LDE225 for Patients With PTCH1 or SMO Mutated Tumors

Phase 2

Terminated

• Conditions: PTCH1 or SMO Activated Solid and Hematologic Tumors
• Interventions: Drug: LDE225

• Registration Number: NCT02002689
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-12-01
• Study First Submitted QC: 2013-12-01
• Study First Posted: 2013-12-06
• Study Start: 2014-02
• Primary Completion: 2015-03
• Study Completion: 2015-03
• Results First Submitted: 2016-03-23
• Status Verified: 2016-04
• Results First Submitted QC: 2016-04-25
• Last Update Submitted: 2016-04-25
• Results First Posted: 2016-05-02
• Last Update Posted: 2016-05-02

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Patient has confirmed diagnosis of a select solid tumor (except medulloblastoma, basal cell carcinoma and pancreatic adenocarcinoma) or hematological malignancy (except CML, ALL and AML).
• Patient has pre-identified tumor with a PTCH1 or SMO mutation.
• Patient has received at least one prior treatment for recurrent, metastatic and /or locally advanced disease and for whom no standard therapy options are anticipated to result in a durable remission.
• Patient has progressive and measurable disease as per RECIST 1.1. or other appropriate hematological guidelines.
• Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1

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Exclusion Criteria

• Patients has received prior treatment with LDE225.
• Patients has neuromuscular disorders associated with elevated CK (i.e. inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis and spinal muscular atrophy) or are on concomitant treatment with drugs that are recognized to cause rhabdomyolysis
• Patients has primary CNS tumor or CNS tumor involvement
• Patient has received chemotherapy or anticancer therapy ≤ 4 weeks prior to starting study drug

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Summary of Overall Response (ORR) and Clinical Benefit (CBR)	16 weeks	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.

Secondary Outcome Measures

Name	Time	Method
Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set	4 months	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.
Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months	4 months

Trial Locations

Locations (10)

Sanford Research Sanford Health; 🇺🇸 Sioux Falls, South Dakota, United States

University of California Davis Cancer Center UC Davis Cancer (3); 🇺🇸 Sacramento, California, United States

Cleveland Clinic Foundation Cleveland Clinic (19); 🇺🇸 Cleveland, Ohio, United States

Rocky Mountain Cancer Centers RMCC - Aurora; 🇺🇸 Greenwood Village, Colorado, United States

Oncology Consultants Oncology Group; 🇺🇸 Houston, Texas, United States

Minnesota Oncology Hematology, P.A. Southdate Medical Center; 🇺🇸 Minneapolis, Minnesota, United States

Lurie Children's Hospital of Chicago Developmental Therapeutics; 🇺🇸 Chicago, Illinois, 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