Clinical trial NCT04517253: A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS
Official title: 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 type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Nakajo-Nishimura Syndrome
Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome
STING-Associated Vasculopathy With Onset in Infancy
Aicardi Goutieres Syndrome
Keywords: Nakajo-Nishimura Syndrome
CANDLE
SAVI
AGS
Lead sponsor: Eli Lilly and Company
Sponsor class: INDUSTRY
First posted: 2020-08-18
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).


Eligibility criteria: 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.

Primary outcomes:
measure: Change From Baseline in Mean Daily Diary Scores in Participants With SAVI
timeFrame: Baseline, up to 32 weeks
measure: Change From Baseline in Mean Daily Diary Scores in Participants With AGS
timeFrame: Baseline, up to 32 weeks
measure: Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE
timeFrame: Baseline, up to 20 weeks
Secondary outcomes:
measure: Change From Baseline in Mean Daily Diary Scores
timeFrame: Baseline, up to 172 weeks
measure: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With SAVI and AGS
timeFrame: Week 32
measure: Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE
timeFrame: Week 20
measure: Number of Participants With Decrease in Daily Dose of Corticosteroids
timeFrame: Baseline, up to 172 weeks
measure: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI
timeFrame: Baseline, up to 32 weeks
measure: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS
timeFrame: Baseline, up to 32 weeks
measure: Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE
timeFrame: Baseline, up to 20 weeks
measure: Change From Baseline in Patient's Symptom Specific Daily Diary Scores
timeFrame: Baseline, up to 172 weeks
measure: Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With SAVI and AGS
timeFrame: Baseline, up to 32 weeks
measure: Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With CANDLE
timeFrame: Baseline, up to 20 weeks
measure: Change From Baseline in the Physician's Global Assessment of Disease Activity Scores
timeFrame: Baseline, up to 172 weeks
measure: 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
timeFrame: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
measure: Change of Percentage of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5
timeFrame: Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
measure: Change in Growth Velocity
timeFrame: Baseline, up to 172 weeks
measure: Change From Pre-treatment Period in Mean Daily Diary Scores For Participants With CANDLE
timeFrame: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
measure: Change From Pre-treatment Period in Mean Daily Diary Scores
timeFrame: Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
measure: Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores For Participants With CANDLE
timeFrame: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks
measure: Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores
timeFrame: Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks
Other outcomes:
Locations:
city: Kashihara
country: Japan
facility: Nara Medical University Hospital
state: Nara
city: Bunkyō
country: Japan
facility: Tokyo Medical And Dental University Medical Hospital
state: Tokyo
city: Setagaya-ku
country: Japan
facility: National Center For Child Health And Development
state: Tokyo
city: Hiroshima
country: Japan
facility: Hiroshima University Hospital
city: Wakayama
country: Japan
facility: Wakayama Medical University Hospital
Study officials:
affiliation: Eli Lilly and Company
name: 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)
role: STUDY_DIRECTOR
References: