Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome
Status:
Not yet recruiting
Trial end date:
2026-12-01
Target enrollment:
Participant gender:
Summary
The overall objectives are to explore the safety and efficacy of Reverse Transcriptase
Inhibitors Tenofovir (TDF)/ Emtricitabine (FTC) administered in AGS affected children 2 to 18
years of age.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Children's Hospital of Philadelphia
Collaborators:
Emerson Resources Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Gilead Sciences National Human Genome Research Institute (NHGRI) National Institutes of Health (NIH)