Reverse Transcriptase Inhibitors for Aicardi-Goutières Syndrome
(RTI in AGS Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety and effectiveness of two medications, Emtricitabine (FTC) and Tenofovir (TDF), for children with Aicardi-Goutières Syndrome (AGS), a rare condition affecting the brain and immune system. The study involves children with confirmed AGS who show signs of immune system activation. Participants will either start with the medication and switch to a placebo (inactive pill) or start with a placebo and switch to the medication. Children diagnosed with AGS and experiencing related symptoms might be suitable candidates for this trial. As a Phase 1, Phase 2 trial, this research seeks to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking research.
Will I have to stop taking my current medications?
The trial requires participants to avoid starting new immune-modulating therapies, like corticosteroids, but it doesn't specify if you need to stop other current medications. It's best to discuss your specific medications with the trial team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that Tenofovir (TDF) and Emtricitabine (FTC) are generally well-tolerated. These drugs often treat HIV and have received FDA approval for this purpose. Most people using them for HIV experience no serious side effects, though some may feel nauseous, have diarrhea, or get headaches.
In past studies with similar treatments, side effects were mostly mild to moderate. Although specific data for Aicardi-Goutières Syndrome (AGS) is not yet available, the safety record from other uses suggests these treatments are fairly safe. Since everyone can react differently, discussing any concerns with a doctor is important.12345Why do researchers think this study treatment might be promising for Aicardi-Goutières Syndrome?
Researchers are excited about using reverse transcriptase inhibitors like Emtricitabine (FTC) and Tenofovir (TDF) for Aicardi-Goutières Syndrome because these drugs target the underlying viral-like activity in the condition. Unlike current treatments, which primarily focus on managing symptoms, these drugs aim to reduce the abnormal interferon signaling that contributes to the disease. By addressing this root cause, FTC and TDF have the potential to modify the disease process itself, offering a new approach that could significantly improve patient outcomes.
What evidence suggests that this trial's treatments could be effective for Aicardi-Goutières Syndrome?
Research suggests that certain medications, such as Tenofovir (TDF) and Emtricitabine (FTC), might help treat Aicardi-Goutières Syndrome (AGS). This trial will evaluate the effects of TDF and FTC, known to block virus growth and potentially reduce harmful immune responses in AGS. Participants in one arm of the trial will receive TDF/FTC for the first 6 months, followed by a placebo, while those in the other arm will start with a placebo and then receive TDF/FTC. Studies have shown these medications work well in other diseases with similar immune problems. Early results indicate possible benefits, but more specific research on AGS remains necessary.16789
Who Is on the Research Team?
Adeline Vanderver, MD
Principal Investigator
Children's Hospital of Philadelphia
William Gahl, MD. PhD
Principal Investigator
National Institute of Health Genome Research Institute
Are You a Good Fit for This Trial?
Children aged 2-18 with Aicardi Goutières Syndrome (AGS) can join this trial. They must weigh at least 10 kg, be able to take oral medication or through a tube, and not start new immune therapies during the trial. Participants need negative HIV and Hepatitis B tests, no severe illnesses as judged by the investigator, and must be part of another specific study (MDBP).Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive TDF/FTC or placebo for 6 months, followed by a 1-month washout period, then crossover to the alternate treatment for another 6 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Emtricitabine (FTC)
- Placebo
- Tenofovir (TDF)
Trial Overview
The trial is testing if Tenofovir (TDF) and Emtricitabine (FTC), which are Reverse Transcriptase Inhibitors, are safe and effective in treating AGS compared to a placebo. Children will receive either the medications or placebo while their health outcomes are monitored.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
This is a double-blind, placebo-controlled, 2 arm, cross-over trial involving 34 children with clinical findings and molecular confirmation of Aicardi Goutieres Syndrome, who also have an abnormal interferon signature. For arm 1, half of the patients will receive TDF/FTC (a combination of Tenofovir \[TDF\] and Emtricitabine \[FTC\]) for the first 6 months of the study. There will be a one month washout period before starting on placebo for 6 months.
For arm 2, half of the patients will receive placebo for the first 6 months of the study. There will be a one month washout period before starting on TDF/FTC (a combination of Tenofovir \[TDF\] and Emtricitabine \[FTC\]) for 6 months.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital of Philadelphia
Lead Sponsor
Emerson Resources
Collaborator
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborator
National Human Genome Research Institute (NHGRI)
Collaborator
Gilead Sciences
Industry Sponsor
Daniel O'Day
Gilead Sciences
Chief Executive Officer since 2019
MBA from Columbia University
Dietmar Berger
Gilead Sciences
Chief Medical Officer
MD and PhD from Albert-Ludwigs University School of Medicine
National Institutes of Health (NIH)
Collaborator
Citations
NCT03304717 | Reverse Transcriptase Inhibitors in Aicardi ...
The overall objectives are to explore the safety and efficacy of Reverse Transcriptase Inhibitors Tenofovir (TDF)/ Emtricitabine (FTC) administered in AGS ...
Trial | NCT03304717
This is a double-blind, placebo-controlled, 2 arm, cross-over trial involving 34 children with clinical findings and molecular confirmation of Aicardi Goutieres ...
HIV and the IFN Signaling System
The Study of Aicardi-Goutières Syndrome Links LINE-1 to Autoimmunity. To answer the above question, we first need to know the nature of ...
Aicardi-Goutieres Syndrome (AGS)
Aicardi-Goutieres syndrome is an inherited disorder affecting the brain, immune system, and skin. It leads to severe brain dysfunction, intellectual and ...
Long-term safety and efficacy of emtricitabine and tenofovir ...
Overall, declines in bone mineral density occurred in participants who received emtricitabine and tenofovir disoproxil fumarate, which were significantly ...
Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome
The overall objectives are to explore the safety and efficacy of Reverse Transcriptase Inhibitors Tenofovir (TDF)/ Emtricitabine (FTC) ...
Trial | NCT03304717
This is a double-blind, placebo-controlled, 2 arm, cross-over trial involving 34 children with clinical findings and molecular confirmation of Aicardi Goutieres ...
Reverse Transcriptase Inhibitors for Aicardi-Goutières ...
... Emtricitabine (FTC) and Tenofovir (TDF) will have tolerable side effects & efficacy for patients with Nervous System Disorders, Autoimmune Neurological ...
Emtricitabine - Drug Targets, Indications, Patents
CONCLUSIONS: There was a significant reduction in eGFR among people with HIV who switched to TLD compared to those switched to DTG + 3TC. Changes in LDL and BMI ...
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