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

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-10-27

Study Completion Date

2024-11-28

Brief Summary

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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).

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Detailed Description

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Conditions

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

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CANDLE

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.

Group Type EXPERIMENTAL

Baricitinib

Intervention Type DRUG

Administered orally

SAVI

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.

Group Type EXPERIMENTAL

Baricitinib

Intervention Type DRUG

Administered orally

Aicardi-Goutières Syndrome (AGS)

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.

Group Type EXPERIMENTAL

Baricitinib

Intervention Type DRUG

Administered orally

Interventions

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Baricitinib

Administered orally

Intervention Type DRUG

Other Intervention Names

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LY3009104

Eligibility Criteria

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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.

Minimum Eligible Age

6 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No