Oral Hedgehog Inhibitors in the Treatment of Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study
NCT ID: NCT05463757
Last Updated: 2025-04-04
Study Results
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Basic Information
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RECRUITING
80 participants
OBSERVATIONAL
2021-11-01
2026-12-31
Brief Summary
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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.
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Detailed Description
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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.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Oral hedgehog inhibitor
Oral hedgehog inhibitors vismodegib (Erivedge) or sonidegib (Odomzo)
Vismodegib
Oral vismodegib (Erivedge), taken daily or every other day (depending on doctor's description)
Sonidegib
Sonidegib (Odomzo), taken daily or every other day (depending on doctor's description)
Interventions
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Vismodegib
Oral vismodegib (Erivedge), taken daily or every other day (depending on doctor's description)
Sonidegib
Sonidegib (Odomzo), taken daily or every other day (depending on doctor's description)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* All ages
* Diagnosed with locally advanced basal cell carcinoma (laBCC), metastatic basal cell carcinoma (mBCC), multiple basal cell carcinomas or Gorlin syndrome
* Use of oral hedgehog inhibitor vismodegib or sonidegib
Exclusion Criteria
ALL
No
Sponsors
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Sun Pharmaceutical Industries Limited
INDUSTRY
Maastricht University Medical Center
OTHER
Responsible Party
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Principal Investigators
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K Mosterd, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Maastricht University Medical Center
Locations
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Radboudumc
Nijmegen, Gelderland, Netherlands
Maastricht University Medical Center +
Maastricht, Limburg, Netherlands
Amsterdam UMC
Amsterdam, North Holland, Netherlands
Netherlands Cancer Institute - AVL
Amsterdam, North Holland, Netherlands
UMCG
Groningen, Provincie Groningen, Netherlands
LUMC
Leiden, South Holland, Netherlands
Erasmus MC
Rotterdam, South Holland, Netherlands
UMC Utrecht
Utrecht, Utrecht, Netherlands
Countries
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Central Contacts
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Facility Contacts
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S Lubeek, MD, PhD
Role: primary
Emmy Cruts, MD
Role: primary
M.W. Bekkenk, MD, PhD
Role: primary
B. Zupan, MD
Role: primary
A.K.L. Reyners, MD, PhD
Role: primary
R. van Doorn, MD, PhD
Role: primary
M. Wakkee, MD, PhD
Role: primary
L.A. Devriese, MD, PhD
Role: primary
References
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Verkouteren BJA, Wakkee M, Reyners AKL, Nelemans P, Aarts MJB, Racz E, Terra JB, Devriese LA, Alers RJ, Kapiteijn E, van Doorn R, Bekkenk MW, Reinders MGHC, Mosterd K. Eight years of experience with vismodegib for advanced and multiple basal cell carcinoma patients in the Netherlands: a retrospective cohort study. Br J Cancer. 2021 Mar;124(7):1199-1206. doi: 10.1038/s41416-020-01220-w. Epub 2021 Jan 19.
Sekulic A, Migden MR, Oro AE, Dirix L, Lewis KD, Hainsworth JD, Solomon JA, Yoo S, Arron ST, Friedlander PA, Marmur E, Rudin CM, Chang AL, Low JA, Mackey HM, Yauch RL, Graham RA, Reddy JC, Hauschild A. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. N Engl J Med. 2012 Jun 7;366(23):2171-9. doi: 10.1056/NEJMoa1113713.
Basset-Seguin N, Hauschild A, Kunstfeld R, Grob J, Dreno B, Mortier L, Ascierto PA, Licitra L, Dutriaux C, Thomas L, Meyer N, Guillot B, Dummer R, Arenberger P, Fife K, Raimundo A, Dika E, Dimier N, Fittipaldo A, Xynos I, Hansson J. Vismodegib in patients with advanced basal cell carcinoma: Primary analysis of STEVIE, an international, open-label trial. Eur J Cancer. 2017 Nov;86:334-348. doi: 10.1016/j.ejca.2017.08.022. Epub 2017 Nov 5.
Dreno B, Kunstfeld R, Hauschild A, Fosko S, Zloty D, Labeille B, Grob JJ, Puig S, Gilberg F, Bergstrom D, Page DR, Rogers G, Schadendorf D. Two intermittent vismodegib dosing regimens in patients with multiple basal-cell carcinomas (MIKIE): a randomised, regimen-controlled, double-blind, phase 2 trial. Lancet Oncol. 2017 Mar;18(3):404-412. doi: 10.1016/S1470-2045(17)30072-4. Epub 2017 Feb 8.
Ben Ishai M, Tiosano A, Fenig E, Ben Simon G, Yassur I. Outcomes of Vismodegib for Periocular Locally Advanced Basal Cell Carcinoma From an Open-label Trial. JAMA Ophthalmol. 2020 Jul 1;138(7):749-755. doi: 10.1001/jamaophthalmol.2020.1539.
Dutch Society for Dermatology and Venereology (NVDV). Basal Cell Carcinoma: Dutch Guideline (Dutch Society for Dermatology and Venereology (NVDV). 2015).
Xie P, Lefrancois P. Efficacy, safety, and comparison of sonic hedgehog inhibitors in basal cell carcinomas: A systematic review and meta-analysis. J Am Acad Dermatol. 2018 Dec;79(6):1089-1100.e17. doi: 10.1016/j.jaad.2018.07.004. Epub 2018 Jul 10.
Sekulic A, Migden MR, Basset-Seguin N, Garbe C, Gesierich A, Lao CD, Miller C, Mortier L, Murrell DF, Hamid O, Quevedo JF, Hou J, McKenna E, Dimier N, Williams S, Schadendorf D, Hauschild A; ERIVANCE BCC Investigators. Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study. BMC Cancer. 2017 May 16;17(1):332. doi: 10.1186/s12885-017-3286-5.
Sekulic A, Migden MR, Lewis K, Hainsworth JD, Solomon JA, Yoo S, Arron ST, Friedlander PA, Marmur E, Rudin CM, Chang AL, Dirix L, Hou J, Yue H, Hauschild A; ERIVANCE BCC investigators. Pivotal ERIVANCE basal cell carcinoma (BCC) study: 12-month update of efficacy and safety of vismodegib in advanced BCC. J Am Acad Dermatol. 2015 Jun;72(6):1021-6.e8. doi: 10.1016/j.jaad.2015.03.021.
Dummer R, Ascierto PA, Basset-Seguin N, Dreno B, Garbe C, Gutzmer R, Hauschild A, Krattinger R, Lear JT, Malvehy J, Schadendorf D, Grob JJ. Sonidegib and vismodegib in the treatment of patients with locally advanced basal cell carcinoma: a joint expert opinion. J Eur Acad Dermatol Venereol. 2020 Sep;34(9):1944-1956. doi: 10.1111/jdv.16230. Epub 2020 Mar 4.
Epstein EH. Basal cell carcinomas: attack of the hedgehog. Nat Rev Cancer. 2008 Oct;8(10):743-54. doi: 10.1038/nrc2503.
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.
Villani A, Costa C, Fabbrocini G, Ruggiero A, Scalvenzi M. Dose reduction during routine treatment of locally advanced basal cell carcinoma with the hedgehog inhibitor sonidegib to manage adverse effects: A retrospective case series. J Am Acad Dermatol. 2021 Apr;84(4):e211-e212. doi: 10.1016/j.jaad.2020.12.006. Epub 2020 Dec 7. No abstract available.
Other Identifiers
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METC 2018-0452-A-10 Vismodegib
Identifier Type: -
Identifier Source: org_study_id
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