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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04517253
Recruitment Status : Active, not recruiting
First Posted : August 18, 2020
Last Update Posted : August 19, 2022
Sponsor:
Information provided by (Responsible Party):
Eli Lilly and Company

Study Description
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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).

Condition or disease Intervention/treatment Phase
Nakajo-Nishimura Syndrome Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome STING-Associated Vasculopathy With Onset in Infancy Aicardi Goutieres Syndrome Drug: Baricitinib Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : October 27, 2020
Actual Primary Completion Date : October 12, 2021
Estimated Study Completion Date : April 30, 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Baricitinib

Arms and Interventions
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Arm Intervention/treatment
Experimental: Baricitinib
Baricitinib administered orally either by tablet or suspension.
 Drug: Baricitinib
Administered orally
Other Name: LY3009104


Outcome Measures
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Primary Outcome Measures :
  1. Change from Baseline in Mean Daily Diary Scores [ Time Frame: Baseline, up to 32 weeks ]
    Change from Baseline in Mean Daily Diary Scores


Secondary Outcome Measures :
  1. Number of participants with decrease in Daily Dose of Corticosteroids [ Time Frame: Baseline, up to 100 weeks ]
    Number of participants with decrease in Daily Dose of Corticosteroids

  2. Change from Baseline in Mean Daily Diary Scores [ Time Frame: Baseline, up to 100 weeks ]
    Change from Baseline in Mean Daily Diary Scores

  3. Change from Baseline in Patient's Symptom Specific Daily Diary Scores [ Time Frame: Baseline, up to 100 weeks ]
    Change from Baseline in Patient's Symptom Specific Daily Diary Scores

  4. Change From Baseline in the Physician's Global Assessment of Disease Activity Scores [ Time Frame: Baseline, up to 100 weeks ]
    Change From Baseline in the Physician's Global Assessment of Disease Activity Scores

  5. Percentage Change of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5 [ Time Frame: Pre-treatment period, up to 100 weeks ]
    Percentage Change of Days Meeting the Criteria of Participant's Mean Daily Diary Score <0.5

  6. Change in Growth Velocity [ Time Frame: Baseline, up to 100 weeks ]
    Change in Growth Velocity

  7. Change from Pre-treatment period in Mean Daily Diary Scores [ Time Frame: Pre-treatment period, up to 100 weeks ]
    Change from Pre-treatment period in Mean Daily Diary Scores

  8. Change from Pre-treatment period in the Physician's Global Assessment of Disease Activity Scores [ Time Frame: Pre-treatment period, up to 100 weeks ]
    Change from Pre-treatment period in the Physician's Global Assessment of Disease Activity Scores


Eligibility Criteria
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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04517253


Locations
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Japan
Hiroshima University Hospital
Hiroshima-shi, Hiroshima-ken, Japan, 734-8551
Nara Medical University Hospital
Kashihara, Nara, Japan, 634-8522
Tokyo Medical And Dental University Medical Hospital
Bunkyō, Tokyo, Japan, 113-8519
National Center For Child Health And Development
Setagaya-ku, Tokyo, Japan, 157-8535
Wakayama Medical University Hospital
Wakayama, Japan, 641-8510
Sponsors and Collaborators
Eli Lilly and Company
Investigators
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Study Director: 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
More Information
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Additional Information:

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Responsible Party: Eli Lilly and Company
ClinicalTrials.gov Identifier: NCT04517253    
Other Study ID Numbers: 17571
I4V-JE-JAJE ( Other Identifier: Eli Lilly and Company )
First Posted: August 18, 2020    Key Record Dates
Last Update Posted: August 19, 2022
Last Verified: August 15, 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame: Data are available 6 months after the primary publication and approval of the indication studied in the US and 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.
URL: http://vivli.org/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Eli Lilly and Company:
Nakajo-Nishimura Syndrome
CANDLE
SAVI
AGS
Additional relevant MeSH terms:
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Lipodystrophy
Skin Diseases
Syndrome
Hyperthermia
Fever
Disease
Pathologic Processes
 Skin Diseases, Metabolic
Lipid Metabolism Disorders
Metabolic Diseases
Body Temperature Changes
Heat Stress Disorders
Wounds and Injuries