SONIDEGIB for Nevoid basal cell carcinoma syndrome | Basal cell carcinoma

Inclusion criteria

• {"criterion_text":"- Be willing and able to provide written informed consent for the trial.\n- Be ≥ 18 years of age on day of signing informed consent.\n- One of the following conditions: - subject with established diagnosis of NBCCS; - subject with common\\sporadic BCC.\n- Clinical stage IIB according to EADO Classification [Very high number of common BCC (>10) or multiple complex BCC (> 5) in the setting of apparently sporadic cases or in Gorlin syndrome. When at least 1 of the multiple BCC can be classified III or IV, the patient will be classified accordingly, and not IIB].\n- Being deemed difficult to treat: not amenable for surgery or RT after the evaluation of a multidisciplinary team involving at least a dermatologist and a medical oncologist and therefore eligible for HHI therapy.\n- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1."}

Exclusion criteria

• {"criterion_text":"- Distant metastatic disease (M1), visceral and/or distant nodal.\n- Prior HHI therapy in the last 2 years.\n- Received previous systemic treatment for BCC; local therapy (e.g. imiquimod) are allowed.\n- Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.\n- Hypersensitivity to sonidegib or any of its excipients."}

Primary endpoints

• {"endpoint_text":"- A discontinuation rate <54% due to AEs at 40 weeks from study entry in a NBCCS - population/sporadic BCC patients in EADO stage IIb treated for 16 weeks with daily sonidegib followed by a pulsed dosing for the subsequent 24 weeks.","definition_or_measurement_approach":"Discontinuation rate due to adverse events measured at 40 weeks from study entry (i.e., proportion of participants who have discontinued treatment because of AEs by week 40)."}

Secondary endpoints

• {"endpoint_text":"- The ORR according to RECIST criteria.\n- The PFS at 40 weeks from study entry.\n- The DOR of a pulsed sonidegib regimen.\n- The rate of any grade drug related (DR) AEs and G3/4 DR AEs of a pulsed sonidegib regimen in a NBCCS - population / sporadic BCC patients in EADO stage IIb after 16 weeks with daily sonidegib.\n- The EORTC QLQ C30 score at baseline, after 16 weeks of continuous daily sonidegib therapy, and after 24 weeks of pulsed sonidegib regimen.","definition_or_measurement_approach":"ORR: objective response rate assessed by RECIST criteria; PFS: progression-free survival measured at 40 weeks from study entry; DOR: duration of response for the pulsed regimen; Safety: rates of any-grade and grade 3/4 drug-related adverse events after 16 weeks of daily sonidegib; QoL: measured using the EORTC QLQ-C30 instrument at baseline, after 16 weeks (continuous therapy) and after 24 weeks (pulsed regimen)."}

Italy

Earliest CTIS Part Ii Submission Date

27-01-2025

Latest Decision Or Authorization Date

18-12-2025

Processing Time Days

325

Number Of Sites

8

Number Of Participants

15

Primary sponsor

Full Name

Gruppo Oncologico Del Nord Ovest

Organisation Type

Patient organisation/association

Country Of Registered Address

Italy

Contract research organisations

Name

Clinical Research Technology S.r.l.

Responsibilities

Sponsor-related duties as listed (sponsorDuties codes: 1,12,5,6,7,8); contact email siblings@cr-technology.com; phone 0039089301545

Third parties

• {"country":"Italy","full_name":"Clinical Research Technology S.r.l.","duties_or_roles":"sponsorDuties codes: 1,12,5,6,7,8; contact email: siblings@cr-technology.com; phone: 0039089301545","organisation_type":"Pharmaceutical company"}