drugset / Trial / NCT03304717
Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome
Phase 1/2
Withdrawn
Children's Hospital of Philadelphia
Emerson Resources · collabEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · collabGilead Sciences · collabNational Human Genome Research Institute (NHGRI) · collabNational Institutes of Health (NIH) · collab
RandomizedCrossoverTriple-blindTreatment
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.
Timeline
- Start
- 2025-12
- Primary completion
- 2029-12
- Completion
- 2029-12
Outcome
Outcome not reported
Stopped: “Results from other clinical trials on AGS established baricitinib as standard of care treatment. Thanks to the knowledge gained in the clinical treatment of AGS, a clinical trial around the effects of RTI in AGS is no longer relevant at this time.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Tenofovir | Small molecule | — | — |
| Subject | emtricitabine | Other / unclassified | — | — |