Neoadjuvant Vismodegib in Patients With Large and/or Recurrent Resectable Basal Cell Carcinoma

NCT03035188 | Completed Phase 2

• 1 other identifier: ADO-EP02 (ML29328)
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 12

Brief Summary

In this study patients with resectable basal cell carcinoma (BCC) who usually undergo surgery without prior anticancer treatment will be treated with antitumor medication. But since BCC is mainly localized in clearly visible regions of the body, as e.g. the face, there is also a need to reduce scars as a consequence of surgery which will be accomplished by neoadjuvant therapy. The used medication - vismodegib - displays controllable adverse events and shows a good efficacy for reduction of BCC lesions. It is expected that the neoadjuvant setting will lead to minor surgical intervention thus minimising surgical risks and scars for the patients.

Conditions

Basal Cell Carcinoma

Keywords

resectable

Outcome Measures

Primary Outcomes (2)

• Disease control rate (DCR) defined as complete response (CR), partial response (PR), or stable disease (SD) after 12 weeks of treatment with vismodegib

  Rate of patients with CR, PR and SD

  12 weeks

• Objective and relative (%) reduction of the involved skin surface after 12 weeks of treatment with vismodegib

  Percent change of the BCC area from baseline to end of study therapy

  12 weeks

Secondary Outcomes (4)

• DCR (CR, PR, or SD) after 12 weeks of treatment with vismodegib in the neoadjuvant treatment setting for different basal cell carcinoma histotypes (superficial, scleroderma, nodular, others)

  12 weeks

• Duration of overall response (DoR)

  12 months

• Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  12 months

• Health-related quality of life in patients receiving vismodegib for neoadjuvant treatment of basal cell carcinoma as measured by the Skindex-16 questionnaire

  12 months

Other Outcomes (1)

• Diagnostic suitability of non-invasive imaging techniques (in vivo confocal laserscan-microscopy and/or optical coherence tomography for the evaluation of response status of patients receiving vismodegib in the neoadjuvant setting)

  12 months

Study Arms (1)

Vismodegib

EXPERIMENTAL

Continuous once-daily oral dosing of vismodegib at a dosage of 150 mg per administration

Drug: Vismodegib

Interventions

Vismodegib DRUG

1 capsule (150 mg vismodegib) taken once daily for a maximum of 12 weeks.

Also known as: Erivedge

Vismodegib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patient aged ≥ 18 years
• Able to participate and willing to give written informed consent including consent for photographs prior to performance of study-related procedures and to comply with the study protocol.
• Patients with at least 1 large (≥ 2 cm in diameter in head/neck region, ≥ 5 cm for trunk/extremities) basal cell carcinoma (BCC), still resectable, but with increased risk for cosmetic disfigurement or functional defects by assessment of the enrolling physician. Patients with large (as defined above) recurrent basal cell carcinoma are also eligible.
• Patients must be naïve to treatment with vismodegib or other hedgehog pathway inhibitors
• Local histopathologic confirmation of BCC (3 mm punch biopsy)
• Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2
• Consent to undergo mapping biopsies upon reaching complete response
• Adequate hematologic and organ function, defined by the following laboratory results, to be obtained within 7 days prior to registration and prior to first dose of study drug treatment:
• Absolute neutrophilic count \> 1,0 x 109/L
• Platelet count ≥ 75 x 109/L
• Hemoglobin ≥ 8,5 g/dL
• Albumin ≥ 2.5 g/dL
• Bilirubin ≤ 1.5 x the upper limit of normal (ULN) or within 3 x ULN for patients
• Aspartate-aminotransferase, Alanine-aminotransferase, and alkaline phosphatase ≤ 3 x ULN
• Serum creatinine ≤ 1.5 x ULN

You may not qualify if:

• History of prior treatment with vismodegib or any other hedgehog pathway inhibitor.
• Radiotherapy that involved the field of the target lesion within 6 months prior to registration. Only one radiotherapy of the target lesion performed \> 6 months prior to registration is allowed. If a second radiotherapy in this field took place, patient will be excluded.
• Any metastatic BCC
• BCC lesion that is considered to be inoperable (e.g. medical contraindication to surgery, suspicion of bone infiltration)
• Metatypic BCC
• Known or suspected Gorlin-Goltz syndrome
• Uncontrolled medical illness, including advanced malignancies (no activities of the malignancies in the past 3 years), at the discretion of the Investigator
• History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that might affect interpretation of the results of the study or renders the patient at high risk for treatment complications
• History (within 6 months prior to registration) or current signs or symptoms of severe, progressive, or uncontrolled renal, hepatic, cardiac, vascular, pulmonary, gastrointestinal, endocrine, neurologic, hematologic, rheumatologic, psychiatric, or metabolic disturbances
• Any medical or psychological illness or condition preventing adequate consent or ability to comply with the protocol
• Inability or unwillingness to swallow capsules
• Inability or unwillingness to comply with study and follow-up procedures
• Current severe, uncontrolled systemic disease
• History of malabsorption or other conditions that would interfere with the absorption of the orally applicated study drug
• Pregnant, lactating, or breast feeding women

Sponsors & Collaborators

• SRH Wald-Klinikum Gera GmbH: lead

Study Sites (12)

SRH Wald-Klinikum Gera GmbH

Gera, Thuringia, 07548, Germany

Location

Klinikum Augsburg Süd

Augsburg, 86179, Germany

Location

Charité - Universitätsmedizin Berlin

Berlin, 10117, Germany

Location

Elbe Kliniken Stade - Buxtehude GmbH

Buxtehude, 21614, Germany

Location

Universitätsklinik Carl Gustav Carus der Technischen Universität Dresden

Dresden, 01307, Germany

Location

HELIOS Klinikum Erfurt

Erfurt, 99089, Germany

Location

Universitätsklinikum Leipzig

Leipzig, 04103, Germany

Location

Universitätsklinikum Münster

Münster, 48149, Germany

Location

Fachklinik Hornheide

Münster, 48157, Germany

Location

Klinikum Nürnberg Nord

Nuremberg, 90419, Germany

Location

Harzklinikum Dorothea Christiane Erxleben GmbH

Quedlinburg, 06484, Germany

Location

Universitätsklinikum Tübingen

Tübingen, 72076, Germany

Location

Related Publications (1)

• Lauterbach B, Kakkassery V, Debus D, Heindl LM, Schultz ES. [Advanced periocular basal cell carcinoma-a therapeutic challenge]. Ophthalmologe. 2019 Mar;116(3):273-277. doi: 10.1007/s00347-018-0734-9. German.

  PMID: 29777299 DERIVED

MeSH Terms

Conditions

Carcinoma, Basal Cell

Interventions

HhAntag691

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Basal Cell

Study Officials

• Martin Kaatz, PD Dr.

  martin.kaatz@wkg.srh.de

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 26, 2016
• First Posted: January 27, 2017
• Study Start: January 1, 2017
• Primary Completion: January 1, 2020
• Study Completion: January 1, 2021
• Last Updated: February 11, 2022

Record last verified: 2019-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (12)