A Single-arm, Open-label Phase 2 Study of Nirogacestat in Adult Japanese Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
This study is being conducted to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis.
• Participant is aged ≥18 at the time of signing the informed consent.
• Participant has histologically confirmed DT (by local pathologist prior to informed consent) that has progressed by ≥20% as measured by RECIST v1.1 within 12 months of the screening visit scan.
• Participant has:
‣ Treatment-naive, measurably progressing DT that is deemed not amenable to surgery without the risk of significant morbidity; OR
⁃ Recurrent, measurably progressing DT following at least 1 line of therapy; OR
⁃ Refractory, measurably progressing DT following at least 1 line of therapy.
• Participant agreed to provide archival or new tumor tissue for re-confirmation of disease.
• Participant has a DT tumor where continued PD will not result in immediate significant risk to the participant.
• Participants who are receiving chronic NSAIDs as treatment for conditions other than DT must be receiving them prior to documented DT progressive disease (inclusion criterion 2) and on a stable dose for at least 28 days prior to the first dose of study treatment.
• Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤2 at screening
• Participant has adequate organ and bone marrow function