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

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

Sponsors

Lead Sponsor: Eli Lilly and Company

Source Eli Lilly and Company
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).

Overall Status Recruiting
Start Date 2020-10-27
Completion Date 2023-09-15
Primary Completion Date 2021-10-12
Phase Phase 2/Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Change from Baseline in Mean Daily Diary Scores Baseline, up to 32 weeks
Secondary Outcome
Measure Time Frame
Number of participants with decrease in Daily Dose of Corticosteroids Baseline, up to 100 weeks
Change from Baseline in Mean Daily Diary Scores Baseline, up to 100 weeks
Change from Baseline in Patient's Symptom Specific Daily Diary Scores Baseline, up to 100 weeks
Change From Baseline in the Physician's Global Assessment of Disease Activity Scores Baseline, up to 100 weeks
Percentage Change of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 Pre-treatment period, up to 100 weeks
Change in Growth Velocity Baseline, up to 100 weeks
Change from Pre-treatment period in Mean Daily Diary Scores Pre-treatment period, up to 100 weeks
Change from Pre-treatment period in the Physician's Global Assessment of Disease Activity Scores Pre-treatment period, up to 100 weeks
Enrollment 5
Condition
Intervention

Intervention Type: Drug

Intervention Name: Baricitinib

Description: Administered orally

Arm Group Label: Baricitinib

Other Name: LY3009104

Eligibility

Criteria:

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.

Gender:

All

Minimum Age:

6 Months

Maximum Age:

N/A

Healthy Volunteers:

No

Overall Official
Last Name Role Affiliation
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) Study Director Eli Lilly and Company
Overall Contact

Last Name: There may be multiple sites in this clinical trial. 1-877-CTLILLY (1-877-285-4559) or

Phone: 1-317-615-4559

Email: [email protected]

Location
Facility: Status: Contact: Investigator:
Hiroshima University Hospital | Hiroshima-shi, Hiroshima-ken, 734-8551, Japan Recruiting 81120023812 Satoshi Okada Principal Investigator
Nara Medical University Hospital | Kashihara, Nara, 634-8522, Japan Recruiting 81120023812 Kazuma Sugie Principal Investigator
Tokyo Medical And Dental University Medical Hospital | Bunkyō, Tokyo, 113-8519, Japan Recruiting 81120023812 Tomohiro Udagawa Principal Investigator
National Center For Child Health And Development | Setagaya-ku, Tokyo, 157-8535, Japan Recruiting 81120023812 Toshinao Kawai Principal Investigator
Wakayama Medical University Hospital | Wakayama, 641-8510, Japan Recruiting 81120023812 Kayo Kunimoto Principal Investigator
Location Countries

Japan

Verification Date

2021-05-01

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Baricitinib

Type: Experimental

Description: Baricitinib administered orally either by tablet or suspension.

Patient Data Yes
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

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