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LDE225 for Patients With PTCH1 or SMO Mutated Tumors (SIGNATURE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02002689
Recruitment Status : Terminated (Low enrollment)
First Posted : December 6, 2013
Results First Posted : May 2, 2016
Last Update Posted : May 2, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( 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

Condition or disease Intervention/treatment Phase
PTCH1 or SMO Activated Solid and Hematologic Tumors Drug: LDE225 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : February 2014
Actual Primary Completion Date : March 2015
Actual Study Completion Date : March 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Sonidegib

Arm Intervention/treatment
Experimental: LDE225
LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule.
Drug: LDE225
LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule




Primary Outcome Measures :
  1. Summary of Overall Response (ORR) and Clinical Benefit (CBR) [ Time Frame: 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 :
  1. Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set [ Time Frame: 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.

  2. Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months [ Time Frame: 4 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02002689


Locations
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United States, California
University of California Davis Cancer Center UC Davis Cancer (3)
Sacramento, California, United States, 95817
United States, Colorado
Rocky Mountain Cancer Centers RMCC - Aurora
Greenwood Village, Colorado, United States
United States, Illinois
Lurie Children's Hospital of Chicago Developmental Therapeutics
Chicago, Illinois, United States, 60611
United States, Minnesota
Minnesota Oncology Hematology, P.A. Southdate Medical Center
Minneapolis, Minnesota, United States, 55404
United States, Ohio
Cleveland Clinic Foundation Cleveland Clinic (19)
Cleveland, Ohio, United States, 44195
United States, South Dakota
Sanford Research Sanford Health
Sioux Falls, South Dakota, United States, 57104
United States, Texas
Oncology Consultants Oncology Group
Houston, Texas, United States, 77024
MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)
Houston, Texas, United States, 77030
United States, Utah
Intermountain Medical Center Intermountain Healthcare
Murray, Utah, United States, 84157
United States, Washington
Seattle Cancer Care Alliance Skagit Valley Hospital
Seattle, Washington, United States, 98109-1023
Sponsors and Collaborators
Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02002689    
Other Study ID Numbers: CLDE225XUS20
First Posted: December 6, 2013    Key Record Dates
Results First Posted: May 2, 2016
Last Update Posted: May 2, 2016
Last Verified: April 2016
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Solid tumor malignancy,
hematologic malignancy,
mutation, translocations,
signature,
PTCH1,
SMO,
LDE225,
endometrial cancer,
colon cancer,
bladder,
NSCLC
Additional relevant MeSH terms:
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Neoplasms