Nakajo-Nishimura Syndrome, Chronic Atypical Neutrophilic Dermatosis with Lipodystrophy and Elevated Temperature Syndrome, STING-Associated Vasculopathy with Onset in Infancy, Aicardi Goutieres Syndrome MedDRA version: 20.0 Level: PT Classification code 10073960 Term: CANDLE syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.1 Level: PT Classification code 10047115 Term: Vasculitis System Organ Class: 10047065 - Vascular disorders MedDRA ver
Conditions
Sponsors
Eli Lilly and Company
Eligibility
Sex/Gender
All
Inclusion 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 Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 5 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine if the administration of baricitinib to patients with NNS/CANDLE, SAVI, or AGS results in reduction in the patient’s mean daily diary scores compared to baseline.;Secondary Objective: The secondary objectives of this trial are: -To determine if administration of baricitinib results in the reduction in the daily dose of corticosteroids -To determine if administration of baricitinib results in improvement with clinical measurements. -To assess the growth of pediatric participants treated with baricitinib;Primary end point(s): -Change From Baseline in Mean Daily Diary Scores in Participants With SAVI -Change From Baseline in Mean Daily Diary Scores in Participants With AGS -Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE;Timepoint(s) of evaluation of this end point: -Baseline, up to 32 weeks -Baseline, up to 32 weeks -Baseline, up to 20 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Change From Baseline in Mean Daily Diary Scores -Number of Participants with Decrease in Daily Dose of Corticosteroids in Participants with SAVI and AGS -Number of Participants with Decrease in Daily Dose of Corticosteroids in Participants With CANDLE -Number of Participants with Decrease in Daily Dose of Corticosteroids -Change From Baseline in Patient's Symptom Specific Daily Diary Scores for Participants With SAVI -Change From Baseline in Patient's Symptom Specific Daily Diary Scores for Participants With AGS -Change From Baseline in Patient's Symptom Specific Daily Diary Scores for Participants With CANDLE -Change From Baseline in Patient's Symptom Specific Daily Diary Scores -Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants with SAVI and AGS -Change From Baseline in the Physician's Global Assessment of Disease Activity Scores in Participants With CANDLE -Change From Baseline in the Physician's Global Assessment of Disease Activity Scores -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 -Change in Growth Velocity -Change From Pre-treatment Period in Mean Daily Diary Scores for Participants With CANDLE -Change From Pre-treatment Period in Mean Daily Diary Scores -Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores for Participants With CANDLE -Change From Pre-treatment Period in the Physician's Global Assessment of Disease Activity Scores;Timepoint(s) of evaluation of this end point: -Baseline, up to 172 weeks -Week 32 -Week 20 -Baseline, up to 172 weeks -Baseline, up to 32 weeks -Baseline, up to 32 weeks -Baseline, up to 20 weeks -Baseline, up to 172 weeks -Baseline, up to 32 weeks -Baseline, up to 20 weeks -Baseline, up to 172 weeks | — |
Countries
Japan
Contacts
Public ContactClinical Trial Registry Office
Eli Lilly
Outcome results
None listed