- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04517253
A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS
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
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Locations
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-
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Hiroshima, Japan, 734-8551
- Hiroshima University Hospital
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Wakayama, Japan, 641-0012
- Wakayama Medical University Hospital
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Nara
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Kashihara, Nara, Japan, 634-0813
- Nara Medical University Hospital
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Tokyo
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Bunkyō, Tokyo, Japan, 113-8519
- Tokyo Medical And Dental University Medical Hospital
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Setagaya-ku, Tokyo, Japan, 157-8535
- National Center for Child Health and Development
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Have systemic signs and symptoms of inflammation as manifested NNS/CANDLE, SAVI, AGS
- Have been diagnosed with genetic diagnosis
- Men must agree to use a reliable method of birth control during the study
- Women not of child-bearing potential or nonbreastfeeding
- Women must agree to use birth control or remain abstinent during the study and for at least 12 weeks after stopping treatment
- NNS/CANDLE and SAVI patients who are ≥17.5 months of age
- AGS patients who are ≥6 months of age
- Are ≥ 5kg in body weight
Exclusion Criteria:
- Have received immunosuppressive biologic agent/monoclonal antibody/oral JAK inhibitor/OAT3 inhibitor and cannot discontinue prior to investigational product initiation. Note: A washout period is required to each drug.
- Have diagnosis of current active tuberculosis (TB) or, latent TB who did not receive appropriate treatment.
- Have had a serious infection within 12 weeks prior to screening.
- Have a history of lymphoproliferative disease
- Have any history of venous thromboembolic event (VTE) (deep vein thrombosis [DVT]/pulmonary embolism [PE]) prior to screening.
- Have had any major surgery within 8 weeks prior to screening.
- Have previously been enrolled in any other study investigating baricitinib.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Baricitinib
CANDLE: Participants with chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE) received an optimized dosage of baricitinib that was determined throughout the dose-adjustment period administered as tablets or oral suspension based on participants weight and estimated glomerular filtration rate (eGFR). SAVI: Participants with STING-associated vasculopathy with onset during infancy (SAVI) received an optimized dosage of baricitinib that was determined throughout the dose-adjustment period administered as tablets or oral suspension based on participants weight and estimated glomerular filtration rate (eGFR). Aicardi-Goutières Syndrome (AGS): Participants with Aicardi-Goutières Syndrome (AGS) received an optimized dosage of baricitinib that was determined throughout the dose-adjustment period administered as tablets or oral suspension based on participants weight and estimated glomerular filtration rate (eGFR). |
Administered orally
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in Mean Daily Diary Scores in Participants With SAVI
Time Frame: Baseline, up to 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.
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Baseline, up to 32 weeks
|
Change From Baseline in Mean Daily Diary Scores in Participants With AGS
Time Frame: Baseline, up to 32 weeks
|
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.
|
Baseline, up to 32 weeks
|
Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE
Time Frame: Baseline, up to 20 weeks
|
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.
|
Baseline, up to 20 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in Mean Daily Diary Scores
Time Frame: Baseline, up to 172 weeks
|
Change from Baseline in Mean Daily Diary Scores
|
Baseline, up to 172 weeks
|
Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With SAVI and AGS
Time Frame: Week 32
|
Decrease was defined as total steroid dose at the visit <0.15 mg/kg/day (prednisone-equivalent) or >=50% decrease from baseline.
|
Week 32
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Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE
Time Frame: Week 20
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Decrease was defined as total steroid dose at the visit <0.15 mg/kg/day (prednisone-equivalent) or >=50% decrease from baseline.
|
Week 20
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Number of Participants With Decrease in Daily Dose of Corticosteroids
Time Frame: Baseline, up to 172 weeks
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Number of participants with decrease in Daily Dose of Corticosteroids
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Baseline, up to 172 weeks
|
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI
Time Frame: Baseline, up to 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.
|
Baseline, up to 32 weeks
|
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS
Time Frame: Baseline, up to 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.
|
Baseline, up to 32 weeks
|
Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE
Time Frame: Baseline, up to 20 weeks
|
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.
|
Baseline, up to 20 weeks
|
Change From Baseline in Patient's Symptom Specific Daily Diary Scores
Time Frame: Baseline, up to 172 weeks
|
Change from Baseline in Patient's Symptom Specific Daily Diary Scores
|
Baseline, up to 172 weeks
|
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With SAVI and AGS
Time Frame: Baseline, up to 32 weeks
|
The Physician's Global Assessment of Disease Activity is used to assess the patient's current disease activity, as it relates to their signs and symptoms.
The instrument uses a 21-circle VAS ranging from 0 to 10 (using 0.5 increments) where 0 = "no activity" and 10 = "maximum activity".
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Baseline, up to 32 weeks
|
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With CANDLE
Time Frame: Baseline, up to 20 weeks
|
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".
|
Baseline, up to 20 weeks
|
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores
Time Frame: Baseline, up to 172 weeks
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Change From Baseline in the Physician's Global Assessment of Disease Activity Scores
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Baseline, up to 172 weeks
|
Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 Compared to That in Pre-treatment Period in Participants With CANDLE
Time Frame: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
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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
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Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
|
Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5
Time Frame: Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
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Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5
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Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
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Change in Growth Velocity
Time Frame: Baseline, up to 172 weeks
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Change in Growth Velocity
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Baseline, up to 172 weeks
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Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE
Time Frame: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
|
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.
|
Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
|
Change From Pre-treatment Period in Mean Daily Diary Scores
Time Frame: Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
|
Change from Pre-treatment period in Mean Daily Diary Scores
|
Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
|
Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores For Participants With CANDLE
Time Frame: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
|
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).
|
Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
|
Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores
Time Frame: Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
|
Change from Pre-treatment period in the Physician's Global Assessment of Disease Activity Scores
|
Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST), Eli Lilly and Company
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 17571
- I4V-JE-JAJE (Other Identifier: Eli Lilly and Company)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Time Frame
IPD Sharing Access Criteria
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
- CSR
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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