DDrare: Database of Drug Development for Rare Diseases

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

5 diseases

ID	Disease name (Link within this page)	Number of trials
2	Amyotrophic lateral sclerosis	0
25	Progressive multifocal leukoencephalopathy	0
93	Primary biliary cholangitis [Primary biliary cirrhosis (~Mar 2017)]	0
265	Lipodystrophy	0
325	Hereditary autoinflammatory syndrome	1

2. Amyotrophic lateral sclerosis [ 508 clinical trials, 530 drugs, (DrugBank: 146 drugs), 170 drug target genes, 221 drug target pathways]

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. Progressive multifocal leukoencephalopathy [ 23 clinical trials, 33 drugs, (DrugBank: 20 drugs), 7 drug target genes, 36 drug target pathways]

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. Primary biliary cholangitis [Primary biliary cirrhosis (~Mar 2017)] [ 230 clinical trials, 215 drugs, (DrugBank: 51 drugs), 34 drug target genes, 107 drug target pathways]

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. Lipodystrophy [ 109 clinical trials, 164 drugs, (DrugBank: 59 drugs), 26 drug target genes, 94 drug target pathways]

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. 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.

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