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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03304717 (ClinicalTrials.gov) | January 2021 | 4/8/2017 | 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;National Institutes of Health (NIH) | Not yet recruiting | 2 Years | 18 Years | All | 34 | Phase 1;Phase 2 | United States |
| 2 | NCT04517253 (ClinicalTrials.gov) | October 27, 2020 | 17/8/2020 | A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS | A 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 AGS | Nakajo-Nishimura Syndrome;Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome;STING-Associated Vasculopathy With Onset in Infancy;Aicardi Goutieres Syndrome | Drug: Baricitinib | Eli Lilly and Company | NULL | Recruiting | 6 Months | N/A | All | 5 | Phase 2;Phase 3 | Japan |
| 3 | NCT03921554 (ClinicalTrials.gov) | June 3, 2019 | 3/4/2019 | 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 | Active, not recruiting | 1 Month | N/A | All | 100 | Phase 2 | United States |
| 4 | NCT02363452 (ClinicalTrials.gov) | September 10, 2015 | 15/1/2015 | 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 | NULL | Completed | 1 Month | 17 Years | All | 11 | Phase 2 | France |
| 5 | NCT01724580 (ClinicalTrials.gov) | June 2009 | 5/11/2012 | 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 | NULL | No longer available | 6 Months | N/A | All | United States;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2015-003424-31-GB (EUCTR) | 07/12/2015 | 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 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 Company | NULL | NA | Female: yes Male: yes | 60 | Phase 2 | France;United States;United Kingdom |