Publications by authors named "Moriah Martindale"
GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS): psychometric properties and clinically meaningful thresholds as assessed in the Phase 3 DeFi randomized controlled clinical trial.
Qual Life Res
October 2023
Article Synopsis
- The GODDESS tool was created to measure the severity of Desmoid Tumor/Aggressive Fibromatosis symptoms and their effects on patients' daily lives, and the study assessed its measurement reliability and validity.
- The study involved 142 participants from a randomized trial of nirogacestat, highlighting the tool's strong internal consistency and ability to differentiate between symptom severity groups over time.
- Results indicated that GODDESS is a reliable and valid endpoint for clinical trials, with defined meaningful change thresholds that can help assess treatment responses in future studies.
Purpose: Because the Hedgehog and Notch pathways are often overexpressed in mesenchymal malignancies, we evaluated the efficacy of concurrent inhibition of Notch and Hedgehog signaling using the gamma-secretase inhibitor (GSI) RO4929097 and the smoothened antagonist vismodegib in unresectable or metastatic sarcoma.
Patients And Methods: In this investigator-initiated trial, phase Ib used standard 3+3 dose escalation in which patients first received vismodegib once daily for 21 days, followed by the combination of RO4929097 concurrently with vismodegib in 21-day cycles. In phase II, patients were randomized to RO4929097 alone or in combination with vismodegib.
Purpose: This phase Ib trial was designed to evaluate the safety and early efficacy signal of the combination of imatinib and binimetinib in patients with imatinib-resistant advanced gastrointestinal stromal tumors (GISTs).
Patients And Methods: This trial used a standard 3 + 3 design to determine the recommended phase II dose (RP2D). Additional patients were enrolled on an expansion cohort at the RP2D enriching for succinate dehydrogenase (SDH)-deficient GISTs to explore potential efficacy.
Article Synopsis
- A clinical trial investigated the combined effects of imatinib and binimetinib as a first-line treatment for advanced gastrointestinal stromal tumors (GISTs) to enhance efficacy compared to imatinib alone.
- The trial included treatment-naive adult patients, aiming for a 20% improvement in best objective response rate (ORR), with 29 out of 42 patients achieving confirmed partial responses, resulting in an ORR of 69%.
- Additional findings showed a median progression-free survival of 29.9 months, successful pathologic responses in patients undergoing surgery, and manageable toxicity levels without unexpected adverse effects.