Sonidegib (Odomzo) neo-adjuvant therapy for participants with Gorlin syndrome – A pilot study

Not Applicable

• Conditions: Gorlin Syndrome; Cancer - Other cancer types; Skin - Other skin conditions; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12620001200976
• Lead Sponsor: Samson Clinical Pty Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-11-12
• Last Update Posted: 16 November 2020

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

1) Males and females participants aged 18 years and above at Screening visit
2) Clinical diagnosis of Gorlin syndrome (Basal Cell Nevus Syndrome). Participants must satisfy the established criteria for diagnosis of Gorlin Syndrome
3) All target lesions (12 lesions) must be biopsied prior to successful enrolment into the study.
4) Participants must not require immediate surgical removal of lesions, or commence radiotherapy
5) No recent treatment for BCCs in the last 4 weeks prior to first dose of study medication
6) Female participants have to be either infertile (WONCBP), not pregnant, lactating, postmenopausal, surgically sterile or using acceptable and highly effective birth control methods.
7) Are willing to comply with all study requirements (primary and secondary treatment phases) including excision, cryotherapy and daily topical treatment of specific lesions.
8) Ability to comprehend and willing to sign and date an informed consent form (ICF).

Exclusion Criteria

1) Have not met the Clinical diagnosis of Gorlin syndrome (Basal Cell Nevus Syndrome). Participants must satisfy the established criteria for diagnosis of Gorlin Syndrome.
2) Insufficient number of BCCs (minimum of 12 lesions)
3) Target tumor biopsy shows evidence of micronodular features, squamous metaplasia, sclerosing BCC, morpheic BCC, or peri-neural involvement
4) Participants receiving medications that are recognized to cause rhabdomyolysis or participants with a prior history of rhabdomyolysis
5) previous use of topical treatment to treat BCCs in the 4 weeks prior to first dose of study drug
6) previous use of photodynamic therapy (PDT), radiation or systemic treatment known to affect BCCs or neoplasm in the 12 weeks prior to first dose of study drug
7) Participants requiring immediate surgical removal of lesions or other related emergency interventions
8) Immunocompromised (e.g. known Hepatitis B or C infection, HIV infection) or is receiving or is expected to receive an immunomodulating agent (including immunosuppressive agents, cytotoxic drugs, biological agents, immunoglobulins, interferon or other immune or cytokine-based therapies.
9) Eastern Cooperative Oncology Group (ECOG) performance status > 1.
10) Known or suspected metastatic disease.
11) Clinically active or uncontrolled skin disease that would interfere with evaluation of the area surrounding the target tumour (e.g. eczema, unstable psoriasis)
12) Any experimental or investigational agents within 6 weeks of first dose of study drug
13) Female participants who are pregnant, planning a pregnancy or nursing a child or not on an acceptable and highly effective birth control method.
14) Female participants who are of child-bearing potential (WOCBP) who are not on 2 methods of contraception (1 highly effective method and 1 barrier method)
15) Any clinically significant abnormal laboratory values as determined by the Principal Investigator.
16) Unwilling to comply with all study procedures and assessments

Study & Design

• Study Type: Interventional
• Study Design: Not specified