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Reverse Transcriptase Inhibitor

Reverse Transcriptase Inhibitors for Aicardi-Goutières Syndrome (RTI in AGS Trial)

Phase 1 & 2
Waitlist Available
Led By Adeline Vanderver, MD
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ages 2-18 years
Willingness to abstain from initiating the use of immune modulating therapies including corticosteroids
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline - 13 months
Awards & highlights

RTI in AGS Trial Summary

This trial is testing a new HIV treatment for children 2-18 years old.

Who is the study for?
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).Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Possible side effects of TDF/FTC may include digestive issues like nausea or vomiting, headaches, dizziness, fatigue, skin rashes or allergic reactions. Long-term use might affect bone density or kidney function.

RTI in AGS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 2 and 18 years old.
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I am willing to avoid starting any immune therapies or steroids.
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I weigh at least 10 kg.

RTI in AGS Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline - 13 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline - 13 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in interferon activation as measured by interferon response genes
Secondary outcome measures
Accumulation of endogenous retroelements as measured in circulating CSF
Endogenous depression
Change in presence of non-specific and specific autoantibodies in blood
+4 more

RTI in AGS Trial Design

2Treatment groups
Experimental Treatment
Group I: TDF/FTC then PlaceboExperimental Treatment2 Interventions
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.
Group II: Placebo then TDF/FTCExperimental Treatment2 Interventions
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 Location

Who is running the clinical trial?

Emerson ResourcesUNKNOWN
National Institutes of Health (NIH)NIH
2,679 Previous Clinical Trials
6,910,562 Total Patients Enrolled
Gilead SciencesIndustry Sponsor
1,077 Previous Clinical Trials
837,495 Total Patients Enrolled

Media Library

Emtricitabine (FTC) (Reverse Transcriptase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03304717 — Phase 1 & 2
Aicardi-Goutières Syndrome Research Study Groups: TDF/FTC then Placebo, Placebo then TDF/FTC
Aicardi-Goutières Syndrome Clinical Trial 2023: Emtricitabine (FTC) Highlights & Side Effects. Trial Name: NCT03304717 — Phase 1 & 2
Emtricitabine (FTC) (Reverse Transcriptase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03304717 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants is this research endeavor accommodating?

"This particular trial has already ceased to accept new participants. It was first listed on December 1st 2022 and the last update was made eight days into February of 2022. If you are seeking other clinical trials, there are currently 1389 studies engaged in recruiting patients with immune disorders, as well as 93 related to Tenofovir (TDF) and Emtricitabine (FTC)."

Answered by AI

Are there any records of past experiments related to the combination of Tenofovir (TDF) and Emtricitabine (FTC)?

"Presently, 93 clinical trials are underway around the world to study Tenofovir (TDF) and Emtricitabine (FTC), with 29 in Phase 3. Many of these experiments are being conducted from Boylston, Massachusetts; however, 1558 locations across the globe have begun their own studies on this medication combination."

Answered by AI

Am I a suitable candidate to participate in this trial?

"This medical trial is searching for 34 participants ages 2 to 18 that present with an immunological disorder and neurological impairment. To be eligible, patients must demonstrate their willingness to participate in lumbar punctures and blood draws; abstain from immune-modulating drugs such as corticosteroids; show molecular, neuroimaging, and clinical signs of AGS (with the exception of ADAR1 or IFIH1); exhibit interferon activation based on CSF neopterin/tetrahydrobiopterin levels upon initial evaluation; weigh at least 10kgs; have no concomitant illnesses preventing safe participation"

Answered by AI

Does this experiment allow for recruitment of senior citizens?

"To qualify for this trial, participants must be aged between 2 and 18. The clinicaltrials database hosts 392 trials targeting patients of that age range as well as 1052 studies focusing on individuals over 65 years old."

Answered by AI

Are any new participants being sought for this research project?

"This study, which was initially posted on December 1st 2022 and last amended on February 8th 2022 is not currently enrolling participants. However, there are 1482 other trials actively recruiting subjects at this time."

Answered by AI

To what ailments are Tenofovir (TDF) and Emtricitabine (FTC) typically prescribed?

"Tenofovir (TDF) and Emtricitabine (FTC) are frequently used to combat the effects of hiv-1. Moreover, they can be prescribed in cases of human immunodeficiency virus transmission or infection."

Answered by AI
~23 spots leftby Dec 2028