LDE225 for Patients With PTCH1 or SMO Mutated Tumors

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-02-28

Study Completion Date

2015-03-31

Brief Summary

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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

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Detailed Description

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Conditions

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PTCH1 or SMO Activated Solid and Hematologic Tumors

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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LDE225

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

Group Type EXPERIMENTAL

LDE225

Intervention Type DRUG

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

Interventions

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LDE225

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

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No