325. Hereditary autoinflammatory syndrome Clinical trials / Disease details
Clinical trials : 7 / Drugs : 16 - (DrugBank : 6) / Drug target genes : 2 - Drug target pathways : 35
| 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) | December 2022 | 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 | National Institutes of Health (NIH);Emerson Resources;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);National Human Genome Research Institute (NHGRI);Gilead Sciences | Not yet recruiting | 2 Years | 18 Years | All | 34 | Phase 1/Phase 2 | United States |
| 2 | NCT04731103 (ClinicalTrials.gov) | February 2022 | 14/7/2020 | Inhibition of Reverse Transcription in Type I Interferon Mediated Neuropathology | Inhibition of Reverse Transcription in Type I Interferon Mediated Neuropathology | Aicardi-Goutières Syndrome | Drug: Abacavir (ABC);Drug: Lamivudine (3TC);Drug: Abacavir (ABC)+Lamivudine (3TC)+Zidovudine (AZT) | University of Edinburgh | NHS Lothian;Medical Research Council | Not yet recruiting | 3 Months | 15 Years | All | 24 | Phase 2 | United Kingdom |
| 3 | 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 | Active, not recruiting | 6 Months | N/A | All | 5 | Phase 2/Phase 3 | Japan |
| 4 | 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 | 50 | Phase 2 | United States |
| 5 | 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 |
| 6 | NCT01724580 (ClinicalTrials.gov) | July 2007 | 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 | ||
| 7 | 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 |