Study Stopped
Low enrollment
LDE225 for Patients With PTCH1 or SMO Mutated Tumors
SIGNATURE
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
1 other identifier
interventional
10
1 country
10
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Feb 2014
Shorter than P25 for phase_2
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 1, 2013
CompletedFirst Posted
Study publicly available on registry
December 6, 2013
CompletedStudy Start
First participant enrolled
February 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2015
CompletedResults Posted
Study results publicly available
May 2, 2016
CompletedMay 2, 2016
April 1, 2016
1.1 years
December 1, 2013
March 23, 2016
April 25, 2016
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
16 weeks
Secondary Outcomes (2)
Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set
4 months
Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months
4 months
Study Arms (1)
LDE225
EXPERIMENTALLDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule.
Interventions
LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (10)
University of California Davis Cancer Center UC Davis Cancer (3)
Sacramento, California, 95817, United States
Rocky Mountain Cancer Centers RMCC - Aurora
Greenwood Village, Colorado, United States
Lurie Children's Hospital of Chicago Developmental Therapeutics
Chicago, Illinois, 60611, United States
Minnesota Oncology Hematology, P.A. Southdate Medical Center
Minneapolis, Minnesota, 55404, United States
Cleveland Clinic Foundation Cleveland Clinic (19)
Cleveland, Ohio, 44195, United States
Sanford Research Sanford Health
Sioux Falls, South Dakota, 57104, United States
Oncology Consultants Oncology Group
Houston, Texas, 77024, United States
MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)
Houston, Texas, 77030, United States
Intermountain Medical Center Intermountain Healthcare
Murray, Utah, 84157, United States
Seattle Cancer Care Alliance Skagit Valley Hospital
Seattle, Washington, 98109-1023, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
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.
Results Point of Contact
- Title
- Clinical Disclosure Office
- Organization
- Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 1, 2013
First Posted
December 6, 2013
Study Start
February 1, 2014
Primary Completion
March 1, 2015
Study Completion
March 1, 2015
Last Updated
May 2, 2016
Results First Posted
May 2, 2016
Record last verified: 2016-04