MedTrace Logo

Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome

NCT03304717 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2025-05-01

No results posted yet for this study

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.

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

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

    collaborator NIH

  • National Human Genome Research Institute (NHGRI)

    collaborator NIH

  • Gilead Sciences

    collaborator INDUSTRY

  • Emerson Resources

    collaborator UNKNOWN

  • National Institutes of Health (NIH)

    collaborator NIH

  • Children's Hospital of Philadelphia

    lead OTHER

Principal Investigators

  • Adeline Vanderver, MD · Children's Hospital of Philadelphia

  • William Gahl, MD. PhD · National Institute of Health Genome Research Institute

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-12-31
Primary Completion
2029-12-31
Completion
2029-12-31
FDA Drug
Yes

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03304717 on ClinicalTrials.gov