Clinical trial NCT06868381: A Trial of Baricitinib in Patients With Cardiac Sarcoidosis
Official title: A Phase IIa, Single-Site, Open-Label Trial of Baricitinib in Patients With Cardiac Sarcoidosis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Cardiac Sarcoidosis
Keywords: baricitinib
sarcoidosis
cardiac sarcoidosis
Janus kinase inhibitor
JAK inhibitor
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Eli Lilly and Company
First posted: 2025-03-10
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this clinical trial is to learn if baricitinib in combination with a background steroid-sparing medication can treat active cardiac sarcoidosis in adults. The main question it aims to answer is:

- In patients with active cardiac sarcoidosis, does treatment with baricitinib improve cardiac sarcoidosis disease activity as assessed by changes on cardiac FDG-PET/CT?

Participants will:

* Take baricitinib in combination with a steroid-sparing therapy for up to 16 weeks
* Visit the clinic every two to four weeks for checkups and tests
* Be asked to complete questionnaires to see how they feel on baricitinib and medication diaries to record when they take baricitinib


Eligibility criteria: Key Inclusion Criteria:

* Diagnosis of cardiac sarcoidosis based on one of the following pathways:

  * Histological Diagnosis

    * Myocardial or extracardiac biopsy demonstrating non-caseating granuloma with no alternative cause identified AND
    * Abnormal FDG uptake on cardiac PET-CT conducted within six weeks of Screening, in a pattern consistent with active cardiac sarcoidosis AND
    * Exclusion of other causes for cardiac manifestations
  * Clinical Diagnosis

    * One or more of the following is present:

      * Steroid +/- immunosuppressant responsive cardiomyopathy or heart block
      * Unexplained reduced LVEF (\< 40%) and/or segmental wall motion abnormalities not related to coronary artery disease or another defined cause
      * Unexplained sustained (spontaneous or induced) VT
      * Mobitz type II 2nd degree heart block or 3rd degree heart block
      * CT chest and/or FDG PET-CT showing features consistent with pulmonary sarcoidosis and/or hilar lymphadenopathy AND
    * Abnormal FDG uptake on cardiac PET-CT conducted within 6 weeks of Screening, in a pattern consistent with active cardiac sarcoidosis AND
    * Exclusion of other causes for cardiac manifestations
* Active cardiac sarcoidosis based on abnormal FDG uptake on cardiac PET-CT conducted within six weeks of Screening, in a pattern consistent with active cardiac sarcoidosis
* No current treatment with immunosuppressive medications other than a steroid-sparing medication (including methotrexate, leflunomide, azathioprine, or mycophenolate mofetil), and/or prednisone (or equivalent) at a dose of ≤ 20mg daily at Baseline

Key Exclusion Criteria:

* Receipt of a non-biologic DMARD or immunosuppressive agent other than methotrexate, leflunomide, azathioprine, mycophenolate mofetil, hydroxychloroquine, or glucocorticoids within 28 days prior to screening
* Receipt of a bDMARD or tsDMARD, including non-depleting B-cell-directed therapy (eg, belimumab), T cell costimulatory blockade (eg, abatacept), TNF-alpha inhibition (eg, infliximab, adalimumab, etanercept, golimumab, certolizumab pegol), interleukin-6 inhibition (eg, tocilizumab, sarilumab), interleukin-1 inhibition (eg, anakinra), JAK inhibition (eg, tofacitinib, upadacitinib, baricitinib), or other biologic immunomodulatory agent within 28 days prior to screening
* Receipt of any biologic B cell-depleting therapy (eg, rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab) in the 6 months prior to screening; receipt of such a B cell-depleting agent in the period 6-12 months prior to screening is exclusionary unless B cell counts have returned to ≥ LLN
* History of venous thromboembolism (VTE) or an increased risk for VTE
* Current smoking
* Estimated glomerular filtration rate \< 30 mL/min/1.73 m2 by Modification of Diet in Renal Disease Study (MDRD) equation
* Blood tests at screening that meet any of the following criteria:

  * Hemoglobin \< 7.5 g/dL
  * Neutrophils \< 1000/mm3
  * Absolute lymphocyte count \< 500/mm3
  * Platelets \< 100 x 109/L
* Subjects with the following abnormal liver function tests:

  * Aspartate aminotransferase (AST) \> 2x ULN
  * Alanine aminotransferase (ALT) \> 2x ULN
  * Total bilirubin (TBL) \> 2x ULN unless AST, ALT, and hemoglobin are within central laboratory normal range and the patient has a known history of Gilbert syndrome
* Active, clinically significant infection at the time of Screening
* Active malignancy or history of malignancy that was active within the last 5 years, except as follows:

  * In situ carcinoma of the cervix following apparently curative therapy \> 12 months prior to screening,
  * Cutaneous basal cell or squamous cell carcinoma following apparently curative therapy, or
  * Prostate cancer treated with radical prostatectomy or radiation therapy with curative intent \> 3 years prior to screening and without known recurrence or current treatment

Primary outcomes:
measure: Proportion of patients with resolution of cardiac FDG uptake on PET-CT
timeFrame: From baseline to end of treatment at 16 weeks
Secondary outcomes:
measure: Percent change in FDG avidity (SUVmax) in the cardiac lesion with greatest FDG avidity on PET-CT
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Percent change in total cardiac metabolic activity on FDG PET-CT
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Proportion of patients with resolution of extracardiac FDG uptake on PET-CT
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Percent change in FDG avidity (SUVmax) in up to six extracardiac lesions on PET-CT
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Percent change in total extracardiac metabolic activity on FDG PET-CT
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Proportion of patients with resolution of cardiac FDG uptake on PET-CT
timeFrame: From baseline to 8 weeks and end of follow-up at 28 weeks
measure: Change in sarcoidosis disease activity assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Change in fatigue assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Change from Baseline in Physician Disease Activity Visual Analogue Scale (VAS)
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Change from Baseline in Patient Disease Activity Visual Analogue Scale (VAS)
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Changes in ACE laboratory assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Changes in high-sensitivity troponin I laboratory assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Changes in NT-proBNP laboratory assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Changes in total IgG laboratory assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Changes in ESR laboratory assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Changes in CRP laboratory assessment
timeFrame: From baseline to 8 weeks and end of treatment at 16 weeks
measure: Number of participants with safety endpoints of interest
timeFrame: From screening to end of follow-up at 28 weeks
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
Study officials:
affiliation: Stanford University
name: Matthew C Baker, MD, MS
role: PRINCIPAL_INVESTIGATOR
References: