Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

May 3, 2017 updated by: Novartis Pharmaceuticals

A Randomized, Double-blind, Vehicle-controlled, Multicenter Trial of Topically Administered LDE225 Cream (0.75% Bid) to Evaluate Clearance of Basal Cell Carcinoma in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome

This 22 week study will assess the efficacy, safety, and tolerability of LDE225 versus vehicle when applied topically to basal cell carcinoma (BCC) in patients with NBCCS. Patients will treat multiple BCCs for up to 12 weeks. Treatment success is defined as complete clinical clearance and complete histological clearance in BCCs.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Bruxelles, Belgium
        • Novartis Investigative Site
      • Leuven, Belgium
        • Novartis Investigative Site
  • Canada
    • Ontario
      • Waterloo, Ontario, Canada
        • Novartis Investigative Site
    • Quebec
      • Sainte-Foy, Quebec, Canada
        • Novartis Investigative Site
  • Czechia
      • Praha, Czechia
        • Novartis Investigative Site
  • Finland
      • Helsinki, Finland
        • Novartis Investigative Site
  • Germany
      • Berlin, Germany
        • Novartis Investigative Site
      • Bonn, Germany
        • Novartis Investigative Site
      • Dresden, Germany
        • Novartis Investigative Site
      • Essen, Germany
        • Novartis Investigative Site
      • Frankfurt, Germany
        • Novartis Investigative Site
      • Heidelberg, Germany
        • Novartis Investigative Site
      • Jena, Germany
        • Novartis Investigative Site
      • Kiel, Germany
        • Novartis Investigative Site
      • Luebeck, Germany
        • Novartis Investigative Site
      • Mainz, Germany
        • Novartis Investigative Site
      • Muenster, Germany
        • Novartis Investigative Site
      • Munich, Germany
        • Novartis Investigative Site
      • Recklinghausen, Germany
        • Novartis Investigative Site
      • Tuebingen, Germany
        • Novartis Investigative Site
      • Wuerzburg, Germany
        • Novartis Investigative Site
  • Greece
      • Athens, Greece
        • Novartis Investigative Site
      • Thessaloniki, Greece
        • Novartis Investigative Site
  • Italy
      • Catania, Italy
        • Novartis Investigative Site
      • L'Aquila, Italy
        • Novartis Investigative Site
      • Napoli, Italy
        • Novartis Investigative Site
      • Padova, Italy
        • Novartis Investigative Site
      • Siena, Italy
        • Novartis Investigative Site
  • Spain
      • Barcelona, Spain
        • Novartis Investigative Site
      • Madrid, Spain
        • Novartis Investigative Site
  • Switzerland
      • Lausanne, Switzerland
        • Novartis Investigative Site
      • Zuerich, Switzerland
        • Novartis Investigative Site
  • United Kingdom
      • Glasgow, United Kingdom
        • Novartis Investigative Site
      • London, United Kingdom
        • Novartis Investigative Site
      • Manchester, United Kingdom
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Adult greater than 18 years old, male or female.
  2. Patient must be able to understand and communicate with the investigator and comply with the requirements of the study.
  3. Typical presentation of NBCCS in the opinion of the investigator
  4. At least one of the major clinical criteria of NBCCS.
  5. Multiple BCCs during the screening period

Exclusion Criteria:

  1. Any concomitant dermatological disease that could confound the evaluations, based on the discretion of the investigator.
  2. Prior exposure to LDE225.
  3. Use of systemic treatment for BCC in the 4 weeks prior to Baseline.
  4. Use of topical treatment or photodynamic therapy (PDT) in the 12 weeks prior to Baseline.
  5. Use of other investigational drugs at Baseline, or within 30 days or 5 half-lives prior to Baseline, whichever is longer.
  6. Clinically significant medical condition, as per judgment of the investigator.
  7. History of hypersensitivity to any of the ingredient of the study drug.
  8. Pregnant or nursing (lactating) women
  9. Women of child-bearing potential and fertile males, UNLESS they are using two birth control methods

Other protocol-defined inclusion/exclusion criteria may apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Vehicle
Drug: Vehicle
cream, 2x daily
Active Comparator: LDE225 0.75% cream
Drug: LDE225B
Cream, 2x daily

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
To demonstrate superiority of topical LDE225 versus vehicle in terms of treatment success in patients with NBCCs. Measure: Complete clinical clearance and complete histological clearance of BCCs
Time Frame: 4 weeks after LDE225 treatment
4 weeks after LDE225 treatment

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
To assess the safety of topical treatment with LDE225, compared to vehicle. Measure: Reported systemic adverse events in patients with NBCCS
Time Frame: Treatment phase and up to 4 weeks after LDE225 treatment
Treatment phase and up to 4 weeks after LDE225 treatment
To evaluate the local tolerability of topical treatment with LDE225, compared to vehicle. Measure:Reported local adverse events in patients with NBCCS
Time Frame: Treatment phase and up to 4 weeks after LDE225 treatment
Treatment phase and up to 4 weeks after LDE225 treatment
To assess the rate of complete clinical clearance of BCCs in patients with NBCCS. Measure: No clinical residual signs of carcinoma
Time Frame: 4 weeks after LDE225 treatment
4 weeks after LDE225 treatment
To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue
Time Frame: 4 weeks after LDE225 treatment
4 weeks after LDE225 treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 7, 2022

Primary Completion

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 7, 2022

Study Completion

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 7, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 6, 2010

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 28, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 3, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 5, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 3, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CLDE225B2307

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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