Neoplasms

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Musculoskeletal Diseases

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Carcinoma

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Carcinoma, Basal Cell

Skin Neoplasms

Neoplasms, Basal Cell

Basal Cell Nevus Syndrome

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Nevus

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Skin Diseases

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Bone Diseases

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Jaw Diseases

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Congenital Abnormalities

Abnormalities, Multiple

Genetic Diseases, Inborn

Nevoid Basal Cell Carcinoma Syndrome

Basal Cell Carcinoma, Multiple

Oral hedgehog inhibitors vismodegib (Erivedge) or sonidegib (Odomzo)

Oral vismodegib (Erivedge), taken daily or every other day (depending on doctor's description)

Vismodegib

Sonidegib (Odomzo), taken daily or every other day (depending on doctor's description)

Sonidegib

Emmy Cruts, MD

S Lubeek, MD, PhD

Klara Mosterd, MD, PhD

M.W. Bekkenk, MD, PhD

B. Zupan, MD

R. van Doorn, MD, PhD

M. Wakkee, MD, PhD

A.K.L. Reyners, MD, PhD

L.A. Devriese, MD, PhD

K Mosterd, MD, PhD

Background: Oral hedgehog inhibitors vismodegib and sonidegib have been used for the treatment of locally advanced (laBCC), metastatic basal cell carcinoma (mBCC) and in basal cell nevus syndrome (BCNS) patients. In the Netherlands, targeted therapy with vismodegib and sonidegib has been available since 2013 and 2021, respectively. No direct comparative studies have been performed between the two oral hedgehog inhibitors (HHI) vismodegib and sonidegib yet . In addition, data for sonidegib are not yet available.

Objective: The aim of this study is 1) to evaluate the effectiveness of oral HHIs in the treatment of laBCC, mBCC and BCNS patients and 2) to compare the oral HHIs vismodegib and sonidegib.

Study design: prospective registration study that includes all patients, regardless of age and gender, with histologically proven basal cell carcinoma receiving treatment with either vismodegib or sonidegib in the Netherlands. Patient, tumor and treatment information was gathered from patient records.

Main study parameters/endpoints: The primary outcome for measuring efficacy/tumor response was median progression free survival (PFS) where the decrease, stagnation or increase in tumor size is measured by maximum diameter. Secondary outcomes are frequency, severity and reversibility of treatment-emergent adverse events and disease-specific quality of life expressed as mean scores on the EORTC-QLQ-C30 and aBCCdex questionnaires.

This is a prospective registration study conducted in eight academic hospitals in the Netherlands. Vismodegib and sonidegib are currently only prescribed in academic hospitals in the Netherlands. Therefore, this multicenter approach with all academic centers in the Netherlands provides a complete insight into the prescription of these oral HHIs in the Netherlands. The study takes place at the dermatology and oncology department of Maastricht University Medical Center+ (MUMC+), Erasmus University Medical Center (Erasmus MC) Rotterdam, Netherlands Cancer Institute (NKI) Amsterdam, University Medical Center Groningen (UMCG), University Medical Center Utrecht (UMC Utrecht), Amsterdam University Medical Center (Amsterdam UMC), Radboud University Medical Center (Radboudumc) and Leiden University Medical Center (LUMC).

All patients receiving at least one dose of vismodegib or sonidegib for the treatment of basal cell carcinoma (laBCC, mBCC, multiple BCCs in BCNS and in non-BCNS) (in regular care) will be included, provided they give permission and sign the informed consent form. Treating physicians then systematically register data on treatment using a uniform registration format. This registration includes the effectiveness of the drug measured by tumor diameter\*, adverse events according to the CTCAE version 5.0, and data such as age, gender, World health organization (WHO) status, scores on the G8 questionnaire, medication use, comorbidities, indication, dosage, treatment duration, laboratory values, and reason for discontinuation of treatment. In addition, twice a year scores on the EORTC-QLQ-C30 and aBCCdex questionnaires are registered. All data will be extracted from electronic patient files en will be entered anonymously in a Castor database.

The primary outcome for measuring efficacy/tumor response is median progression free survival (PFS) where the decrease, stagnation or increase in tumor size is measured by maximum tumor diameter\*. Secondary outcomes are frequency, severity and reversibility of treatment-emergent adverse events and disease-specific quality of life expressed as mean scores on the EORTC-QLQ-C30 and aBCCdex questionnaires. In addition, the association of patient characteristics on drug efficacy and adverse events will be analysed.

\*In case of gorlin goltz syndrome or multiple BCCs, at least 3 target lesions are registered whose diameters are monitored over time.

Oral Hedgehog Inhibitors in the Treatment of Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study

Registration of Oral Hedgehog Inhibitors Vismodegib and Sonidegib in the Treatment of Advanced and Multiple Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study.

The main study outcome measure is the median progression free survival (PFS) where the decrease, stagnation or increase in tumor size is measured by maximum diameter.

Frequency, severity and reversibility of treatment-emergent adverse events, measured according to the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.

Disease-specific quality of life expressed as mean scores on the validated European Organisation for Research and Treatment of Cancer (EORTC) core quality of life (EORTC-QLQ-C30) questionnaire.

The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus a high score for a functional scale represents a high / healthy level of functioning, a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology / problems.

Disease-specific quality of life expressed as mean scores on the validated advanced basal cell carcinoma index (aBCCdex) questionnaire.

The aBCCdex consists of 26 questions related to symptoms, emotions, worry, avoidance behavior and influence on daily life. Scales range in score from 1 to 7 and from 1 to 6. A higher score correlates with a poorer quality of life.

Sun Pharmaceutical Industries Limited

Maastricht University Medical Center

Wounds and Injuries

Blister

The procurement of the epidermal micrograft involves the use of the MoMelan Technologies suction blister system which consists of a control box that creates the vacuum necessary to create the microblisters and a harvester. The device is applied to the subject's thigh to create blisters. The epidermal micrografts are then harvested and transferred to Tegaderm™ and then placed on the surgical wound and the wound is bandaged.

Momelan Technologies Epidermal Graft Harvesting System

Robert LaRoche

Ashish C. Bhatia, MD

Peri Todd

This case series will assess the use of suction blister epidermal grafts harvested by a novel device on select patients that have wounds from Mohs or excisional surgery.

This study evaluates a novel micrografting technique to determine how it will influence the healing and cosmetic result of selected post surgical wounds in 12 subjects. The Epidermal Expansion System (designed by MoMelan Technologies) will generate an array of small microblisters and transfer the micrografts to a sterile wound dressing for application to the subject's surgical area. The sponsor hypothesizes that applying expanded micrografts to wounds that otherwise would have healed by second intention alone will hasten healing and possibly reduce scarring.

Use of the Epidermal Micrografts for Wound Healing After Mohs or Excisional Surgery for Skin Cancer

Use of the Epidermal Expansion System to Harvest and Place Suction Blister Epidermal Grafts on Wounds in Patients After Mohs or Excisional Surgery for Skin Cancer

Momelan Technologies

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Oral Hedgehog Inhibitors in the Treatment of Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study	NCT05463757	Basal Cell Carc... Locally Advance... Metastatic Basa... Gorlin Syndrome Basal Cell Nevu... Carcinoma, Basa... Carcinoma Basal Cell Tumo... Skin Cancer Neoplasm of Ski... Neoplasms, Basa...	Vismodegib Sonidegib	-	Maastricht University Medical Center
Use of the Epidermal Micrografts for Wound Healing After Mohs or Excisional Surgery for Skin Cancer	NCT01536444	Neoplasm of Ski...	Momelan Technol...	35 Years - 90 Years	Momelan Technologies
Use of the Epidermal Micrografts for Wound Healing After Mohs or Excisional Surgery for Skin Cancer	NCT01536444	Neoplasm of Ski...	Momelan Technol...	35 Years - 90 Years	Momelan Technologies

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