DDrare: Database of Drug Development for Rare Diseases

Tenofovir (tdf) and emtricitabine (ftc) (DrugBank: Emtricitabine, Tenofovir)

5 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
2	筋萎縮性側索硬化症	0
25	進行性多巣性白質脳症	0
93	原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］	0
265	脂肪萎縮症	0
325	遺伝性自己炎症疾患	1

2. 筋萎縮性側索硬化症　［臨床試験数：508，薬物数：530（DrugBank：146），標的遺伝子数：170，標的パスウェイ数：221］

Searched query = "Amyotrophic lateral sclerosis", "ALS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 508 trial found

25. 進行性多巣性白質脳症　［臨床試験数：23，薬物数：33（DrugBank：20），標的遺伝子数：7，標的パスウェイ数：36］

Searched query = "Progressive multifocal leukoencephalopathy", "PML", "Leukoencephalopathy, progressive multifocal"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 23 trial found

93. 原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］　［臨床試験数：230，薬物数：215（DrugBank：51），標的遺伝子数：34，標的パスウェイ数：107］

Searched query = "Primary biliary cholangitis", "Primary biliary cirrhosis", "PBC"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 230 trial found

265. 脂肪萎縮症　［臨床試験数：109，薬物数：164（DrugBank：59），標的遺伝子数：26，標的パスウェイ数：94］

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 109 trial found

325. 遺伝性自己炎症疾患　［臨床試験数：6，薬物数：10（DrugBank：6），標的遺伝子数：2，標的パスウェイ数：33］

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.

1 / 6 trial found

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