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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04517253
Other study ID # 17571
Secondary ID I4V-JE-JAJE
Status Active, not recruiting
Phase Phase 2/Phase 3
First received
Last updated
Start date October 27, 2020
Est. completion date April 30, 2025

Study information

Verified date April 2024
Source Eli Lilly and Company
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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).


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 8
Est. completion date April 30, 2025
Est. primary completion date October 12, 2021
Accepts healthy volunteers No
Gender All
Age group 6 Months and older
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

Drug:
Baricitinib
Administered orally

Locations

Country Name City State
Japan Tokyo Medical And Dental University Medical Hospital Bunkyo Tokyo
Japan Hiroshima University Hospital Hiroshima
Japan Nara Medical University Hospital Kashihara Nara
Japan National Center For Child Health And Development Setagaya-ku Tokyo
Japan Wakayama Medical University Hospital Wakayama

Sponsors (1)

Lead Sponsor Collaborator
Eli Lilly and Company

Country where clinical trial is conducted

Japan, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change From Baseline in Mean Daily Diary Scores in Participants With SAVI 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. Baseline, up to 32 weeks
Primary Change From Baseline in Mean Daily Diary Scores in Participants With 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. Baseline, up to 32 weeks
Primary Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE 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 Change From Baseline in Mean Daily Diary Scores Change from Baseline in Mean Daily Diary Scores Baseline, up to 172 weeks
Secondary Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With SAVI and AGS Decrease was defined as total steroid dose at the visit <0.15 mg/kg/day (prednisone-equivalent) or >=50% decrease from baseline. Week 32
Secondary Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE Decrease was defined as total steroid dose at the visit <0.15 mg/kg/day (prednisone-equivalent) or >=50% decrease from baseline. Week 20
Secondary Number of Participants With Decrease in Daily Dose of Corticosteroids Number of participants with decrease in Daily Dose of Corticosteroids Baseline, up to 172 weeks
Secondary Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI 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
Secondary Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS 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
Secondary Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE 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 Change From Baseline in Patient's Symptom Specific Daily Diary Scores Change from Baseline in Patient's Symptom Specific Daily Diary Scores Baseline, up to 172 weeks
Secondary Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With SAVI and AGS 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". Baseline, up to 32 weeks
Secondary Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With CANDLE 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
Secondary Change From Baseline in the Physician's Global Assessment of Disease Activity Scores Change From Baseline in the Physician's Global Assessment of Disease Activity Scores Baseline, up to 172 weeks
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 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 Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
Secondary Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
Secondary Change in Growth Velocity Change in Growth Velocity Baseline, up to 172 weeks
Secondary Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE 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
Secondary Change From Pre-treatment Period in Mean Daily Diary Scores Change from Pre-treatment period in Mean Daily Diary Scores Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
Secondary Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores For Participants With CANDLE 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
Secondary Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores 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
See also
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