Ensayos clínicos

Inclusion Criteria:

• Patients ≥ 18 years of age.
• TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening).
• Symptomatic disease as defined as at least moderate pain per BPI Worst Pain or at least moderate stiffness per Worst Stiffness NRS item (defined as a score of 4 or more, with 10 describing the worst condition) within the screening period, prior to the first dose, and documented in the medical record.
• Participants should complete 14 consecutive days of questionnaires during the screening period and must meet minimum requirements as outlined in study protocol.
• Must have stable analgesic regimen, as judged by the investigator, for at least 2 weeks prior to first dose of study drug.
• Must have at least 1 measurable lesion according to RECIST Version 1.1, with a minimum tumor size of 2cm.
• Adequate organ and bone marrow function.
• If a female of childbearing potential, must have a negative pregnancy test prior to enrollment and agree to follow the contraception requirements.
• Must provide signed consent to participate in the study and is willing to comply with study-specific procedures.
• Willing and able to complete the patient-reported outcome (PRO) assessments on an electronic device.

Exclusion Criteria:

• Previous use of systemic therapy targeting colony stimulating factor 1 (CSF1) or CSFR1 receptor (CSF1R); previous therapy with imatinib and nilotinib is allowed.
• Received therapy for TGCT, including investigational therapy within 14 days prior to the administration of study drug or within 28 days for therapies with a half-life longer than 3 days or an unknown half-life prior to the administration of study drug.
• Known metastatic TGCT or other active cancer that requires concurrent treatment (exceptions will be considered on a case-by-case basis).
• QT interval corrected by Fridericia's formula (QTcF) > 450 ms in males or > 470 ms in females or history of long QT syndrome.
• Concurrent treatment with any study-prohibited medications.
• Major surgery within 14 days of the first dose of study drug.
• Any clinically significant comorbidities.
• Active liver or biliary disease including evidence of fatty liver, nonalcoholic steatohepatitis (NASH), or cirrhosis.
• Malabsorption syndrome or other illness that could affect oral absorption.
• Known active human immunodeficiency virus (HIV), active or chronic hepatitis B, active or chronic hepatitis C, or known active mycobacterium tuberculosis infection.
• If female, the participant is pregnant or lactating.
• Known allergy or hypersensitivity to any component of the study drug.
• Contraindication to MRI.