A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS

• Register: ClinicalTrials.gov
• Last refreshed on: 5 August 2024
• Main ID: NCT04517253
• Date of registration: 17/08/2020
• Prospective Registration: Yes
• Primary sponsor: Eli Lilly and Company
• Public title: A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS
• Scientific 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
• Date of first enrolment: October 27, 2020
• Target sample size: 8
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04517253
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Japan

Contacts

• 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)
• Affiliation: Eli Lilly and Company

Key inclusion & exclusion 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.


Age minimum: 6 Months
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

STING-Associated Vasculopathy With Onset in Infancy

Aicardi Goutieres Syndrome

Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome

Nakajo-Nishimura Syndrome

Intervention(s)

Drug: Baricitinib

Primary Outcome(s)

Change From Baseline in Mean Daily Diary Scores in Participants With SAVI [Time Frame: Baseline, up to 32 weeks]

Change From Baseline in Mean Daily Diary Scores in Participants With AGS [Time Frame: Baseline, up to 32 weeks]

Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE [Time Frame: Baseline, up to 20 weeks]

Secondary Outcome(s)

Number of Participants With Decrease in Daily Dose of Corticosteroids [Time Frame: Baseline, up to 172 weeks]

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With AGS [Time Frame: 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]

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]

Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With SAVI and AGS [Time Frame: Week 32]

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With SAVI [Time Frame: Baseline, up to 32 weeks]

Change From Baseline in the Physician's Global Assessment of Disease Activity Scores [Time Frame: Baseline, 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]

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]

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 in Growth Velocity [Time Frame: Baseline, up to 172 weeks]

Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE [Time Frame: Week 20]

Change From Baseline in Mean Daily Diary Scores [Time Frame: Baseline, up to 172 weeks]

Change From Baseline in Patient's Symptom Specific Daily Diary Scores [Time Frame: 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]

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 For Participants With CANDLE [Time Frame: Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks]

Change From Baseline in Patient's Symptom Specific Daily Diary Scores For Participants With CANDLE [Time Frame: Baseline, up to 20 weeks]

Secondary ID(s)

I4V-JE-JAJE

17571

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 21/12/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04517253