Study of Cabiralizumab in Patients With Pigmented Villonodular Synovitis / Diffuse Type Tenosynovial Giant Cell Tumor

A Phase 1/2 study was an open-label, dose escalation and dose expansion study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and preliminary efficacy of cabiralizumab, a CSF1-R monoclonal antibody, inpatients with unresectable diffuse tenosynovial giant cell tumors (TGCT).

Inclusion criteria

• Histologically confirmed diagnosis of inoperable PVNS/ dt-TGCT or potentially resectable tumor that would result in unacceptable functional loss or morbidity as determined by a qualified surgeon or multi-disciplinary tumor board (must be documented in the CRF during screening)
• Measurable PVNS/dt-TGCT by RECIST 1.1 on MRI
• ECOG performance status <1

Exclusion criteria

• Prior therapy with an anti-CSF1R antibody
• Prior therapy with PLX3397 unless discontinued for intolerance (i.e., non-progression on prior kinase inhibitor)
• Liver function tests (including ALT, AST, and total bilirubin), outside of the range of local laboratory normal at Screening
• Inadequate organ or bone marrow function
• History of congestive heart failure or myocardial infarction <1 year prior to first study dose administration
• Significant abnormalities on ECG at Screening
• Contraindications to MRI and use of intravenous gadolinium-based contrast agents
• Creatine Kinase ≥ 1.5x the upper limit of normal
• Positive test for latent TB at Screening (Quantiferon test)
• Active known or suspected autoimmune disease