Clinical trial NCT02002689: LDE225 for Patients With PTCH1 or SMO Mutated Tumors
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 type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: PTCH1 or SMO Activated Solid and Hematologic Tumors
Keywords: Solid tumor malignancy,
hematologic malignancy,
mutation, translocations,
signature,
PTCH1,
SMO,
LDE225,
endometrial cancer,
colon cancer,
bladder,
NSCLC
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-12-06
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility 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

Primary outcomes:
measure: Summary of Overall Response (ORR) and Clinical Benefit (CBR)
timeFrame: 16 weeks
Secondary outcomes:
measure: Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set
timeFrame: 4 months
measure: Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months
timeFrame: 4 months
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: University of California Davis Cancer Center UC Davis Cancer (3)
state: California
city: Greenwood Village
country: United States
facility: Rocky Mountain Cancer Centers RMCC - Aurora
state: Colorado
city: Chicago
country: United States
facility: Lurie Children's Hospital of Chicago Developmental Therapeutics
state: Illinois
city: Minneapolis
country: United States
facility: Minnesota Oncology Hematology, P.A. Southdate Medical Center
state: Minnesota
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation Cleveland Clinic (19)
state: Ohio
city: Sioux Falls
country: United States
facility: Sanford Research Sanford Health
state: South Dakota
city: Houston
country: United States
facility: Oncology Consultants Oncology Group
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)
state: Texas
city: Murray
country: United States
facility: Intermountain Medical Center Intermountain Healthcare
state: Utah
city: Seattle
country: United States
facility: Seattle Cancer Care Alliance Skagit Valley Hospital
state: Washington
Study officials:
References: