Ensayos clínicos

Inclusion Criteria:

• Male or female patient aged ≥ 18 years at Screening.
• Diagnosis of active pulmonary sarcoidosis:
  • Bilateral hilar adenopathy (BHA) on chest imaging within last 3 months of enrolment; OR
  • Perilymphatic nodules, peribronchial thickening (on chest CT), or upper lobe or diffuse infiltrates (on any chest imaging) within last 3 months of enrolment and at least one of the two:
    • Known positive biopsy of a mediastinal and/or hilar or lymph node biopsy, consistent with sarcoidosis
    • History of or active Lupus pernio or Heerfordt’s syndrome
• Parenchymal pulmonary involvement evidenced by [ 18F]FDG PET/CT imaging at Screening.
• Symptomatic patient about to start first-line therapy for pulmonary sarcoidosis.
• Body-mass Index within the range of 18 - 46 kg/m^2.
• Willingness to avoid pregnancy or fathering a child:
  • Woman of nonchildbearing potential (i.e., surgically sterile with a hysterectomy and/or bilateral oophorectomy or ≥ 12 months of amenorrhea and at least 50 years of age);
  • Woman of childbearing potential who has a negative serum pregnancy test at Screening and before the first study treatment dose on Day 1 and who agrees to take highly effective contraceptive measure to avoid pregnancy (with at least 99% effectiveness) from Screening until 120 days after EOT;
  • Man who agrees to use double barrier contraception (condoms - or diaphragm/ cervical cap used by their female partner- plus spermicidal agent: foam, gel, film etc.) to avoid fathering a child from Screening until 120 days after EOT, or is surgically sterilized;
  • Patients who have chosen sexual abstinence as a lifestyle.
• Capable of understanding and complying with protocol requirements.
• Written informed consent given by the patient before the initiation of any study procedures:
  • Note: A witnessed consent is not allowed for patients unable to read or write. Proxy consent is not allowed.

Exclusion Criteria: 

• Evidence of pulmonary sarcoidosis that requires immediate start of treatment (or start within the next 3 months) with corticosteroids, immunosuppressants, or anti-Tumor Necrosis Factor (anti-TNF) agents (or other anti-inflammatory/anti-fibrotic treatment), according to treating physician following evaluation of risk for: future mortality from sarcoidosis, permanent disability from sarcoidosis, and deterioration of quality of life due to sarcoidosis
• Cardiac sarcoidosis (known or diagnosed at Screening using the cardiac Magnetic Resonance Imaging).
• History of or active Löfgren’s syndrome.
• Established alternative diagnosis of a non-infectious or infectious systemic disease, or suspicion thereof, undermining the suspicion/diagnosis of sarcoidosis.
• If performed pre-study, mediastinal and/or hilar lymph node biopsy result not consistent with sarcoidosis (per Key Pathological Features of Sarcoidosis by the Official American Thoracic Society Clinical Practice Guideline 2020).
• Clinically significant lung disease other than sarcoidosis (including but not limited to asthma, Chronic Obstructive Pulmonary Disease, interstitial lung disease, lung cancer) or any current systemic disease other than sarcoidosis.
• Previous potentially effective systemic or inhaled pharmacological (including investigational) therapy for sarcoidosis (whether pulmonary or other disease), with the exception of any of the following :
  • Corticosteroids received prior to last 3 months before enrolment;
  • Immunosuppressants or anti-TNF agents (or other antiinflammatory/anti-fibrotic treatment) received prior to last 4 months before enrolment.
• Current systemic pharmacological treatment for sarcoidosis or any systemic disease, including any investigational drugs.
• Primary systemic treatment indication being an extrapulmonary location of sarcoidosis (e.g., neurological).
• Any contraindication to cardiac MRI procedure, including severe claustrophobia and known hypersensitivity to the contrast medium.
• Concomitant or need for treatment with a drug known for QT prolongation effect.
• History or current diagnosis of arrhythmia (other than non-sustained supraventricular arrhythmia).
• Heart failure (New York Heart Association class III or IV) and/or known myocardial hypertrophy.
• PET imaging, or other diagnostic or therapeutic procedure with administration of a radiopharmaceutical, performed within 6 weeks prior to screening.
• Known small fiber neuropathy.
• Total serum bilirubin > 1.5 x upper limit of normal (ULN) or alanine aminotransferase (ALT) or asparagine aminotransferase (AST) > 2.5 x ULN, or alkaline phosphatase (ALP) > 1.5 x ULN, or liver failure and/or cirrhosis
• Creatinine clearance (CrCL) < 60 mL/min (by Cockcroft-Gault formula).
• Hypocalcemia at Screening.
• Uncontrolled hypercalcemia or uncontrolled hypermagnesemia at Screening.
• Marked fasting hyperglycemia or uncontrolled diabetes at Screening with plasma glucose exceeding 8.3 mmol/L, or other contraindication to [ 18F]FDG administration and/or PET procedure (including body temperature > 37°C and any metabolic disease affecting the energy metabolism of muscles) as described in the PET protocol.
• Pregnancy, breastfeeding, or planning to become pregnant or breastfeed during the study treatment.
• Known positivity for Human Immunodeficiency Virus (HIV 1/2 antibodies), hepatitis B virus, or hepatitis C virus.
• Patients with psychiatric disorder that could affect the conduct of the study and/or compliance with the study treatment.
• Alcohol consumption above 20 units/week for men and 10 units/week for women.
• Known allergy to excipients of the study drug.
• Severe, uncontrolled systemic disease (e.g., clinically significant cardiovascular, pulmonary, or metabolic disease) at Screening, or other condition, which in the opinion of the investigator, would compromise the safety of the patient or the patient's ability to participate in the study.
• Unable to take oral medications.
• Participation in another clinical study within 1 month before enrollment.
• Patient deprived of liberty by a judicial or administrative decision, patient admitted to a social institution or who is under a measure of legal protection, patient hospitalized without consent or who is in an emergency situation.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 6/9/23. Questions regarding updates should be directed to the study team contact.