A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS

Sponsor

Eli Lilly and Company (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04517253

Collaborator

(none)

Enrollment

Locations

Arm

54.1

Anticipated Duration (Months)

1.6

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The main purpose of this study is to evaluate the efficacy and safety of baricitinib in adult and pediatric Japanese participants with Nakajo-Nishimura Syndrome/chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (NNS/CANDLE), STING-associated vasculopathy with onset during infancy (SAVI), and Aicardi-Goutières Syndrome (AGS).

Condition or Disease	Intervention/Treatment	Phase
Nakajo-Nishimura Syndrome Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome STING-Associated Vasculopathy With Onset in Infancy Aicardi Goutieres Syndrome	Drug: Baricitinib	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Actual Enrollment :

8 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients With NNS/CANDLE, SAVI, and AGS

Actual Study Start Date :

Oct 27, 2020

Actual Primary Completion Date :

Oct 12, 2021

Anticipated Study Completion Date :

Apr 30, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Baricitinib Baricitinib administered orally either by tablet or suspension.	Drug: Baricitinib Administered orally Other Names: LY3009104

Arm

Intervention/Treatment

Experimental: Baricitinib

Baricitinib administered orally either by tablet or suspension.

Drug: Baricitinib

Administered orally

Other Names:

LY3009104

Outcome Measures

Primary Outcome Measures

Change from Baseline in Mean Daily Diary Scores [Baseline, up to 32 weeks]
Change from Baseline in Mean Daily Diary Scores

Secondary Outcome Measures

Number of participants with decrease in Daily Dose of Corticosteroids [Baseline, up to 100 weeks]
Number of participants with decrease in Daily Dose of Corticosteroids
Change from Baseline in Mean Daily Diary Scores [Baseline, up to 100 weeks]
Change from Baseline in Mean Daily Diary Scores
Change from Baseline in Patient's Symptom Specific Daily Diary Scores [Baseline, up to 100 weeks]
Change from Baseline in Patient's Symptom Specific Daily Diary Scores
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores [Baseline, up to 100 weeks]
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores
Percentage Change of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 [Pre-treatment period, up to 100 weeks]
Percentage Change of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5
Change in Growth Velocity [Baseline, up to 100 weeks]
Change in Growth Velocity
Change from Pre-treatment period in Mean Daily Diary Scores [Pre-treatment period, up to 100 weeks]
Change from Pre-treatment period in Mean Daily Diary Scores
Change from Pre-treatment period in the Physician's Global Assessment of Disease Activity Scores [Pre-treatment period, up to 100 weeks]
Change from Pre-treatment period in the Physician's Global Assessment of Disease Activity Scores

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Months and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Have systemic signs and symptoms of inflammation as manifested NNS/CANDLE, SAVI, AGS
Have been diagnosed with genetic diagnosis
Men must agree to use a reliable method of birth control during the study
Women not of child-bearing potential or nonbreastfeeding
Women must agree to use birth control or remain abstinent during the study and for at least 12 weeks after stopping treatment
NNS/CANDLE and SAVI patients who are ≥17.5 months of age
AGS patients who are ≥6 months of age
Are ≥ 5kg in body weight

Exclusion Criteria:

Have received immunosuppressive biologic agent/monoclonal antibody/oral JAK inhibitor/OAT3 inhibitor and cannot discontinue prior to investigational product initiation. Note: A washout period is required to each drug.
Have diagnosis of current active tuberculosis (TB) or, latent TB who did not receive appropriate treatment.
Have had a serious infection within 12 weeks prior to screening.
Have a history of lymphoproliferative disease
Have any history of venous thromboembolic event (VTE) (deep vein thrombosis [DVT]/pulmonary embolism [PE]) prior to screening.
Have had any major surgery within 8 weeks prior to screening.
Have previously been enrolled in any other study investigating baricitinib.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hiroshima University Hospital	Hiroshima-shi	Hiroshima-ken	Japan	734-8551
2	Nara Medical University Hospital	Kashihara	Nara	Japan	634-8522
3	Tokyo Medical And Dental University Medical Hospital	Bunkyō	Tokyo	Japan	113-8519
4	National Center For Child Health And Development	Setagaya-ku	Tokyo	Japan	157-8535
5	Wakayama Medical University Hospital	Wakayama		Japan	641-8510

Sponsors and Collaborators

Eli Lilly and Company

Investigators

Study Director: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST), Eli Lilly and Company