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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03534947




Registration number
NCT03534947
Ethics application status
Date submitted
1/05/2018
Date registered
23/05/2018

Titles & IDs
Public title
A Study to Evaluate Neoadjuvant Sonidegib Followed by Surgery or Imiquimod in the Management of Basal Cell Carcinoma
Scientific title
A Pilot Study to Evaluate Short Term Neoadjuvant Sonidegib Followed by Surgery or Imiquimod in the Management of Basal Cell Carcinomas in Cosmetically Challenging Locations
Secondary ID [1] 0 0
HREC file number 18/087
Secondary ID [2] 0 0
MIA2017/CT/220
Universal Trial Number (UTN)
Trial acronym
SONIB
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Basal Cell Carcinoma 0 0
Basal Cell Carcinoma of Skin, Site Unspecified 0 0
Skin Cancer 0 0
Invasive Carcinoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Non melanoma skin cancer
Cancer 0 0 0 0
Kidney

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sonidegib
Treatment: Drugs - Imiquimod
Treatment: Surgery - Surgery
Other interventions - Best supportive care

Experimental: Sonidegib followed by imiquimod - Sonidegib 200mg taken orally once a day for 12 weeks. COMPLETE OR PARTIAL RESPONSE WITH SUPERFICAL REMNANT LESION For patients with a complete response or a partial response resulting in a superficial lesion, treatment with topical imiquimod for 5 days a week for 6 weeks will be prescribed.

Experimental: Sonidegib followed by surgery - Sonidegib 200mg taken orally once a day for 12 weeks. PARTIAL RESPONSE WITH REMNANT INVASIVE LESION For patients with a no change on BCC size / depth or patients with a partial response but a remaining invasive lesion, will have surgical excscion of the remaining lesion.

Other: Sonidegib then best supportive care - Sonidegib 200mg taken orally once a day for 12 weeks. PROGRESSIVE DISEASE Patients with lesions that have progressed in size and/or depth will receive the best supportive care deemed appropriate by the treating clinician. This may be surgery, imiquimod, a clinical trial treatment, radiotherapy or any combination of these interventions.


Treatment: Drugs: Sonidegib
Sonidegib is a selective and orally bioavailable Smoothened (Smo) antagonist. The dose will be 200mg taken once a day for 12 weeks.

Treatment: Drugs: Imiquimod
Imiquimod is an immune response modifier that promotes NF-kappa-B-mediated secretion of pro-inflammatory cytokines, chemokines and other mediators. These immune responses produce cytotoxic effects that are antiproliferative and anti-tumour. Imiquimod treatment requires an extended treatment period of 6 weeks for superficial BCC. Imiquimod is an option for the treatment of small, low-risk superficial BCC when surgery, curettage or cryotherapy are inappropriate. Treatment with imiquimod will be for 5 days a week for a total of 6 weeks.

Treatment: Surgery: Surgery
Although most BCCs are amenable to surgery, excision of large tumours in aesthetically sensitive sites may compromise function or cosmesis. Patients whose BCC has not shrunk in size or depth following 12 weeks of sonidegib will undergo surgical excision of the remaining tumour.

Other interventions: Best supportive care
Patients with lesions that have progressed in size and/or depth will receive the best supportive care deemed appropriate by the treating clinician. This may be surgery, imiquimod, a clinical trial treatment, radiotherapy or any combination of these interventions
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Surgery
Intervention code [3] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Neoadjuvant treatment response determined by optical coherence tomography
Timepoint [1] 0 0
12 weeks
Secondary outcome [1] 0 0
Neoadjuvant treatment response determined by histopathology
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Histologic response to neoadjuvant treatment in basal cell carcinoma sub types
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Response to neoadjuvant treatment in basal cell carcinoma sub types measured with optical coherence tomography.
Timepoint [3] 0 0
12 weeks
Secondary outcome [4] 0 0
Recurrence rate
Timepoint [4] 0 0
6 and 12 months after surgery or at the end of 6 weeks of treatment with imiquimiod.
Secondary outcome [5] 0 0
Drug related adverse reactions
Timepoint [5] 0 0
12 weeks
Secondary outcome [6] 0 0
Discontinuation of sonidegib due to adverse reactions
Timepoint [6] 0 0
12 weeks
Secondary outcome [7] 0 0
Quality of life based on SKINDEX-16
Timepoint [7] 0 0
Weeks 12 and 18 and months 6 and 12.
Secondary outcome [8] 0 0
Patient rated cosmetic outcome after neoadjuvant treatment followed by surgery
Timepoint [8] 0 0
Week 12
Secondary outcome [9] 0 0
Surgeon rated cosmetic outcome after neoadjuvant treatment followed by surgery
Timepoint [9] 0 0
Week 12
Secondary outcome [10] 0 0
Patient rated cosmetic outcome after neoadjuvant treatment
Timepoint [10] 0 0
Week 12
Secondary outcome [11] 0 0
Surgeon rated cosmetic outcome after neoadjuvant treatment
Timepoint [11] 0 0
Week 12

Eligibility
Key inclusion criteria
* =18 years of age.
* Written informed consent.
* Histologically confirmed, resectable, invasive basal cell carcinoma.
* Site and size of BCC considered to be in a cosmetically challenging position for surgery.
* Patient has expressed concerns of the cosmetic outcome of surgery.
* Eastern Cooperative Oncology Group (ECOG) performance status =2.
* Ability to swallow and retain oral medication.
* Anticipated life expectancy of > 12 months.
* Adequate organ function as demonstrated by blood tests.
* Willing to abstain from blood donations for 20 months from the last dose of sonidegib.
* Men with female partner of childbearing potential to use effective contraception from 14 days prior to study treatment until 6 months after the last dose.
* Female patients with active contraception or no menstrual cycle for >12 months
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Inoperable basal cell carcinoma tumours.
* A concurrent cancer diagnosis requiring any systemic anti-cancer therapy.
* Serious or unstable pre-existing medical conditions or other conditions or laboratory abnormalities that could interfere with the patient's safety, consent, or compliance.
* History of malabsorption or other conditions that would interfere with the absorption of sonidegib.
* Known psychiatric or substance abuse disorders that would interfere with cooperation and compliance with the requirements of the trial.
* Prior treatment with hedgehog pathway inhibitors.
* Concomitant medications that may result in increased or decreased bioavailability of sonidegib.
* Patients with neuromuscular disorders (e.g. inflammatory myopathies, muscular dystrophy, rhabdomyolysis, amyotrophic lateral sclerosis and spinal muscular atrophy) due to an increased risk of muscle toxicity with sonidegib.
* Male patients expecting to father children or donate sperm during the 12 weeks of sonidegib treatment and for a further 6 months from the end of treatment.
* Pregnant or breastfeeding women

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/07/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2024
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Melanoma Institute Australia - North Sydney
Recruitment postcode(s) [1] 0 0
2060 - North Sydney

Funding & Sponsors
Primary sponsor type
Other
Name
Melanoma Institute Australia
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pascale Guitera, MD PhD
Address 0 0
Melanoma Institute Australia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Pascale Guitera, MD PhD
Address 0 0
Country 0 0
Phone 0 0
+61299117296
Fax 0 0
Email 0 0
pascale.guitera@melanoma.org.au
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03534947