Neoplasms

All Conditions

Skin and Connective Tissue Diseases

Carcinoma

Carcinoma, Basal Cell

Neoplasms, Basal Cell

Dermatitis, Phototoxic

Dermatitis

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Dermatitis, Irritant

Dermatitis, Contact

Skin Diseases

Photosensitivity Disorders

Skin Diseases, Eczematous

Dermatologic Agents

All Drugs and Chemicals

Aminolevulinic Acid

Methyl 5-aminolevulinate

Photosensitizing Agents

Fluoxymesterone

2% Hexylaminolevulinate (Hexvix, Photocure) mixed with Unguentum M (Allmiral) cream

78 mg/g Aminolevulinic Acid Nano Emulsion

The cream is mixed up by the Pharmacy Yliopiston Apteekki for the study and every set is analysed by mass spectrometry.

Hexylaminolevulinate cream

Aminolevulinic Acid Nano Emulsion

Methylaminolevulinate cream

Mari Grönroos, MD, PhD

Mari K Salmivuori, MD

This pilot study compares three photosensitisers, hexylaminolevulinate (HAL) and aminolevulinic acid nano emulsion (BF-200 ALA) to methylaminolevulinate (MAL) in photodynamic therapy of superficially growing basal cell carcinomas. Study is conducted using randomised prospective double blinded comparing design. Fluorescence is measured in A.U. (Arbitrary Unit) with standardised set-up before and after the exposure. Efficacy is assessed clinically, histologically and by hyperspectral imaging system at 3 months, 12 months and 5 years.

Study recruits volunteering patients, who are referred to the department of dermatology and allergology, Päijät-Häme Central Hospital, Lahti, with 99 clinically assessed superficial basal cell carcinoma on the body area, not face and scalp. Diagnoses is confirmed histologically by punch biopsies and hyperspectral images are taken before the biopsies. The lesions are randomised in three groups: interventions HAL and BF-200 ALA and comparator MAL. Photodynamic therapy is given by the standard procedure. Fluorescence is measured in A.U. (Arbitrary Unit) with standardised set-up, with Wood's light, digital camera and a yellow lens, before and after the exposure. Pain is measured in VAS-scale before, during and after the exposure. Efficacy is assessed clinically, histologically and by hyperspectral imaging system at 3 months, 12 months and 5 years.

Superficial Basal Cell Cancer's Photodynamic Therapy: Comparing Three Photosensitizers: HAL and BF-200 ALA Versus MAL

Superficial Basal Cell Cancer's Photodynamic Therapy: Comparing Three Photosensitizers: Hexylaminolevulinate and Aminolevulinic Acid Nano Emulsion Versus Methylaminolevulinate

Tampere University

University of Jyvaskyla

Joint Authority for Päijät-Häme Social and Health Care

Symptoms and General Pathology

Musculoskeletal Diseases

Mouth and Tooth Diseases

Diseases and Abnormalities at or Before Birth

Rare Diseases

Syndrome

Skin Neoplasms

Basal Cell Nevus Syndrome

Nevus, Pigmented

Nevus

Cysts

Odontogenic Cysts

Jaw Cysts

Bone Cysts

Neoplastic Syndromes, Hereditary

Bone Diseases

Bone Diseases, Developmental

Jaw Diseases

Stomatognathic Diseases

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

Carcinoma, Squamous Cell

Neoplasms, Squamous Cell

Anti-Inflammatory Agents

Antineoplastic Agents

Triamcinolone

Triamcinolone Acetonide

Triamcinolone hexacetonide

Triamcinolone diacetate

Fluorouracil

Tin Fluorides

Antimetabolites

Immunosuppressive Agents

Immunologic Factors

Group assigned to blinded 5-FU (5-fluorouracil) cream applied to face and ears twice daily for maximum of 56 doses

Group assigned to blinded placebo, vehicle control cream applied to face and ears twice daily for maximum of 56 doses

Apply thin layer of topical 5-FU 5% cream twice daily to face and ears for 4 weeks. Treatment to be initiated immediately after randomization. If unable to tolerate the twice daily 5-FU, they will discontinue the treatment and initiate "cool-down" treatment with triamcinolone 0.1% cream twice daily until the symptoms resolve. At 3 weeks after stopping 5-FU, if and only if the participant has not received at least the minimum 2 week (28 dose) course, 5-FU treatment will be resumed on a once-daily basis to complete the 56 dose course. If this is not tolerated, the "cool-down" routine will be followed, but 5-FU will be stopped.

5-fluorouracil

Apply thin layer of vehicle control cream twice daily to face and ears for 4 weeks. Treatment to be initiated immediately after randomization. If unable to tolerate the twice daily vehicle control cream, they will discontinue the treatment and initiate "cool-down" treatment with triamcinolone 0.1% cream twice daily until the symptoms resolve. At 3 weeks after stopping vehicle control cream, if and only if the participant has not received at least the minimum 2 week (28 dose) course, vehicle control cream treatment will be resumed on a once-daily basis to complete the 56 dose course. If this is not tolerated, the "cool-down" routine will be followed, but vehicle control cream will be stopped.

Placebo, vehicle control

Martin A. Weinstock, MD

The main purpose of this study is to see if 5-fluorouracil (5-FU) skin cream can prevent the growth of new skin cancers on the face and ears. The cost of trying to prevent skin cancer will be compared to the usual cost of treating skin cancer. Participants are being asked to be a part of this study because the participants have been treated for two or more skin cancers within the past five (5) years. At least one of these cancers occurred on the face or ears. Having had two or more skins cancers in the past 5 years makes it likely that participants will develop additional skin cancers in the future.

Exposure to ultraviolet radiation from the sun or artificial sources such as tanning beds is a major cause of basal cell and squamous cell carcinoma of the skin. Using lotions, creams, or gels that contain sunscreens can help protect the skin from premature aging and damage that may lead to skin cancer.

The 5-FU skin cream used in this study is FDA-approved to treat some types of skin cancers and spots that might become skin cancer. However, 5-FU skin cream has never been studied to see if it can prevent skin cancer. This drug is not approved by the FDA for how it will be used in this study.

In this study, one half of the patients will use the 5-FU cream and the other half will use a skin cream that looks identical to the 5-FU cream but does not have 5-FU or any other active drug in it.

Approximately twelve VA medical centers will work together in this study. About one thousand (1000) patients will be in this study. The study is sponsored by the U.S. Department of Veterans Affairs Cooperative Studies Program.

Basal cell carcinoma (BCC) and squamous cell carcinoma (SCC) of the skin, both of which are keratinocyte carcinomas (KCs), account for a half of all cancers in the United States, and over 100,000 diagnoses per year in the VA. The standard treatment for these KCs is excision of the lesion, and they cost the US health care system some $2.5 billion annually and about $100 million annually in the VA. There is no proven means to prevent KCs (except perhaps for a modest benefit of intensive daily sunscreen use). An effective prevention strategy would dramatically change the way high-risk patients are managed and could substantially reduce the costs of care. The investigators' preliminary analysis indicates that the savings will be $116 per high-risk patient and will account for a total national savings of over $11 million. These findings imply that the study would pay for itself by the end of 4 years. The investigators hypothesize that topical 5-fluorouracil (5-FU) chemoprevention will prevent skin cancer surgeries and will be cost-saving. To test this the investigators propose a randomized controlled trial of 5-FU compared to a vehicle control to the face and ears in a high-risk population.

In the study, 1000 Veterans at high-risk of skin cancer defined as at least 2 KCs in the prior 5 years, at least one of which was on the face or ears, will be randomized to 5-FU or a vehicle control cream, and followed for 2 to 4 years. The primary endpoint will be surgery for any KC on the face and ears. The investigators will also assess the cost of care, quality of life, the side effects associated with treatment, and the prevalence and number of actinic keratoses, a skin cancer precursor and itself a cause of morbidity and cost. By targeting patients at high-risk, the study focuses on high-cost patients for whom this treatment could both improve outcomes (cancers and quality of life) and reduce costs.

CSP #562 - The VA Keratinocyte Carcinoma Chemoprevention Trial

Diagnosis of the first Primary Basil Cell Carcinoma (BCC) or primary Squamous Cell Carcinoma (SCC) on the face or ears that was removed surgically.

VA Office of Research and Development

Adverse events collected were those related to the study intervention. Data on adverse events were collected spontaneously through patient reports, actively elicited at each clinic visit through open ended questionings and examination, and gathered at the time of telephone contact during the therapy period. All serious adverse events were collected, including those considered related and unrelated to the study interventions.Subjects were monitored for SAE at each study visit and telephone call.

Group assigned to blinded 5-FU (5-fluorouracil) cream applied to face and ears twice daily for maximum of 56 doses

5-fluorouracil: Apply thin layer of topical 5-FU 5% cream twice daily to face and ears for 4 weeks. Treatment to be initiated immediately after randomization. If unable to tolerate the twice daily 5-FU, they will discontinue the treatment and initiate "cool-down" treatment with triamcinolone 0.1% cream twice daily until the symptoms resolve. At 3 weeks after stopping 5-FU, if and only if the participant has not received at least the minimum 2 week (28 dose) course, 5-FU treatment will be resumed on a once-daily basis to complete the 56 dose course. If this is not tolerated, the "cool-down" routine will be followed, but 5-FU will be stopped.

Arm 1: 5-fluorouracil

Group assigned to blinded placebo, vehicle control cream applied to face and ears twice daily for maximum of 56 doses

Placebo, vehicle control: Apply thin layer of vehicle control cream twice daily to face and ears for 4 weeks. Treatment to be initiated immediately after randomization. If unable to tolerate the twice daily vehicle control cream, they will discontinue the treatment and initiate "cool-down" treatment with triamcinolone 0.1% cream twice daily until the symptoms resolve. At 3 weeks after stopping vehicle control cream, if and only if the participant has not received at least the minimum 2 week (28 dose) course, vehicle control cream treatment will be resumed on a once-daily basis to complete the 56 dose course. If this is not tolerated, the "cool-down" routine will be followed, but vehicle control cream will be stopped.

Arm 2: Placebo

Arm 2: Placebo, Vehicle Control

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Martin Weinstock MD, PhD

Group assigned to blinded 5-fluorouracil (5-FU) cream applied to face and ears twice daily for maximum of 56 doses

5-fluorouracil: Apply thin layer of topical 5-FU 5% cream twice daily to face and ears for 4 weeks. Treatment to be initiated immediately after randomization. If unable to tolerate the twice daily 5-FU, they will discontinue the treatment and initiate "cool-down" treatment with triamcinolone 0.1% cream twice daily until the symptoms resolve. At 3 weeks after stopping 5-FU, if and only if the participant has not received at least the minimum 2 week (28 dose) course, 5-FU treatment will be resumed on a once-daily basis to complete the 56 dose course. If this is not tolerated, the "cool-down" routine will be followed, but 5-FU will be stopped.

The Time to Diagnosis of the First Keratinocyte Carcinoma (KC) on the Face or Ears for Which Surgery is Performed

Hazard Ratio for Surgically Treated KC

Overall Study

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Superficial Basal Cell Cancer's Photodynamic Therapy: Comparing Three Photosensitizers: HAL and BF-200 ALA Versus MAL	NCT02367547	Neoplasms, Basa... Carcinoma, Basa... Photochemothera... Photosensitizin...	Hexylaminolevul... Aminolevulinic ... Methylaminolevu...	18 Years -	Joint Authority for Päijät-Häme Social and Health Care
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
CSP #562 - The VA Keratinocyte Carcinoma Chemoprevention Trial	NCT00847912	Carcinoma, Basa... Carcinoma, Squa... Skin Neoplasms Skin Diseases Neoplasms, Basa... Neoplasms, Squa... Carcinoma	5-fluorouracil Placebo, vehicl...	-	VA Office of Research and Development

Spots Global Cancer Trial Database for neoplasms, basal cell