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

Lilly

Status and phase: Terminated; Phase 3; Phase 2

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

Treatments: Drug: Baricitinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT04517253

I4V-JE-JAJE (Other Identifier)

17571

2025-000001-16 (EudraCT Number)

Details and patient eligibility

About

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).

Enrollment: 10 patients

Sex: All

Ages: 6+ months old

Volunteers: No 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 3 patient groups

CANDLE

Experimental group

Description:

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.

Treatment:

Drug: Baricitinib

SAVI

Experimental group

Description:

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.

Treatment:

Drug: Baricitinib

Aicardi-Goutières Syndrome (AGS)

Experimental group

Description:

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.

Treatment:

Drug: Baricitinib