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A Study to Evaluate Neoadjuvant Sonidegib Followed by Surgery or Imiquimod in the Management of Basal Cell Carcinoma

PHASE2COMPLETED

In this study, patients with BCC will be given neoadjuvant treatment with a drug called sonidegib. Sonidegib is a daily tablet usually given for BCC that cannot be removed by surgery or that has spread through the body. The study aims to see if sonidegib given for 12 weeks will reduce the size of tumours so surgery results in less scarring or may be avoided, with only short term topical treatment required to treat remaining tumour.

Study details:

Surgery is the first line treatment for most basal cell carcinomas (BCCs) with cure rates of 88 to 96%. However, excision of large lesions in sensitive locations such as the face and scalp may result in disfigurement and impairment of function. Neoadjuvant drug treatment given before surgery aims to reduce tumour size so that surgery and recovery is easier.

In this study, patients with BCC will be given neoadjuvant treatment with a drug called sonidegib. Sonidegib is a daily tablet usually given for BCC that cannot be removed by surgery or that has spread through the body. The study aims to see if sonidegib given for 12 weeks will reduce the size of tumours so surgery results in less scarring or may be avoided, with only short term topical treatment required to treat remaining tumour.

This approach may also reduce the risk of recurrence. To assess response to treatment, we will use a new technology for skin tumours called optical coherence technology (OCT). This is like an ultrasound scan and is non invasive.

OCT can detect the extent and nature of the tumour and build a 3D image with great accuracy. OCT will be used together with pathological analysis of tumour tissue to determine response to sonidegib.

Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  1. ≥18 years of age.
  2. Written informed consent.
  3. Histologically confirmed, resectable, invasive basal cell carcinoma.
  4. Site and size of BCC considered to be in a cosmetically challenging position for surgery.
  5. Patient has expressed concerns of the cosmetic outcome of surgery.
  6. Eastern Cooperative Oncology Group (ECOG) performance status ≤2.
  7. Ability to swallow and retain oral medication.
  8. Anticipated life expectancy of > 12 months.
  9. Adequate organ function as demonstrated by blood tests.
  10. Willing to abstain from blood donations for 20 months from the last dose of sonidegib.
  11. 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.
  12. Female patients with active contraception or no menstrual cycle for >12 months

Exclusion criteria

  1. Inoperable basal cell carcinoma tumours.
  2. A concurrent cancer diagnosis requiring any systemic anti-cancer therapy.
  3. Serious or unstable pre-existing medical conditions or other conditions or laboratory abnormalities that could interfere with the patient's safety, consent, or compliance.
  4. History of malabsorption or other conditions that would interfere with the absorption of sonidegib.
  5. Known psychiatric or substance abuse disorders that would interfere with cooperation and compliance with the requirements of the trial.
  6. Prior treatment with hedgehog pathway inhibitors.
  7. Concomitant medications that may result in increased or decreased bioavailability of sonidegib.
  8. 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.
  9. 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.
  10. Pregnant or breastfeeding women

Eligibility

Age eligible for study : NA

Healthy volunteers accepted : No

Gender eligible for study: ALL

Things to know

Study dates

Study start:

Primary completion:

Study completion finish:

study type

Study type

TREATMENT

phase

Phase

    PHASE2

trial

Study ID

NCT03534947

Intervention or treatment

DRUG: Sonidegib

DRUG: Imiquimod

PROCEDURE: Surgery

OTHER: Best supportive care

Conditions

  • Basal Cell Carcinoma
  • Basal Cell Carcinoma of Skin, Site Unspecified
  • Skin Cancer
  • Invasive Carcinoma

  • Condition: Basal Cell Carcinoma, Basal Cell Carcinoma of Skin, Site Unspecified and more

  • DRUG: Sonidegib and other drugs

  • North Sydney, New South Wales, Australia

  • Sponsor: Melanoma Institute Australia

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Are you running this trial?

If you're a clinic or sponsor, you can claim this study

Research sites nearby

Closest Location:

Melanoma Institute Australia

Select from list below to view details:

  • Melanoma Institute Australia

    North Sydney, New South Wales, Australia

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Who is running the clinical trial?

MIA

Melanoma Institute Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/ArmIntervention/Treatment
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.
Drug: Sonidegib, Drug: Imiquimod
    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.
    Drug: Sonidegib, Procedure: Surgery
      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.
      Drug: Sonidegib, Other: Best supportive care

        What is the study measuring?

        Primary Outcome

        MeasureDescriptionTime Frame
        Neoadjuvant treatment response determined by optical coherence tomographyThe size and spread of abnormal skin structures associated with basal cell carcinoma detected by optical coherence tomography.12 weeks

        Secondary Outcome

        MeasureDescriptionTime Frame
        Neoadjuvant treatment response determined by histopathologyThe size and spread of basal cell carcinoma tumour in a biopsy taken from the known worse affected area of disease demonstrated by histopathologic analysis.12 weeks
        Histologic response to neoadjuvant treatment in basal cell carcinoma sub typesThe amount and spread of basal cell carcinoma tumour cells and structures detected at histopathology analysis in nodular and in infiltrative sub types of BCC.12 weeks
        Response to neoadjuvant treatment in basal cell carcinoma sub types measured with optical coherence tomography.The amount and spread of basal cell carcinoma tumour cells and structures detected at histopathology analysis in nodular and in infiltrative sub types of BCC.12 weeks
        Recurrence rateThe number of patients who have a recurrence of basal cell carcinoma at the original site of disease after having surgery or 6 weeks of topical treatment with imiquimod.6 and 12 months after surgery or at the end of 6 weeks of treatment with imiquimiod.
        Drug related adverse reactionsThe proportion of patients experiencing any adverse drug reactions graded according to CTCAE version 4.12 weeks
        Discontinuation of sonidegib due to adverse reactionsThe proportion of patients who discontinue sonideigb because of adverse drug reactions.12 weeks
        Quality of life based on SKINDEX-16The scores obtained from the validated SKINDEX-16 quality of life questionnaire compared to baseline.Weeks 12 and 18 and months 6 and 12.
        Patient rated cosmetic outcome after neoadjuvant treatment followed by surgeryScores from a 3 likert scale questions assessing the patient's agreement with statements about the appearance of their surgical scar for patients who need surgery after neoadjuvant treatment.Week 12
        Surgeon rated cosmetic outcome after neoadjuvant treatment followed by surgeryScores from a 3 likert scale questions assessing the surgeon's agreement with statements about the appearance of their surgical scar for patients who need surgery after neoadjuvant treatment.Week 12
        Patient rated cosmetic outcome after neoadjuvant treatmentScores from a 3 likert scale questions assessing the patient's agreement with statements about the appearance of the remmant BCC area for patients who did not need surgery after neoadjuvant treatment.Week 12
        Surgeon rated cosmetic outcome after neoadjuvant treatmentScores from a 3 likert scale questions assessing the surgeons's agreement with statements about the appearance of the remmant BCC area for patients who did not need surgery after neoadjuvant treatment.Week 12
        Are you running this trial?If you're a clinic or sponsor, you can claim this study.
        Claim this trial

        References

        Clinical Trials Gov: A Study to Evaluate Neoadjuvant Sonidegib Followed by Surgery or Imiquimod in the Management of Basal Cell Carcinoma

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