Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome

PHASE1/PHASE2WithdrawnINTERVENTIONAL
0
Timeline

Start Date

December 31, 2025

Primary Completion Date

December 31, 2029

Study Completion Date

December 31, 2029

Conditions
Aicardi Goutières Syndrome
Interventions
DRUG

Tenofovir (TDF) and Emtricitabine (FTC)

"Tenofovir (TDF): a nucleotide reverse transcriptase inhibitor (NtRTI) an acyclic nucleotide analog of adenosine 5'-monophosphate. This is used in children as young as age 2.~Emtricitabine (FTC): a nucleoside reverse transcriptase inhibitor (NRTI), a synthetic analog of cytidine which binds at the active site of the reverse transcriptase."

OTHER

Placebo

Placebo for Tenofovir and Placebo for Emtricitabine

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

collaborator

National Human Genome Research Institute (NHGRI)

NIH

collaborator

Gilead Sciences

INDUSTRY

collaborator

Emerson Resources

UNKNOWN

collaborator

National Institutes of Health (NIH)

NIH

lead

Children's Hospital of Philadelphia

OTHER

NCT03304717 - Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome | Biotech Hunter | Biotech Hunter