E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Nakajo-Nishimura Syndrome, Chronic Atypical Neutrophilic Dermatosis with Lipodystrophy and Elevated Temperature Syndrome, STING-Associated Vasculopathy with Onset in Infancy, Aicardi Goutieres Syndrome |
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E.1.1.1 | Medical condition in easily understood language |
Autoinflammatory diseases |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10073960 |
E.1.2 | Term | CANDLE syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10047115 |
E.1.2 | Term | Vasculitis |
E.1.2 | System Organ Class | 10047065 - Vascular disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 27.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10054935 |
E.1.2 | Term | Aicardi's syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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. |
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E.2.2 | Secondary objectives of the trial |
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 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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 |
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E.4 | Principal 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. |
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E.5 End points |
E.5.1 | 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 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
-Baseline, up to 32 weeks -Baseline, up to 32 weeks -Baseline, up to 20 weeks |
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E.5.2 | 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 |
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E.5.2.1 | 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 -Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks -Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks -Baseline, up to 172 weeks -Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks -Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks -Pre-treatment period (average of 12-week pre-treatment data), up to 20 weeks -Pre-treatment period (average of 12-week pre-treatment data), up to 172 weeks |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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End of the study is the date of the last visit or last scheduled procedure for the last participant. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 4 |