Trial · NCT03921554
JAK Inhibitor Treatment in AGS
NCT03921554, Phase 2, Completed, Adeline Vanderver, MD; results first posted 2025-02-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. JAK Inhibitor Treatment in AGS. Trials, record NCT03921554. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03921554
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- JAK Inhibitor Treatment in AGS
- NCT number
- NCT03921554
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 54 participants, actual
- Lead sponsor
- Adeline Vanderver, MD
- Conditions
- Aicardi Goutieres Syndrome
- Interventions
- Baricitinib
- First posted
- 2019-04-19
- Last updated
- 2025-12-30
- Results first posted
- 2025-02-12
- Limitations and caveats
Skin manifestations as measured by the CLASI (Cutaneous Lupus Erythematosus Disease Area and Severity Index) is a secondary objective of this study. Completion of the CLASI was not consistently possible during the pandemic; video study visits made this evaluation infeasible. Analysis of the CLASI scores would not be meaningful. Moreover, 4 international subjects did not participate in video visits at 52 weeks, making the AGS severity scale infeasible in those 4 subjects for the primary outcome.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean and Standard Deviation (SD) of the AGS Scale at 52 Weeks | 52 weeks | The primary objective is to determine if the administration of baricitinib to participants with Aicardi-Goutières Syndrome (AGS) results in a change or stability of the AGS scale from baseline to 52 weeks. The AGS scale is a neurologic scale used to evaluate neurologic function of participants under treatment. The scale includes items for head circumference and developmental milestones. A lower score suggests a worse outcome. The range of scores is from 0 (most severe) to 11 (least severe). |
| Secondary | Improvement of the AGS Scale From Screening to Treatment Over Time | All longitudinal values are included. ASG scores were observed from 578 days (on some patients) before treatment to 2383 days after treatment start (on some patients). At visit 215 (with median treatment days = 673), the mean AGS score was 6.2 (SD = 3.5). | The AGS scale is a neurologic scale used to evaluate neurologic function of participants under treatment. The scale includes items for head circumference and developmental milestones. A lower score suggests a worse outcome. The range of scores is from 0 (most severe) to 11 (least severe). Longitudinal changes are evaluated, based on measurements collected at baseline, 1 month, 3 months, and every 3 months post-baseline for up to 288 weeks. Participants who transferred from the compassionate use study started treatment under the compassionate use study, which was initiated in 2016. AGS scores were assessed retrospectively for study visits completed under the compassionate use study, based on the study visit notes. The Outcome Measure Time Frame for these subjects may therefore be longer than the study duration specified in the Protocol Section of this study record, as the total time of treatment between the two studies they participated in is longer. |
| Secondary | Improvement of the GMFM-88 Between Screening and Treatment Over Time | 52 weeks | The Gross Motor Function Measure-88 (GMFM-88) assessment tool includes 88 items, each receiving a score from 0 to 3 (0 = does not initiate; 1 = initiates; 2 = partially completes; 3 = completes). Items span the spectrum of gross motor activities in five dimensions: A: Lying and Rolling (17 items), B: Sitting (20 items), C: Crawling and Kneeling (14 items), D: Standing (13 items), E: Walking, Running, Jumping (24 items). Every dimension score is expressed with a percentage. All dimension scores are then averaged to a total GMFM-88 percentage score, which is an estimate of the participant's gross motor function (0 = low motor function; 100 = high motor function). GMFM-88 is performed every 6 months. |
| Secondary | Change in Interferon Signaling Gene (ISG) Score Between Screening and Treatment Over Time | All longitudinal values are included. ISG scores were observed from 431 days before treatment to 2383 days after treatment start. At first on treatment visit (median treatment days = 3), the mean ISG score was 14.4 z-score (Standard Deviation = 12.8). | Interferon (IFN) scores are based on the messenger ribonucleic acid (mRNA) expression of six type I IFN signaling genes (ISG) and represent a surrogate marker for autoinflammation in AGS. Scores are derived from blood sampling, which occurs every three months. ISG scores are elevated in the AGS population and not elevated in healthy controls. The scale has not been published yet but it is hypothesized that a lower value in ISG score reflects a better outcome. Participants who transferred from the compassionate use study started treatment under the compassionate use study, which was initiated in 2016. ISG scores were measured from samples collected under the compassionate use study. The Outcome Measure Time Frame for these subjects may therefore be longer than the study duration specified in the Protocol Section of this study record, as the total time of treatment between the two studies they participated in is longer. |
| Secondary | Measurement of Disease Severity Assessed by Daily Diary Disease Severity Scale | All longitudinal values are included. Diary scores were observed from 117 days before treatment to 2476 days after treatment start. At visit 210 (median treatment days = 335), the mean diary average score was 1.2 (SD = 0.4). | The diary disease severity scale is a daily survey that caregivers complete based on the participant's clinical condition during the day and includes items such as uninterrupted sleep, irritability, or skin involvement. Each item is scored from 0 to 4 (0 = no symptoms; 1 = mild symptoms; 2 = moderate symptoms; 3 = more severe symptoms; 4 = severe symptoms). All item scores are then averaged to obtain a total diary score. Participants who transferred from the compassionate use study started treatment under the compassionate use study, which was initiated in 2016. Diaries were already completed and diary scores calculated under the compassionate use study. The Outcome Measure Time Frame for these subjects may therefore be longer than the study duration specified in the Protocol Section of this study record, as the total time of treatment between the two studies they participated in is longer. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean and Standard Deviation (SD) of the AGS Scale at 52 Weeks | 52 weeks | The overall number of baseline participants totals 51. However, challenges to score the AGS scale at 52 weeks were encountered during the pandemic. Some participants were located outside of the United States and their Visit 210 (52-week time point) was completed by phone and did not allow to gather enough information to complete the AGS severity scale. 45 patients had an AGS score at Visit 210. The mean below is at 52 weeks. | score on a scale | MEAN |
| SECONDARY | Improvement of the AGS Scale From Screening to Treatment Over Time | All longitudinal values are included. ASG scores were observed from 578 days (on some patients) before treatment to 2383 days after treatment start (on some patients). At visit 215 (with median treatment days = 673), the mean AGS score was 6.2 (SD = 3.5). | The Overall Number of Baseline Participants (with genetic confirmation) are included in this analysis. | score on a scale | MEAN |
| SECONDARY | Improvement of the GMFM-88 Between Screening and Treatment Over Time | 52 weeks | The overall number of baseline participants totals 51. Note one participant did not have a GMFM-88 completed at baseline. Additionally, challenges to score the GMFM-88 scale at 52 weeks were encountered during the pandemic since this assessment could only be completed in person at the time. 35 patients had a GMFM-88 score at Visit 210. The mean below is at 52 weeks. | score on a scale | MEAN |
| SECONDARY | Change in Interferon Signaling Gene (ISG) Score Between Screening and Treatment Over Time | All longitudinal values are included. ISG scores were observed from 431 days before treatment to 2383 days after treatment start. At first on treatment visit (median treatment days = 3), the mean ISG score was 14.4 z-score (Standard Deviation = 12.8). | The Overall Number of Baseline Participants (with genetic confirmation) are included in this analysis. | z-score | MEAN |
| SECONDARY | Measurement of Disease Severity Assessed by Daily Diary Disease Severity Scale | All longitudinal values are included. Diary scores were observed from 117 days before treatment to 2476 days after treatment start. At visit 210 (median treatment days = 335), the mean diary average score was 1.2 (SD = 0.4). | The Overall Number of Baseline Participants (with genetic confirmation) are included in this analysis. | score | MEAN |
Source ClinicalTrials.gov
Results by group7
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean and Standard Deviation (SD) of the AGS Scale at 52 Weeks | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 5.9 | Standard Deviation 3.6 | score on a scale | |
| Improvement of the AGS Scale From Screening to Treatment Over Time | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 6.2 | Standard Deviation 3.5 | score on a scale | |
| Improvement of the GMFM-88 Between Screening and Treatment Over Time | Cohort A: AGS Subjects With Baseline AGS Score 0-3 | 6.1 | Standard Deviation 6.6 | score on a scale | |
| Improvement of the GMFM-88 Between Screening and Treatment Over Time | Cohort B: AGS Subjects With Baseline AGS Score 4-8 | 30.0 | Standard Deviation 18.9 | score on a scale | |
| Improvement of the GMFM-88 Between Screening and Treatment Over Time | Cohort C: AGS Subjects With Baseline AGS Score 9-11 | 57.2 | Standard Deviation 26.9 | score on a scale | |
| Change in Interferon Signaling Gene (ISG) Score Between Screening and Treatment Over Time | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 14.4 | Standard Deviation 12.8 | z-score | |
| Measurement of Disease Severity Assessed by Daily Diary Disease Severity Scale | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 1.2 | Standard Deviation 0.4 | score |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 54 | |
| Overall Study | COMPLETED | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 48 | |
| Overall Study | NOT COMPLETED | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 6 |
Source ClinicalTrials.gov
Adverse events144
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anemia | Blood and lymphatic system disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 15 | 51 | 16 |
| other | Blood and lymphatic system disorders - Other, Thrombocytosis | Blood and lymphatic system disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 4 | 51 | 4 |
| other | Sinus tachycardia | Cardiac disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 4 | 51 | 4 |
| other | Vomiting | Gastrointestinal disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 22 | 51 | 39 |
| other | Diarrhea | Gastrointestinal disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 11 | 51 | 14 |
| other | Constipation | Gastrointestinal disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 11 | 51 | 12 |
| other | Mucositis oral | Gastrointestinal disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 3 | 51 | 8 |
| other | Gastroesophageal reflux disease | Gastrointestinal disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 4 | 51 | 5 |
| other | Gastrointestinal disorders - Other, Increased salivation | Gastrointestinal disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 4 | 51 | 5 |
| other | Abdominal pain | Gastrointestinal disorders | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 4 | 51 | 4 |
1 to 10 of 144Source ClinicalTrials.gov
See all 144 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 3 | 54 |
| Total, other adverse events | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 50 | 51 |
| Total, serious adverse events | Aicardi-Goutières Syndrome Patients Receiving Baricitinib | 33 | 51 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 35786528 | DERIVED |
Source PubMed
Grants0
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.
None in this release.
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/NCT03921554
GET https://conductscience.org/api/trials/v1/NCT03921554/events?page=2