjects
Japan	5	3	2	10
Weight
All enrolled participants who received at least one dose of study drug.
Units: Subjects
>=10 -<20	0	2	2	4
>=40 -<50	4	1	0	5
>=50 -<60	1	0	0	1
Pre- Treatment Period:Baseline (Average) Mean Daily Diary Score
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. 9999= Data Not available: SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected
Units: score on a scale
arithmetic mean (standard deviation)	0.642 ( 0.322 )	9999 ( 9999 )	9999 ( 9999 )	-
Pre-Treatment Period:Baseline (Average) Physician’s Global Assessment of Disease Activity Scores
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. 9999= Data Not available: SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.
Units: score on a scale
arithmetic mean (standard deviation)	5.45 ( 1.33 )	9999 ( 9999 )	9999 ( 9999 )	-
Pre-Treatment Period: Baseline (Average) % of days where participants' daily diary score was < 0.5
Baseline (Average) Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 was reported. 9999= Data Not available: SAVI and AGS did not have Pre-treatment period data therefore only CANDLE cohort data were collected.
Units: percentage (%)
arithmetic mean (standard deviation)	35.6 ( 24.4 )	9999 ( 9999 )	9999 ( 9999 )	-
Mean Daily Diary Score
Diaries were specific to conditions (NNS/CANDLE, SAVI, AGS). For NNS/CANDLE, participants rated symptoms (fever, rash, pain, headache, fatigue), and for SAVI, participants rated symptoms (fever, rash, pain, fatigue, respiratory issues, ulcers) on a scale from 0 (no symptoms) to 4 (severe). The mean daily score range was 0-4, with higher scores indicating more severe symptoms. For AGS, symptoms (neurologic disability, crying, sleep, seizures, fever, irritability, skin findings) were rated similarly. The mean daily score range was 0-4.25. Total score was not utilized.
Units: score on a scale
arithmetic mean (standard deviation)	0.92 ( 0.345 )	0.786 ( 0.275 )	0.705 ( 0.77 )	-
Baseline estimated glomerular filtration rate (eGFR)
All enrolled participants who received at least one dose of study drug.
Units: mL/min/1.73m^2
arithmetic mean (standard deviation)	124.69 ( 19.32 )	109.37 ( 22.50 )	99.80 ( 9.77 )	-

End points

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Reporting group title

CANDLE

Reporting group description

Reporting group title

SAVI

Reporting group description

Reporting group title

Aicardi-Goutières Syndrome (AGS)

Reporting group description

Reporting group title

CANDLE

Reporting group description

Reporting group title

SAVI

Reporting group description

Reporting group title

Aicardi-Goutières Syndrome (AGS)

Reporting group description

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period)

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End point title

Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period) ^[1] ^[2]

End point description

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. Total score was not utilized. Analysis Population Description (APD): CANDLE: All enrolled participants who received at least one dose of study drug.

End point type

Primary

End point timeframe

Baseline, 20 weeks

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.
[2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	-0.217 ( 0.586 )

No statistical analyses for this end point

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period)

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End point title

Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period) ^[3] ^[4]

End point description

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

End point type

Primary

End point timeframe

Baseline, 32 weeks

Notes
[3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: The endpoint was planned only for SAVI reporting arm in the baseline period.
[4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for SAVI reporting arm in the baseline period.

End point values	SAVI
Number of subjects analysed	3
Units: score on a scale
arithmetic mean (standard deviation)	-0.23 ( 0.238 )

No statistical analyses for this end point

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period)

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End point title

Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period) ^[5] ^[6]

End point description

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

End point type

Primary

End point timeframe

Baseline, 32 weeks

Notes
[5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: The endpoint was planned only for AGS reporting arm in the baseline period.
[6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for AGS reporting arm in the baseline period.

End point values	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	2
Units: score on a scale
arithmetic mean (standard deviation)	-0.045 ( 0.164 )

No statistical analyses for this end point

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period) ^[7]

End point description

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. Total score was not utilized. APD : CANDLE: All enrolled participants who received at least one dose of study drug.

End point type

Secondary

End point timeframe

Baseline, 191.1 weeks

Notes
[7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	-0.24 ( 0.613 )

No statistical analyses for this end point

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period) ^[8]

End point description

End point type

Secondary

End point timeframe

Baseline, 202.9 weeks

Notes
[8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for SAVI reporting arm in the baseline period.

End point values	SAVI
Number of subjects analysed	2
Units: score on a scale
arithmetic mean (standard deviation)	-0.286 ( 0.333 )

No statistical analyses for this end point

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period) ^[9]

End point description

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

End point type

Secondary

End point timeframe

Baseline, 206.1 weeks

Notes
[9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for AGS reporting arm in the baseline period.

End point values	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	2
Units: score on a scale
arithmetic mean (standard deviation)	-0.08 ( 0.114 )

No statistical analyses for this end point

Secondary: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period)

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End point title

Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period)

End point description

Decrease was defined as total steroid dose at the visit <0.15 mg/kg/day (prednisone-equivalent) or >=50% decrease from baseline. APD : CANDLE, SAVI and AGS: All enrolled participants who received at least one dose of study drug and took corticosteroids at baseline.

End point type

Secondary

End point timeframe

CANDLE: Week 20, SAVI and AGS: Week 32

End point values	CANDLE	SAVI	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	5	1	1
Units: participants
number (not applicable)	4	1	1

No statistical analyses for this end point

Secondary: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period)

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End point title

Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period)

End point description

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

End point type

Secondary

End point timeframe

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

End point values	CANDLE	SAVI	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	5	1	1
Units: participants
number (not applicable)	3	1	1

No statistical analyses for this end point

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period)

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End point title

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) ^[10]

End point description

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

End point type

Secondary

End point timeframe

Baseline, 20 weeks

Notes
[10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)
Fatigue	-0.029 ( 0.997 )
Fever	0 ( 0 )
Headache	0.057 ( 0.128 )
Musculo-skeletal Pain	-0.514 ( 1.128 )
Rash	-0.6 ( 0.894 )

No statistical analyses for this end point

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period)

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End point title

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) ^[11]

End point description

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

End point type

Secondary

End point timeframe

Baseline, 32 weeks

Notes
[11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for SAVI reporting arm in the baseline period.

End point values	SAVI
Number of subjects analysed	3
Units: score on a scale
arithmetic mean (standard deviation)
Fatigue	-0.381 ( 0.541 )
Fever	-0.095 ( 0.165 )
Musculo-skeletal Pain	-0.238 ( 0.412 )
Rash	0.333 ( 0.577 )
Respiratory / Breathing Symptoms	0.333 ( 0.577 )
Ulcers / Ischemic Lesions	0 ( 0 )

No statistical analyses for this end point

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period)

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End point title

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period) ^[12]

End point description

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

End point type

Secondary

End point timeframe

Baseline, 32 weeks

Notes
[12] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for AGS reporting arm in the baseline period.

End point values	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	2
Units: score on a scale
arithmetic mean (standard deviation)
Crying	0 ( 0 )
Excessive Irritability	0 ( 0 )
Fever	-0.071 ( 0.101 )
Generalized Seizure	0 ( 0 )
Length of Uninterrupted Sleep	0.286 ( 0.404 )
Neurologic Disability	0 ( 0 )
Skin Findings (Body)	-0.5 ( 0.707 )
Skin Findings (Hands, Feet, Ears)	-0.707 ( 0.101 )

No statistical analyses for this end point

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) ^[13]

End point description

End point type

Secondary

End point timeframe

Baseline, 191.1 weeks

Notes
[13] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)
Fatigue	-0.057 ( 1.172 )
Fever	0 ( 0 )
Headache	0.057 ( 0.128 )
Musculo-skeletal Pain	-0.514 ( 1.128 )
Rash	-0.686 ( 0.842 )

No statistical analyses for this end point

Secondary: change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period)

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End point title

change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period) ^[14]

End point description

End point type

Secondary

End point timeframe

Baseline, 202.9 weeks

Notes
[14] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for SAVI reporting arm in the baseline period.

End point values	SAVI
Number of subjects analysed	3
Units: score on a scale
arithmetic mean (standard deviation)
Fatigue	-0.381 ( 0.541 )
Fever	-0.095 ( 0.165 )
Musculo-skeletal Pain	-0.238 ( 0.412 )
Rash	-0.333 ( 0.577 )
Respiratory / Breathing Symptoms	-0.667 ( 1.155 )
Ulcers / Ischemic Lesions	0 ( 0 )

No statistical analyses for this end point

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period) ^[15]

End point description

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

End point type

Secondary

End point timeframe

Baseline, 206.1 weeks

Notes
[15] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for AGS reporting arm in the baseline period.

End point values	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	2
Units: score on a scale
arithmetic mean (standard deviation)
Crying	0 ( 0 )
Excessive Irritability	-0.071 ( 0.101 )
Fever	0 ( 0 )
Generalized Seizure	0 ( 0 )
Length of Uninterrupted Sleep	0 ( 0 )
Neurologic Disability	0 ( 0 )
Skin Findings (Body)	-0.5 ( 0.707 )
Skin Findings on (Hands, Feet, Ears)	-0.071 ( 0.101 )

No statistical analyses for this end point

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment Period)

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End point title

Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment Period) ^[16]

End point description

End point type

Secondary

End point timeframe

Baseline, 20 weeks

Notes
[16] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	-2.1 ( 0.82 )

No statistical analyses for this end point

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment Period)

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End point title

Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment Period) ^[17]

End point description

End point type

Secondary

End point timeframe

Baseline, 32 weeks

Notes
[17] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for SAVI and AGS reporting arm in the baseline period.

End point values	SAVI	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	3	2
Units: score on a scale
arithmetic mean (standard deviation)	-1.33 ( 1.76 )	-3 ( 3.54 )

No statistical analyses for this end point

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment and Maintenance Period) ^[18]

End point description

End point type

Secondary

End point timeframe

Baseline, 191.1 weeks

Notes
[18] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	-2.6 ( 2.07 )

No statistical analyses for this end point

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment and Maintenance Period) ^[19]

End point description

End point type

Secondary

End point timeframe

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

Notes
[19] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for SAVI and AGS reporting arm in the baseline period.

End point values	SAVI	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	3	2
Units: score on a scale
arithmetic mean (standard deviation)	-1.5 ( 1.73 )	-3.75 ( 3.89 )

No statistical analyses for this end point

Secondary: 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)

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End point title

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) ^[20]

End point description

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 APD : 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.

End point type

Secondary

End point timeframe

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

Notes
[20] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: Percentage Change
arithmetic mean (standard deviation)	4.4 ( 48.7 )

No statistical analyses for this end point

Secondary: 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)

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End point title

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) ^[21]

End point description

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. APD : 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.

End point type

Secondary

End point timeframe

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

Notes
[21] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: Percentage Change
arithmetic mean (standard deviation)	2.0 ( 47.1 )

No statistical analyses for this end point

Secondary: Change From Baseline in Growth Velocity (Height and Weight Z Score) (Primary Treatment and Maintenance Period)

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End point title

Change From Baseline in Growth Velocity (Height and Weight Z Score) (Primary Treatment and Maintenance Period)

End point description

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

End point type

Secondary

End point timeframe

CANDLE: Baseline, 191.1 weeks; SAVI: Baseline, 202.9 weeks and AGS: Baseline, 206.1 weeks APD: CANDLE, SAVI and AGS: All enrolled participants who received at least one dose of study drug and had evaluable data for this outcome.

End point values	CANDLE	SAVI	Aicardi-Goutières Syndrome (AGS)
Number of subjects analysed	1 ^[22]	2	2
Units: Z-score
arithmetic mean (standard deviation)
Height	3.03 ( 99999 )	0.75 ( 0.88 )	0.13 ( 1.17 )
Weight	0.92 ( 99999 )	1.17 ( 0.03 )	0.46 ( 1.16 )

Notes
[22] - 99999; standard deviation is not calculable as n=1

No statistical analyses for this end point

Secondary: Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment Period)

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End point title

Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) ^[23]

End point description

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. APD : 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.

End point type

Secondary

End point timeframe

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

Notes
[23] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	0.061 ( 0.432 )

No statistical analyses for this end point

Secondary: Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

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End point title

Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) ^[24]

End point description

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. APD : 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

End point type

Secondary

End point timeframe

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

Notes
[24] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	0.038 ( 0.464 )

No statistical analyses for this end point

Secondary: Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment Period)

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End point title

Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment Period) ^[25]

End point description

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. APD : 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. APD : 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.

End point type

Secondary

End point timeframe

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

Notes
[25] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	-1.75 ( 0.83 )

No statistical analyses for this end point

Secondary: Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

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End point title

Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment and Maintenance Period) ^[26]

End point description

End point type

Secondary

End point timeframe

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

Notes
[26] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: The endpoint was planned only for CANDLE reporting arm in the baseline period.

End point values	CANDLE
Number of subjects analysed	5
Units: score on a scale
arithmetic mean (standard deviation)	-2.25 ( 1.79 )

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

CANDLE: Baseline Up to 191.1 Weeks; SAVI: Baseline Up to 202.9 Weeks and AGS: Baseline Up to 206.1 Weeks

Adverse event reporting additional description

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.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

27.1

Reporting group title

CANDLE

Reporting group description

Reporting group title

Aicardi-Goutières Syndrome (AGS)

Reporting group description

Reporting group title

SAVI

Reporting group description

Serious adverse events	CANDLE	Aicardi-Goutières Syndrome (AGS)	SAVI
Total subjects affected by serious adverse events
subjects affected / exposed	3 / 5 (60.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
number of deaths (all causes)	0	0	1
number of deaths resulting from adverse events
Cardiac disorders
acute coronary syndrome
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Nervous system disorders
haemorrhage intracranial
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 1
hypoaesthesia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Blood and lymphatic system disorders
pancytopenia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
nausea
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Infections and infestations
atypical mycobacterial infection
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences causally related to treatment / all	0 / 0	0 / 0	1 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
bronchopulmonary aspergillosis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences causally related to treatment / all	0 / 0	0 / 0	1 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
pneumonia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 0%

Non-serious adverse events	CANDLE	Aicardi-Goutières Syndrome (AGS)	SAVI
Total subjects affected by non serious adverse events
subjects affected / exposed	5 / 5 (100.00%)	2 / 2 (100.00%)	3 / 3 (100.00%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
skin papilloma
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Surgical and medical procedures
continuous haemodiafiltration
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
haematoma evacuation
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
lung assist device therapy
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
mechanical ventilation
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
pneumonectomy
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
tracheostomy
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
General disorders and administration site conditions
oedema peripheral
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
pyrexia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
xerosis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Reproductive system and breast disorders
menstruation irregular
alternative dictionary used: MedDRA 27.1
subjects affected / exposed ^[1]	1 / 1 (100.00%)	0 / 1 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Respiratory, thoracic and mediastinal disorders
cough
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	0 / 3 (0.00%)
occurrences all number	0	1	0
nasal inflammation
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
rhinorrhoea
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
upper respiratory tract inflammation
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	1 / 3 (33.33%)
occurrences all number	0	2	1
rhinitis allergic
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
Psychiatric disorders
head banging
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
Investigations
bk polyomavirus test positive
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	3 / 5 (60.00%)	1 / 2 (50.00%)	0 / 3 (0.00%)
occurrences all number	3	1	0
blood creatine phosphokinase increased
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	1 / 2 (50.00%)	2 / 3 (66.67%)
occurrences all number	1	1	2
lymphocyte count decreased
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	3
lymphocyte count increased
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
weight increased
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
weight decreased
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Injury, poisoning and procedural complications
contusion
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
spinal compression fracture
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
tooth fracture
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Nervous system disorders
dizziness
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	2	0	0
headache
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
neuropathy peripheral
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
restless legs syndrome
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
post herpetic neuralgia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Blood and lymphatic system disorders
anaemia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	2	0	0
iron deficiency anaemia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
pancytopenia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
thrombocytosis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	1 / 3 (33.33%)
occurrences all number	0	2	1
Eye disorders
blepharitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	0 / 3 (0.00%)
occurrences all number	0	1	0
conjunctivitis allergic
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Gastrointestinal disorders
abdominal distension
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
dental caries
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	2 / 3 (66.67%)
occurrences all number	0	1	3
enterocolitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
diarrhoea
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	1	0	2
gastritis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
mouth ulceration
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
nausea
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	2	0	0
stomatitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	2	0	0
Hepatobiliary disorders
hepatic function abnormal
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
hepatic steatosis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Skin and subcutaneous tissue disorders
acne
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
dermatitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
eczema
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	2 / 2 (100.00%)	1 / 3 (33.33%)
occurrences all number	4	2	1
pruritus
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	4	0	0
rosacea
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
seborrhoeic dermatitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
urticaria
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
Musculoskeletal and connective tissue disorders
arthralgia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
Infections and infestations
bk virus infection
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
covid-19
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	2	0	1
adenoviral conjunctivitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	0 / 3 (0.00%)
occurrences all number	0	1	0
cytomegalovirus chorioretinitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
conjunctivitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
diarrhoea infectious
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
folliculitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
gastroenteritis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	2 / 3 (66.67%)
occurrences all number	0	0	2
influenza
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	2	0	1
hordeolum
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	1
herpes zoster
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
localised infection
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
metapneumovirus infection
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	0 / 3 (0.00%)
occurrences all number	0	1	0
nasopharyngitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	2 / 2 (100.00%)	0 / 3 (0.00%)
occurrences all number	2	23	0
otitis media
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	1 / 2 (50.00%)	0 / 3 (0.00%)
occurrences all number	1	1	0
parotitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
periodontitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
pharyngitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	0 / 3 (0.00%)
occurrences all number	0	1	0
pneumocystis jirovecii pneumonia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
pneumonia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	1 / 2 (50.00%)	1 / 3 (33.33%)
occurrences all number	0	1	1
pneumonia cytomegaloviral
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
respiratory syncytial virus infection
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	0 / 5 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)
occurrences all number	0	0	2
upper respiratory tract infection
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	2 / 3 (66.67%)
occurrences all number	1	0	17
tinea capitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0
tracheitis
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	2 / 5 (40.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	2	0	0
Metabolism and nutrition disorders
hyperuricaemia
alternative dictionary used: MedDRA 27.1
subjects affected / exposed	1 / 5 (20.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)
occurrences all number	1	0	0

Notes
[1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
Justification: Gender specific events occurring only in male or female participants have had the number of participants at risk adjusted accordingly.

More information

Top of page

Were there any global substantial amendments to the protocol? Yes

18 Jun 2020

- Modified endpoints for secondary objective on participant’s daily diary scores - Schedule of activities were modified - Modified sentences and added information for Overall Design - inclusion and exclusion criteria were modified - Modified sentences on Adverse Events of Special Interest for virus infection and thrombocytosis (add the definition of thrombocytosis)

28 Jul 2020

- CANDLE was changed to NNS or NNS/CANDLE - Hepatic Monitoring Tests for Treatment-Emergent Abnormality Table was replaced with the latest version of the required template - Inequality symbols were corrected to appropriately describe patient weight class above and below40 kilogram (kg)

09 Dec 2020

- Schedule of Activities, Benefit/Risk Assessment, Inclusion and Exclusion criteria were modified - Added a note in Hepatic Safety Data Collection To follow latest lilly guidance for hepatic monitoring - Added new procedure during exceptional circumstances (To maintain this study during COVID-19 restrictions)

07 Apr 2022

- Added wording to explain that the study will be considered a postmarketing clinical trial after marketing authorization under summary of design - Updated visit and week numbers for maintenance treatment period from Visit 24 (Week 100) to Visit 30 (Week 172) - Schedule of Activities were modified - Added “The investigator will be responsible for reporting significant issues related to participant safety, participant rights, or data integrity.” to align with protocol requirement update - Revised Final Report Signature to “The clinical study report (CSR) coordinating investigator will sign the final CSR for this study, indicating agreement that, to the best of his or her knowledge, the report accurately describes the conduct and results of the study.”

04 Sep 2023

- Update visit and week numbers for maintenance treatment period from visit 30 (Week 172) to Visit 36 (Week 244) - Schedule of Activities were updated - Added relevant visits to Hepatitis B Virus DNA Monitoring To reflect changes made to the schedule of activities

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

The study was terminated due to efficacy/effectiveness reasons.

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Clinical trials

Clinical Trial Results: A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients with NNS/CANDLE, SAVI, and AGS

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period)

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period)

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period)

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period)

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period)

Primary: Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period)

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period)

Secondary: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period)

Secondary: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period)

Secondary: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period)

Secondary: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period)

Secondary: change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in the Physician’s Global Assessment of Disease Activity Scores in Participants With SAVI and AGS (Primary Treatment and Maintenance Period)

Secondary: 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)

Secondary: 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)

Secondary: 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)

Secondary: 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)

Secondary: Change From Baseline in Growth Velocity (Height and Weight Z Score) (Primary Treatment and Maintenance Period)

Secondary: Change From Baseline in Growth Velocity (Height and Weight Z Score) (Primary Treatment and Maintenance Period)

Secondary: Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment Period)

Secondary: Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

Secondary: Change From Pre-treatment Period in the Physician’s Global Assessment of Disease Activity Scores For Participants With CANDLE (Primary Treatment and Maintenance Period)

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients with NNS/CANDLE, SAVI, and AGS