Trial · NCT03304717
Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome
NCT03304717, Phase 1, Phase 2, Withdrawn, Children's Hospital of Philadelphia
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome. Trials, record NCT03304717. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03304717
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome
- NCT number
- NCT03304717
- Status
- Withdrawn
- Phases
- Phase 1, Phase 2
- Enrollment
- 0 participants, actual
- Lead sponsor
- Children's Hospital of Philadelphia
- Conditions
- Aicardi Goutières Syndrome
- Interventions
- Tenofovir (TDF) and Emtricitabine (FTC), Placebo
- First posted
- 2017-10-09
- Last updated
- 2025-05-01
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in interferon activation as measured by interferon response genes | From Baseline to 13 months | The investigators propose to measure genes in the IFN stimulatory pathway in AGS patients, as a measure of disease activity and as a possible biomarker of therapeutic activity. Current data suggests that IFN related genes remain elevated in the late teens in AGS affected subjects, making it a more reliable measure of disease activity than IFN alpha. Validation of this preliminary data includes serial measurements in blood across several time points, to assess for variability. |
| Secondary | Determination of immune cell composition in CSF | From Baseline to 13 months | The investigators will pursue immunophenotyping of CSF cells in AGS subjects. Immunophenotyping and target antigens will further understanding of end organ damage in AGS. Additionally, this may provide biomarkers for therapeutic outcome and disease activity. |
| Secondary | Determination of immune cell composition in blood | From Baseline to 13 months | The investigators will pursue immunophenotyping of peripheral blood monocytes in AGS participants. Immunophenotyping and target antigens will further our understanding of end organ damage in AGS. Additionally, this may provide biomarkers for therapeutic outcome and disease activity. |
| Secondary | Accumulation of endogenous retroelements as measured in circulating immune cells | From Baseline to 13 months | Performed by assays from previously collected samples |
| Secondary | Accumulation of endogenous retroelements as measured in circulating CSF | From Baseline to 13 months | Performed by assays from previously collected samples |
| Secondary | Change in presence of non-specific and specific autoantibodies in blood | From Baseline to 13 months | Performed by assays from previously collected samples |
| Secondary | Changes in Adverse Events - Safety monitoring laboratory tests | From Baseline to 13 months and as clinically warranted | Patient monitoring for adverse effects |
| Secondary | Changes in total days hospitalized for disease-related illnesses. | Baseline - 13 months | Assess the effects of the treatment |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U01HD082806 | Reverse Transcriptase Inhibitors in Aicardi Goutieres Syndrome | VANDERVER, ADELINE LUCIE | CHILDREN'S HOSP OF PHILADELPHIA | EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT | 2015 to 2018 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03304717
GET https://conductscience.org/api/trials/v1/NCT03304717/registered