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Herbal and Botanical

All Drugs and Chemicals

Cola

LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule.

LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule

LDE225

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

LDE225 for Patients With PTCH1 or SMO Mutated Tumors

Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module - 5 LDE225 for Patients With PTCH1 or SMO Mutated Tumors

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.

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.

Novartis Pharmaceuticals

All the patients received sonidegib on a flat scale of 800 mg (e.g., 4 x 200 mg hard gelatin capsules) once daily on a continuous dosing cycle.

Sonidegib

Age, Categorical

Age, Continuous

Sex: Female, Male

The study was closed for accrual when the sponsor realized that not enough patients will be recruited for any meaningful stat analysis even if the study were kept open beyond the original planned accrual window.

Clinical Disclosure Office

Complete response (CR)

Partial response (PR)

Stable disease (SD)

Progressive disease (PD)

Non-evaluable (NE)

Overall response rate (ORR: CR+PR)

Clinical benefit rate (CBR: CR+PR+SD)

Summary of Overall Response (ORR) and Clinical Benefit (CBR)

1 Month

2 Months

3 Months

4 Months

Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set

Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months

Overall Study

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
LDE225 for Patients With PTCH1 or SMO Mutated Tumors	NCT02002689	PTCH1 or SMO Ac...	LDE225	18 Years -	Novartis
LDE225 for Patients With PTCH1 or SMO Mutated Tumors	NCT02002689	PTCH1 or SMO Ac...	LDE225	18 Years -	Novartis

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