325. 遺伝性自己炎症疾患 ［臨床試験数：5，薬物数：9（DrugBank：6），標的遺伝子数：2，標的パスウェイ数：32］
Searched query = "Hereditary autoinflammatory syndrome", "NLRC4 abnormality", "Adenosine deaminase 2 deficiency", "ADA2 deficiency", "Aicardi Goutieres syndrome", "A20 haploinsufficiency"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03921554 | June 3, 2019 | 30 September 2019 | JAK Inhibitor Treatment in AGS | Janus Kinase Inhibitor (Baricitinib) for Aicardi Goutières Syndrome | Aicardi Goutieres Syndrome | Drug: Baricitinib | Adeline Vanderver, MD | Eli Lilly and Company | Recruiting | 6 Months | N/A | All | 100 | Phase 2 | United States |
| 2 | NCT03304717 | September 5, 2017 | 13 May 2019 | Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome | Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome | Aicardi Goutières Syndrome | Drug: Tenofovir (TDF) and Emtricitabine (FTC);Other: Placebo | Children's Hospital of Philadelphia | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);National Human Genome Research Institute (NHGRI);Gilead Sciences;Emerson Resources | Not recruiting | 2 Years | 18 Years | All | 34 | Phase 1/Phase 2 | United States |
| 3 | NCT02363452 | September 10, 2015 | 25 March 2019 | Reverse Transcriptase Inhibitors in AGS | A Pilot Clinical Trial of Reverse Transcriptase Inhibitors in Children With Aicardi-Goutières Syndrome (AGS) | Aicardi-Goutières Syndrome (AGS) | Drug: Reverse transcriptase inhibitors: Zidovudine, Lamivudine, Abacavir | Assistance Publique - Hôpitaux de Paris | Not recruiting | 1 Month | 17 Years | All | 11 | Phase 2 | France | |
| 4 | EUCTR2015-003424-31-GB | 28 February 2019 | Treatment of autoinflammatory diseases | Treatment of Conditions Expected to Benefit from JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis - JAGA | CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis, Aicardi-Goutieres Syndrome;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Baricitinib Product Code: LY3009104 Pharmaceutical Form: Coated tablet INN or Proposed INN: Baricitinib Current Sponsor code: LY3009104 Other descriptive name: BARICITINIB Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1- INN or Proposed INN: Baricitinib Current Sponsor code: LY3009104 Other descriptive name: BARICITINIB Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 4- INN or Proposed INN: baricitinib Current Sponsor code: LY3009104 Other descriptive name: BARICITINIB Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2- | Eli Lilly and Company | Not Available | Female: yes Male: yes | 60 | Phase 2 | France;United States;United Kingdom | ||||
| 5 | NCT01724580 | October 25, 2012 | 17 September 2018 | Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes | Compassionate Use Treatment Protocol I4V-MC-JAGA: Treatment of Conditions Expected to Benefit From JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI and Severe Juvenile Dermatomyositis | Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE);Juvenile Dermatomyositis (JDM);Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI);Aicardi-Goutières Syndrome (AGS) | Drug: Baricitinib | Eli Lilly and Company | Not recruiting | 6 Months | N/A | All | Phase 1 | United States;United Kingdom |