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This study evaluates nirogacestat (PF-03084014) in the treatment of desmoid tumor/aggressive fibromatosis (DT/AF). In the double-blind phase, half of the participants will receive nirogacestat while the other half will receive placebo. Once participants are eligible to roll into the open-label phase, they will receive nirogacestat.
Full description
Desmoid tumors, also referred to as aggressive fibromatosis, are rare, locally invasive, slow growing soft tissue tumors. Although considered benign because of their inability to metastasize, desmoid tumors can cause significant morbidity and occasionally mortality in patients.
Nirogacestat (PF-03084014) is a potent, small molecule, selective, reversible, noncompetitive inhibitor of γ-secretase (GS) with a potential antitumor activity.
Nirogacestat is being investigated for the treatment of desmoid tumors due to its ability to bind to GS, blocking proteolytic activation of Notch receptors. Previous clinical study data have shown that Notch signaling plays an important role in cancer development. Hence, inhibition of Notch signaling is an important strategy for therapeutic treatment.
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Inclusion and exclusion criteria
Double-Blind Key Inclusion Criteria:
Participant has histologically confirmed DT/AF (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:
Participant has a DT/AF tumor where continued progressive disease will not result in immediate significant risk to the participant.
Participant agrees to provide archival or new tumor tissue for re-confirmation of disease.
If participant is currently being treated with any therapy for the treatment of DT/AF, this must be completed at least 28 days (or 5 half-lives, whichever is longer) prior to first dose of study treatment. All toxicities from prior therapy must be resolved to ≤Grade 1 or clinical baseline.
Participants who are receiving chronic nonsteroidal anti-inflammatory drugs (NSAIDs) as treatment for conditions other than DT/AF must be receiving them prior to the documented DT/AF progressive disease (inclusion criteria 2) and on a stable dose for at least 28 days prior to 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.
Double-Blind Key Exclusion Criteria:
OR
Participant has started any treatment for DT/AF after the documented DT/AF progressive disease.
Open-Label Key Inclusion
Open-Label Key Exclusion
Primary purpose
Allocation
Interventional model
Masking
142 participants in 3 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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