A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma

NCT ID: NCT01327053

Last Updated: 2019-08-28

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

230 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-06-29

Study Completion Date

2018-06-29

Brief Summary

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This study assessed the efficacy and safety of oral treatment with two dose levels of LDE225 in patients with locally advanced or metastatic BCC.

Detailed Description

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Conditions

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Basal Cell Carcinoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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LDE225 200 mg

The study was double blinded and enrolled at least 50 evaluable patients in the 200 mg LDE225 arm. The efficacy and safety of LDE225 was analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent.

Group Type EXPERIMENTAL

LDE225

Intervention Type DRUG

LDE225 was administered orally, on a continuous once daily dosing schedule and was supplied as 200 mg hard gelatin capsules in bottles. Every 4 weeks on the day of study visit, patients received a prescription of an adequate drug supply for self-administration at home. The 800 mg dose patients received 4 capsules of LDE225 and 200 mg dose arm patients received 1 LDE225 capsule + 3 placebo capsules.

LDE225 800 mg

The study was double blinded and enrolled at least 100 evaluable patients in the 800 mg LDE225 arm. The efficacy and safety of LDE225 was analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent.

Group Type EXPERIMENTAL

LDE225

Intervention Type DRUG

LDE225 was administered orally, on a continuous once daily dosing schedule and was supplied as 200 mg hard gelatin capsules in bottles. Every 4 weeks on the day of study visit, patients received a prescription of an adequate drug supply for self-administration at home. The 800 mg dose patients received 4 capsules of LDE225 and 200 mg dose arm patients received 1 LDE225 capsule + 3 placebo capsules.

Interventions

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LDE225

LDE225 was administered orally, on a continuous once daily dosing schedule and was supplied as 200 mg hard gelatin capsules in bottles. Every 4 weeks on the day of study visit, patients received a prescription of an adequate drug supply for self-administration at home. The 800 mg dose patients received 4 capsules of LDE225 and 200 mg dose arm patients received 1 LDE225 capsule + 3 placebo capsules.

Intervention Type DRUG

Other Intervention Names

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Sonidegib

Eligibility Criteria

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Inclusion Criteria

* Patients with locally advanced BCC and metastatic BCC
* Patients with adequate bone marrow, liver, and renal function

Exclusion Criteria

* Patients who had had major surgery within 4 weeks of initiation of study medication
* Patients unable to take oral drugs or with lack of physical integrity of the upper gastrointestinal tract, or known malabsorption syndromes.
* Patients with concurrent medical conditions that may interfere or potentially affect the interpretation of the study.
* Patients with neuromuscular disorders or are on concurrent treatment with drugs that may cause muscle damage.
* Patients who were on concurrent therapy with other anti-neoplastic agents.
* Patients who had taken part in an experimental drug within 4 weeks of initiation of study medication.
* Pregnant or nursing (lactating) women
* Women of child bearing potential unwilling to use 2 forms of highly effective contraception throughout the study and for 3 months after the last treatment
* Fertile males not willing to use condoms throughout the study and for 3 months after the last treatment.
* Patients who were unwilling or unable to comply with the protocol.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Highlands Oncology Group

Fayetteville, Arkansas, United States

Site Status

University of California at Los Angeles UCLA 3

Los Angeles, California, United States

Site Status

Stanford University Medical Center Stanford Univ 2

Stanford, California, United States

Site Status

University of Colorado School of Medicine UC

Aurora, Colorado, United States

Site Status

Washington Hospital Center Wash Hospital

Washington D.C., District of Columbia, United States

Site Status

H Lee Moffitt Cancer Center and Research Institute Cutaneous Onc Dept

Tampa, Florida, United States

Site Status

NorthwesternUniv.Med.School/Robert H. Lurie Comp.Cancer Ctr Study coordinator

Chicago, Illinois, United States

Site Status

Dana Farber Cancer Institute DFCI - MA

Boston, Massachusetts, United States

Site Status

Henry Ford Hospital Henry Ford

Detroit, Michigan, United States

Site Status

Washington University School Of Medicine-Siteman Cancer Ctr Siteman

St Louis, Missouri, United States

Site Status

Comprehensive Cancer Centers of Nevada CCC of Nevada- Southwest (2)

Las Vegas, Nevada, United States

Site Status

Hackensack University Medical Center Hackensack (SC)

Hackensack, New Jersey, United States

Site Status

New York University Medical Center SC-2

New York, New York, United States

Site Status

Penn State University / Milton S. Hershey Medical Center Hershey Medical

Hershey, Pennsylvania, United States

Site Status

University of Pittsburgh Medical Center UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Baylor Health Care System/Sammons Cancer Center Baylor Texas Oncology

Dallas, Texas, United States

Site Status

Texas Oncology Tex Onc 3

Dallas, Texas, United States

Site Status

Texas Oncology Texas Onc - Amarillo

Dallas, Texas, United States

Site Status

University of Texas MD Anderson Cancer Center MD Anderson

Houston, Texas, United States

Site Status

Texas Oncology Cancer Care & Research Center

Waco, Texas, United States

Site Status

Texoma Cancer Center Texoma Cancer Center

Wichita Falls, Texas, United States

Site Status

University of Utah / Huntsman Cancer Institute Huntsman/Univ UT

Salt Lake City, Utah, United States

Site Status

Novartis Investigative Site

St Leonards, New South Wales, Australia

Site Status

Novartis Investigative Site

Geelong, Victoria, Australia

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Novartis Investigative Site

Brussels, , Belgium

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Novartis Investigative Site

Wilrijk, , Belgium

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Novartis Investigative Site

Waterloo, Ontario, Canada

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Novartis Investigative Site

Sainte-Foy, Quebec, Canada

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Novartis Investigative Site

Lyon, , France

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Novartis Investigative Site

Marseille, , France

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Novartis Investigative Site

Pierre-Bénite, , France

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Novartis Investigative Site

Toulouse, , France

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Novartis Investigative Site

Villejuif, , France

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Novartis Investigative Site

Berlin, , Germany

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Novartis Investigative Site

Essen, , Germany

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Novartis Investigative Site

Freiburg im Breisgau, , Germany

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Novartis Investigative Site

Gera, , Germany

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Novartis Investigative Site

Hanover, , Germany

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Novartis Investigative Site

Kiel, , Germany

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Novartis Investigative Site

Mainz, , Germany

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Novartis Investigative Site

München, , Germany

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Novartis Investigative Site

Münster, , Germany

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Novartis Investigative Site

Stade, , Germany

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Novartis Investigative Site

Athens, , Greece

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Novartis Investigative Site

Budapest, , Hungary

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Novartis Investigative Site

Debrecen, , Hungary

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Novartis Investigative Site

Szeged, , Hungary

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Novartis Investigative Site

Torino, TO, Italy

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Novartis Investigative Site

Napoli, , Italy

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Novartis Investigative Site

Barcelona, Catalonia, Spain

Site Status

Novartis Investigative Site

Madrid, , Spain

Site Status

Novartis Investigative Site

Madrid, , Spain

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Novartis Investigative Site

Bern, , Switzerland

Site Status

Novartis Investigative Site

Geneva, , Switzerland

Site Status

Novartis Investigative Site

Zurich, , Switzerland

Site Status

Novartis Investigative Site

High Heaton, Newcastle Upon Tyne, United Kingdom

Site Status

Novartis Investigative Site

Yeovil, Somerset, United Kingdom

Site Status

Novartis Investigative Site

Cardiff, , United Kingdom

Site Status

Novartis Investigative Site

Glasgow, , United Kingdom

Site Status

Novartis Investigative Site

Leicester, , United Kingdom

Site Status

Novartis Investigative Site

London, , United Kingdom

Site Status

Novartis Investigative Site

Manchester, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada France Germany Greece Hungary Italy Spain Switzerland United Kingdom

References

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Gutzmer R, Robert C, Loquai C, Schadendorf D, Squittieri N, Arntz R, Martelli S, Dummer R. Assessment of various efficacy outcomes using ERIVANCE-like criteria in patients with locally advanced basal cell carcinoma receiving sonidegib: results from a preplanned sensitivity analysis. BMC Cancer. 2021 Nov 19;21(1):1244. doi: 10.1186/s12885-021-08968-1.

Reference Type DERIVED
PMID: 34798846 (View on PubMed)

Lewis K, Dummer R, Farberg AS, Guminski A, Squittieri N, Migden M. Effects of Sonidegib Following Dose Reduction and Treatment Interruption in Patients with Advanced Basal Cell Carcinoma During 42-Month BOLT Trial. Dermatol Ther (Heidelb). 2021 Dec;11(6):2225-2234. doi: 10.1007/s13555-021-00619-4. Epub 2021 Oct 20.

Reference Type DERIVED
PMID: 34669179 (View on PubMed)

Lear JT, Migden MR, Lewis KD, Chang ALS, Guminski A, Gutzmer R, Dirix L, Combemale P, Stratigos A, Plummer R, Castro H, Yi T, Mone M, Zhou J, Trefzer U, Kaatz M, Loquai C, Kudchadkar R, Sellami D, Dummer R. Long-term efficacy and safety of sonidegib in patients with locally advanced and metastatic basal cell carcinoma: 30-month analysis of the randomized phase 2 BOLT study. J Eur Acad Dermatol Venereol. 2018 Mar;32(3):372-381. doi: 10.1111/jdv.14542. Epub 2017 Nov 6.

Reference Type DERIVED
PMID: 28846163 (View on PubMed)

Migden MR, Guminski A, Gutzmer R, Dirix L, Lewis KD, Combemale P, Herd RM, Kudchadkar R, Trefzer U, Gogov S, Pallaud C, Yi T, Mone M, Kaatz M, Loquai C, Stratigos AJ, Schulze HJ, Plummer R, Chang AL, Cornelis F, Lear JT, Sellami D, Dummer R. Treatment with two different doses of sonidegib in patients with locally advanced or metastatic basal cell carcinoma (BOLT): a multicentre, randomised, double-blind phase 2 trial. Lancet Oncol. 2015 Jun;16(6):716-28. doi: 10.1016/S1470-2045(15)70100-2. Epub 2015 May 14.

Reference Type DERIVED
PMID: 25981810 (View on PubMed)

Other Identifiers

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2010-022629-14

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CLDE225A2201

Identifier Type: -

Identifier Source: org_study_id

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