Trial · NCT04517253
A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS
NCT04517253, Phase 2, Phase 3, Terminated, Eli Lilly and Company; results first posted 2022-12-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS. Trials, record NCT04517253. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04517253
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS
- NCT number
- NCT04517253
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 10 participants, actual
- Lead sponsor
- Eli Lilly and Company
- Conditions
Nakajo-Nishimura Syndrome, Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome, STING-Associated Vasculopathy With Onset in Infancy, Aicardi Goutieres Syndrome
- Interventions
- Baricitinib
- First posted
- 2020-08-18
- Last updated
- 2025-07-10
- Other registries' IDs
- eudract:2025-000001-16
- Results first posted
- 2022-12-21
- Limitations and caveats
- The study was terminated early due to efficacy/effectiveness reasons.
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period) | Baseline, 20 weeks | 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. |
| Primary | Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period) | Baseline, 32 weeks | 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. |
| Primary | Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period) | Baseline, 32 weeks | 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. |
| Secondary | Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period) | Baseline, 191.1 weeks | 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. |
| Secondary | Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period) | Baseline, 202.9 weeks | 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. |
| Secondary | Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period) | Baseline, 206.1 weeks | 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. |
| Secondary | Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period) | CANDLE: Week 20, SAVI and AGS: Week 32 | Decrease was defined as total steroid dose at the visit \<0.15 mg/kg/day (prednisone-equivalent) or \>=50% decrease from baseline. |
| Secondary | Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period) | CANDLE: Week 191.1; SAVI: 202.9 and AGS: Week 206.1 | Decrease was defined as total steroid dose \<0.15 mg/kg/day (prednisone-equivalent) or \>=50% decrease from baseline. |
| Secondary | Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) | Baseline, 20 weeks | 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. |
| Secondary | Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) | Baseline, 32 weeks | 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. |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes25
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period) | Baseline, 20 weeks | CANDLE: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
| PRIMARY | Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period) | Baseline, 32 weeks | SAVI: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
| PRIMARY | Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period) | Baseline, 32 weeks | AGS: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period) | Baseline, 191.1 weeks | CANDLE: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period) | Baseline, 202.9 weeks | SAVI: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period) | Baseline, 206.1 weeks | AGS: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
| SECONDARY | Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period) | CANDLE: Week 20, SAVI and AGS: Week 32 | 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. | participants | NUMBER |
| SECONDARY | Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period) | CANDLE: Week 191.1; SAVI: 202.9 and AGS: Week 206.1 | 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. | participants | NUMBER |
| SECONDARY | Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE (Primary Treatment Period) | Baseline, 20 weeks | CANDLE: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI (Primary Treatment Period) | Baseline, 32 weeks | SAVI: All enrolled participants who received at least one dose of study drug. | score on a scale | MEAN |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 outcomesResults by group68
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period) | CANDLE | -0.217 | Standard Deviation 0.586 | score on a scale | |
| Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period) | SAVI | -0.23 | Standard Deviation 0.238 | score on a scale | |
| Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period) | Aicardi-Goutières Syndrome (AGS) | -0.045 | Standard Deviation 0.164 | score on a scale | |
| Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period) | CANDLE | -0.24 | Standard Deviation 0.613 | score on a scale | |
| Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period) | SAVI | -0.286 | Standard Deviation 0.333 | score on a scale | |
| Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period) | Aicardi-Goutières Syndrome (AGS) | -0.08 | Standard Deviation 0.114 | score on a scale | |
| Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period) | CANDLE | 4 | participants | ||
| Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period) | SAVI | 1 | participants | ||
| Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period) | Aicardi-Goutières Syndrome (AGS) | 1 | participants | ||
| Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period) | CANDLE | 3 | participants |
1 to 10 of 68Source ClinicalTrials.gov
See all 68 results by groupParticipant flow21
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Treatment Period | STARTED | CANDLE | 5 | |
| Primary Treatment Period | STARTED | SAVI | 3 | |
| Primary Treatment Period | STARTED | Aicardi-Goutières Syndrome (AGS) | 2 | |
| Primary Treatment Period | Received at Least One Dose of Study Drug | CANDLE | 5 | |
| Primary Treatment Period | Received at Least One Dose of Study Drug | SAVI | 3 | |
| Primary Treatment Period | Received at Least One Dose of Study Drug | Aicardi-Goutières Syndrome (AGS) | 2 | |
| Primary Treatment Period | COMPLETED | CANDLE | 4 | |
| Primary Treatment Period | COMPLETED | SAVI | 2 | |
| Primary Treatment Period | COMPLETED | Aicardi-Goutières Syndrome (AGS) | 2 | |
| Primary Treatment Period | NOT COMPLETED | CANDLE | 1 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 participant flowAdverse events267
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | CANDLE | 2 | 5 | 2 |
| other | Anaemia | Blood and lymphatic system disorders | SAVI | 0 | 3 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Aicardi-Goutières Syndrome (AGS) | 0 | 2 | 0 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | CANDLE | 0 | 5 | 0 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | SAVI | 1 | 3 | 1 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Aicardi-Goutières Syndrome (AGS) | 0 | 2 | 0 |
| other | Pancytopenia | Blood and lymphatic system disorders | CANDLE | 1 | 5 | 1 |
| other | Pancytopenia | Blood and lymphatic system disorders | SAVI | 0 | 3 | 0 |
| other | Pancytopenia | Blood and lymphatic system disorders | Aicardi-Goutières Syndrome (AGS) | 0 | 2 | 0 |
| other | Thrombocytosis | Blood and lymphatic system disorders | CANDLE | 0 | 5 | 0 |
1 to 10 of 267Source ClinicalTrials.gov
See all 267 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | CANDLE | 0 | 5 |
| Total, all-cause mortality | SAVI | 1 | 3 |
| Total, all-cause mortality | Aicardi-Goutières Syndrome (AGS) | 0 | 2 |
| Total, other adverse events | CANDLE | 5 | 5 |
| Total, other adverse events | SAVI | 3 | 3 |
| Total, other adverse events | Aicardi-Goutières Syndrome (AGS) | 2 | 2 |
| Total, serious adverse events | CANDLE | 3 | 5 |
| Total, serious adverse events | SAVI | 1 | 3 |
| Total, serious adverse events | Aicardi-Goutières Syndrome (AGS) | 0 | 2 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37087470 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04517253
GET https://conductscience.org/api/trials/v1/NCT04517253/registered?page=2