SARC044
13 evidence topics · 5 sources
Objective
Safety and efficacy of combination treatment
Location and study date
Participating U.S. locations
| Location | Reported site location |
|---|---|
| Massachusetts | “Boston, MA, USA” |
| Pennsylvania | “Philadelphia, PA, USA” |
| Oregon | “Portland, OR, USA” |
| Florida | “Miami, FL, USA” |
Study design
Eligibility criteria
Treatment
Bezuclastinib lead-in and added sunitinib
Study outcomes
Rationale for using progression-free survival as the primary endpoint
Primary endpoint and planned patient-reported outcome instrument
| Measure | Reported measure |
|---|---|
| Primary endpoint | “mPFS” |
| Patient-reported outcome instrument | “EORTC QLQ-C30” |
Regulatory context for progression-free survival
Study-specific endpoint justification
No evidence found.
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
Summary
The design used a one-sample log-rank calculation with a one-sided 5% significance level. Thirty-five evaluable participants provided 83% power for an assumed median progression-free survival of 6.5 months compared with a 4-month historical benchmark. This is a historical-control calculation, not a randomized comparison.
Statistical planning assumptions
| Parameter | Value |
|---|---|
| Statistical test | “one-sample log-rank test” |
| One-sided significance level | “5%” |
| Power | “83%” |
| Assumed median progression-free survival, months | “6.5” |
| Historical-control median progression-free survival, months | “4” |
Results
Participant disposition
Baseline characteristics
No evidence found.
Efficacy results
No evidence found.
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
No evidence found.
Study limitations
Summary
SARC044 is a single-arm, open-label phase 2 study with planned enrollment of up to 40 participants. Its statistical comparison uses a historical progression-free-survival benchmark rather than a concurrently randomized control. Eligibility primarily specifies KIT exon 9- or 11-mutant GIST, with other primary KIT mutations considered case by case. The enrollment-completion announcement does not establish efficacy or safety results.