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

Sponsor
Eli Lilly and Company
Study ID
NCT04517253
Phase
PHASE2/PHASE3
Status
Terminated

Conditions

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

Eligibility Criteria

Sex
ALL
Age
6 Months - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

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).

Key Dates

Start date
Oct 27, 2020
Status verified
Jun 2025
Primary completion
Apr 4, 2023
Completion
Nov 28, 2024

Study Design

Enrollment
10 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: 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.
  • Experimental: 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.
  • Experimental: 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.

Primary Outcome Measure

Change From Baseline in Mean Daily Diary Scores in Participants With CANDLE (Primary Treatment Period) [ Time Frame: Baseline, 20 weeks ]

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