Your session is about to expire
← Back to Search
Janus Kinase (JAK) Inhibitor
Baricitinib for AGS
Phase 2
Waitlist Available
Research Sponsored by Adeline Vanderver, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Females after menarche must have a negative urine/serum pregnancy test and must use an acceptable method of contraception
≥4.5 kg in body weight
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
Study Summary
This trial is testing a new drug to see if it is safe and effective for people with Aicardi-Goutieres Syndrome, a rare disorder that affects many parts of the body.
Who is the study for?
This trial is for patients with Aicardi Goutières Syndrome (AGS), showing neurological disease on scans, specific clinical features, and genetic mutations linked to AGS. Participants must be over 1 month old and weigh at least 4.5 kg. Women capable of pregnancy must test negative and use contraception. Exclusions include current/recent participation in other trials, certain medication use, TB or hepatitis infection, HIV positive status.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of Baricitinib, a JAK inhibitor drug designed to reduce excessive immune activity in AGS patients by blocking certain enzymes involved in inflammation.See study design
What are the potential side effects?
While not explicitly listed here, common side effects of JAK inhibitors like Baricitinib may include infections due to lowered immunity, blood clots, cholesterol changes, liver enzyme elevations among others.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am a woman of childbearing age and not pregnant, using birth control.
Select...
I weigh more than 4.5 kg.
Select...
I have been diagnosed with Aicardi Goutières Syndrome.
Select...
My genetic test shows a mutation linked to my condition.
Select...
My brain scans show signs of disease, like calcifications or white matter disease.
Select...
I am older than 1 month.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Measurement of change with AGS scale at baseline
Secondary outcome measures
Functional measures of neurologic disability
Gross Motor Function Measure-88 (GMFM-88)
Measurement of disease severity assessed by daily diary disease severity scale
+6 moreSide effects data
From 2015 Phase 3 trial • 1307 Patients • NCT0171035810%
Nasopharyngitis
4%
Upper respiratory tract infection
4%
Urinary tract infection
4%
Headache
4%
Pharyngitis
3%
Alanine aminotransferase increased
3%
Back pain
3%
Erectile dysfunction
3%
Nausea
3%
Hypertension
2%
Diarrhoea
2%
Cough
2%
Dyspepsia
2%
Bronchitis
2%
Influenza
2%
Rash
1%
Hypercholesterolaemia
1%
Hyperlipidaemia
1%
Rheumatoid arthritis
1%
Anaemia
1%
Blood creatine phosphokinase increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
Adalimumab Treatment A
Adalimumab Treatment B
Placebo Treatment B
Baricitinib Follow-up
Baricitinib Treatment A
Placebo Follow-up
BaricitinibTreatment B
Adalimumab Follow-up
Placebo Treatment A
Rescue
Trial Design
1Treatment groups
Experimental Treatment
Group I: Aicardi Goutières Syndrome patients receiving BaricitinibExperimental Treatment1 Intervention
Baricitinib will be taken by mouth or via gastrostomy feeding tube or nasogastric tube as directed by the study doctor. Baricitinib will be dosed by patient age, weight range and estimated glomerular filtration rate (eGFR). Dosing formulations in use in this study will include 1 mg and 2 mg tablets and will be used without splitting. Dispersion will be permitted to aid in swallowing.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Baricitinib
2017
Completed Phase 3
~9510
Find a Location
Who is running the clinical trial?
Eli Lilly and CompanyIndustry Sponsor
2,615 Previous Clinical Trials
3,201,003 Total Patients Enrolled
Adeline Vanderver, MDLead Sponsor
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your lab test results are not within the normal range for your age and location, and it's not due to a known condition.I haven't taken immune-weakening drugs recently.I am a woman of childbearing age and not pregnant, using birth control.I weigh more than 4.5 kg.I have been diagnosed with Aicardi Goutières Syndrome.I have tested positive for TB or have a history of TB.I have symptoms like small head size, muscle weakness, stiff muscles, skin issues, liver inflammation, or blood problems.I am willing and able to follow the study's requirements.My genetic test shows a mutation linked to my condition.My brain scans show signs of disease, like calcifications or white matter disease.You have tested positive for hepatitis B.I have tested positive for Hepatitis C.I am older than 1 month.You have HIV infection or test positive for HIV antibodies.I am not taking any medication that is not allowed in the study.
Research Study Groups:
This trial has the following groups:- Group 1: Aicardi Goutières Syndrome patients receiving Baricitinib
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Have any other investigations been conducted around Baricitinib?
"Currently, Baricitinib is being tested in 31 distinct studies, 15 of which are entering the final phase. While several trials take place within Pune, Maharashtra, a total of 1416 medical centres have been enlisted to conduct research on this drug."
Answered by AI
How many individuals are currently participating in this clinical experiment?
"This clinical trial is no longer open for recruitment. It was initially announced on June 3rd 2019 and the last update occurred on July 19th 2022. If you are looking to participate in other studies, there are 1389 trials actively enrolling patients with Aicardi-Goutieres Syndrome and 31 studies involving Baricitinib recruiting participants."
Answered by AI
To what end is Baricitinib usually prescribed?
"Commonly administered to hospitalized individuals, baricitinib can also be utilized for the management of conditions such as coronavirus disease 2019 (covid‑19), extracorporeal membrane oxygenation treatment, and rheumatoid arthritis."
Answered by AI
What deleterious effects can Baricitinib have on patients?
"Baricitinib has only been tested for safety in a Phase 2 trial, so it is assigned an estimated risk level of 2. There have yet to be any reported studies validating its efficacy."
Answered by AI
Are enrollment slots currently available for this research study?
"Per the clinicaltrials.gov database, this trial is not actively searching for enrollees at present. Originally posted on June 3rd 2019 with a last update of July 19th 2022, it currently has no recruitment requirements; however, there are over 1400 other trials looking to take in participants as we speak."
Answered by AI
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger