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 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:3] 

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. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03921554June 3, 201930 September 2019JAK Inhibitor Treatment in AGSJanus Kinase Inhibitor (Baricitinib) for Aicardi Goutières SyndromeAicardi Goutieres SyndromeDrug: BaricitinibAdeline Vanderver, MDEli Lilly and CompanyRecruiting6 MonthsN/AAll100Phase 2United States
2NCT03304717September 5, 201713 May 2019Reverse Transcriptase Inhibitors in Aicardi Goutières SyndromeReverse Transcriptase Inhibitors in Aicardi Goutières SyndromeAicardi Goutières SyndromeDrug: Tenofovir (TDF) and Emtricitabine (FTC);Other: PlaceboChildren's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);National Human Genome Research Institute (NHGRI);Gilead Sciences;Emerson ResourcesNot recruiting2 Years18 YearsAll34Phase 1/Phase 2United States
3NCT02363452September 10, 201525 March 2019Reverse Transcriptase Inhibitors in AGSA Pilot Clinical Trial of Reverse Transcriptase Inhibitors in Children With Aicardi-Goutières Syndrome (AGS)Aicardi-Goutières Syndrome (AGS)Drug: Reverse transcriptase inhibitors: Zidovudine, Lamivudine, AbacavirAssistance Publique - Hôpitaux de ParisNot recruiting1 Month17 YearsAll11Phase 2France
4EUCTR2015-003424-31-GB28 February 2019Treatment of autoinflammatory diseasesTreatment of Conditions Expected to Benefit from JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis - JAGACANDLE, 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 CompanyNot Available Female: yes
Male: yes
60Phase 2France;United States;United Kingdom
5NCT01724580October 25, 201217 September 2018Compassionate Use Protocol for the Treatment of Autoinflammatory SyndromesCompassionate 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 DermatomyositisChronic 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: BaricitinibEli Lilly and CompanyNot recruiting6 MonthsN/AAllPhase 1United States;United Kingdom

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