325. Hereditary autoinflammatory syndrome
6 clinical trials,   10 drugs   (DrugBank: 6 drugs),   2 drug target genes,   33 drug target pathways

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03304717
(ClinicalTrials.gov)
January 20214/8/2017Reverse 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 Resources;National Institutes of Health (NIH)Not yet recruiting2 Years18 YearsAll34Phase 1;Phase 2United States
2NCT04517253
(ClinicalTrials.gov)
October 27, 202017/8/2020A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGSA 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 AGSNakajo-Nishimura Syndrome;Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome;STING-Associated Vasculopathy With Onset in Infancy;Aicardi Goutieres SyndromeDrug: BaricitinibEli Lilly and CompanyNULLRecruiting6 MonthsN/AAll5Phase 2;Phase 3Japan
3NCT03921554
(ClinicalTrials.gov)
June 3, 20193/4/2019JAK Inhibitor Treatment in AGSJanus Kinase Inhibitor (Baricitinib) for Aicardi Goutières SyndromeAicardi Goutieres SyndromeDrug: BaricitinibAdeline Vanderver, MDEli Lilly and CompanyActive, not recruiting1 MonthN/AAll100Phase 2United States
4NCT02363452
(ClinicalTrials.gov)
September 10, 201515/1/2015Reverse 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 ParisNULLCompleted1 Month17 YearsAll11Phase 2France
5NCT01724580
(ClinicalTrials.gov)
June 20095/11/2012Compassionate 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 CompanyNULLNo longer available6 MonthsN/AAllUnited States;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2015-003424-31-GB
(EUCTR)
07/12/2015Treatment of autoinflammatory diseasesTreatment 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
INN or Proposed INN: Baricitinib
Other descriptive name: BARICITINIB
INN or Proposed INN: Baricitinib
Other descriptive name: BARICITINIB
INN or Proposed INN: baricitinib
Other descriptive name: BARICITINIB
Eli Lilly and CompanyNULLNAFemale: yes
Male: yes
60Phase 2France;United States;United Kingdom