The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule
University of California Davis Cancer Center UC Davis Cancer (3)
Sacramento, California, United States
Rocky Mountain Cancer Centers RMCC - Aurora
Greenwood Village, Colorado, United States
Lurie Children's Hospital of Chicago Developmental Therapeutics
Chicago, Illinois, United States
Summary of Overall Response (ORR) and Clinical Benefit (CBR)
Clinical benefit rate (CBR) Number and percentage of subjects with CBR (responses of CR, PR or SD ≥ 16 weeks) as assessed by investigator was reported for all patients along with 95% exact confidence interval (CI). Overall Response Rate (ORR) Overall response was to be determined by investigator assessment for each tumor in the study. For subjects with solid tumors, the assessment criteria was RECIST 1.1 and included responses of CR and/or PR. The number and percentage of subjects for different categories of overall response (e.g., for solid tumors - CR, PR, SD, PD, Not Evaluable) were to be provided for solid tumors, and each hematological tumor type (if applicable). Ninety-five percent (95%) exact CI was to be provided for the response rate(s) (e.g., for solid tumors - CRn and/or PR) as well.
Time frame: 16 weeks
Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set
Progression-free survival (PFS) is the time from the date of start of treatment to the date of event defined as the first documented progression or death due to any cause within 30 days of last dose. If a subject has not had an event, progression-free survival is censored at the date of last adequate tumor assessment.
Time frame: 4 months
Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months
Time frame: 4 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Minnesota Oncology Hematology, P.A. Southdate Medical Center
Minneapolis, Minnesota, United States
Cleveland Clinic Foundation Cleveland Clinic (19)
Cleveland, Ohio, United States
Sanford Research Sanford Health
Sioux Falls, South Dakota, United States
Oncology Consultants Oncology Group
Houston, Texas, United States
MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)
Houston, Texas, United States
Intermountain Medical Center Intermountain Healthcare
Murray, Utah, United States
Seattle Cancer Care Alliance Skagit Valley Hospital
Seattle, Washington, United States