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

NCT04517253 | Terminated Phase 2 Results FDA Drug

• 3 other identifiers: 17571I4V-JE-JAJE2025-000001-16
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 5

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).

Conditions

Nakajo-Nishimura Syndrome; Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome; STING-Associated Vasculopathy With Onset in Infancy; Aicardi Goutieres Syndrome

Keywords

Nakajo-Nishimura Syndrome; CANDLE; SAVI; AGS

Outcome Measures

Primary Outcomes (3)

• Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period)

  Diaries were specific to individual indications or conditions (i.e., NNS/CANDLE, SAVI, or AGS). For NNS/CANDLE, participant or caregiver was instructed to rate each symptom (fever, rash, musculoskeletal pain, headache and fatigue in the diary on a scale from 0 to 4 (where a score of 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms, 3 = more severe symptoms, and 4 = severe symptoms \[equivalent to "worst" symptoms\]. The mean daily score range was 0-4 with the higher score indicating a more severe symptom. Total score was not utilized.

  Baseline, 20 weeks

• Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment Period)

  Diaries were specific to individual indications or conditions (i.e. NNS/CANDLE, SAVI, or AGS). For SAVI, participant or caregiver was instructed to rate each symptom (fever, rash, musculoskeletal pain, fatigue, respiratory/breathing problems, and ulcers/ischemic lesions in the diary on a scale from 0 to 4 (where a score of 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms,3 = more severe symptoms, and 4 = severe symptoms \[equivalent to "worst" symptoms\].The mean daily score range was 0-4 with the higher score indicating a more severe symptom. Total score was not utilized.

  Baseline, 32 weeks

• Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment Period)

  Diaries were specific to individual indications or conditions (i.e. NNS/CANDLE, SAVI, or AGS). For AGS, participant or caregiver was instructed to rate each symptom (rating) (neurologic disability (0, 5, 7,10) crying (0, 1, 2, 3), length of uninterrupted sleep (0, 1, 2, 3), generalized seizure (0, 8), fever (0,1), excessive irritability (0, 1, 2, 3), skin findings(body) (0, 1, 2, 3), and skin findings (hands, feet, and ears) (0, 1, 2, 3) with a higher score for each symptom indicating a more severe symptom. The mean daily diary score was the average of all symptom scores and the range was 0 - 4.25 with the higher score indicating a more severe symptom. Total score was not utilized.

  Baseline, 32 weeks

Secondary Outcomes (22)

• Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment and Maintenance Period)

  Baseline, 191.1 weeks

• Change From Baseline in Mean Daily Diary Scores in Participants With SAVI (Primary Treatment and Maintenance Period)

  Baseline, 202.9 weeks

• Change From Baseline in Mean Daily Diary Scores in Participants With AGS (Primary Treatment and Maintenance Period)

  Baseline, 206.1 weeks

• Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment Period)

  CANDLE: Week 20, SAVI and AGS: Week 32

• Number of Participants With Decrease in Daily Dose of Corticosteroids in Participants With CANDLE, SAVI and AGS (Primary Treatment and Maintenance Period)

  CANDLE: Week 191.1; SAVI: 202.9 and AGS: Week 206.1

Study Arms (3)

CANDLE

EXPERIMENTAL

Participants with chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE) were administered an optimized final dosage of baricitinib, ranging from 8 mg to 12 mg daily (initially 8 mg, with gradual escalation to 10 mg and 12 mg), either as tablets or oral suspension, for 12 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and estimated glomerular filtration rate (eGFR). Participants then continued receiving baricitinib at their optimized dosage for 191.1 weeks.

Drug: Baricitinib

SAVI

EXPERIMENTAL

Participants with stimulator of interferon genes (STING)-associated vasculopathy with onset during infancy (SAVI) were administered an optimized final dosage of baricitinib, ranging from 6 mg to 12 mg daily (initially 6 mg, with gradual escalation to 8 mg, 10 mg, and 12 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 202.9 weeks.

Drug: Baricitinib

Aicardi-Goutières Syndrome (AGS)

EXPERIMENTAL

Participants with Aicardi-Goutières Syndrome (AGS) were administered an optimized final dosage of baricitinib, ranging from 6 mg to 8 mg daily (initially 6 mg, with gradual escalation to 8 mg), either as tablets or oral suspension, for 24 weeks. This dosage was determined during a prior 8-week dose-adjustment period, tailored to each participant's weight and eGFR. Participants then continued receiving baricitinib at their optimized dosage for 206.1 weeks.

Drug: Baricitinib

Interventions

Baricitinib DRUG

Administered orally

Also known as: LY3009104

Aicardi-Goutières Syndrome (AGS); CANDLE; SAVI

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Eli Lilly and Company: lead

Study Sites (5)

Nara Medical University Hospital

Kashihara, Nara, 634-0813, Japan

Location

Tokyo Medical And Dental University Medical Hospital

Bunkyō, Tokyo, 113-8519, Japan

Location

National Center For Child Health And Development

Setagaya-ku, Tokyo, 157-8535, Japan

Location

Hiroshima University Hospital

Hiroshima, 734-8551, Japan

Location

Wakayama Medical University Hospital

Wakayama, 641-0012, Japan

Location

Related Publications (1)

• Kanazawa N, Ishii T, Takita Y, Nishikawa A, Nishikomori R. Efficacy and safety of baricitinib in Japanese patients with autoinflammatory type I interferonopathies (NNS/CANDLE, SAVI, And AGS). Pediatr Rheumatol Online J. 2023 Apr 22;21(1):38. doi: 10.1186/s12969-023-00817-8.

  PMID: 37087470 DERIVED

Related Links

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

MeSH Terms

Conditions

Nakajo syndrome; Aicardi-Goutieres syndrome

Interventions

baricitinib

Limitations and Caveats

The study was terminated early due to efficacy/effectiveness reasons.

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Eli Lilly and Company

ClinicalTrials.gov@lilly.com

800-545-5979

Study Officials

• 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

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 17, 2020
• First Posted: August 18, 2020
• Study Start: October 27, 2020
• Primary Completion: April 4, 2023
• Study Completion: November 28, 2024
• Last Updated: July 10, 2025
• Results First Posted: December 21, 2022

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Data are available 6 months after the primary publication and approval of the indication studied in the US and European Union (EU), whichever is later. Data will be indefinitely available for requesting.
• Access Criteria: A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement.

Locations

JP (5)