Sonidegib in Adult Patients With Locally Advanced Basal Cell Carcinoma (laBCC) in China

A Prospective, Open-Label, Single-Arm, Multi-Center, Phase IV Efficacy and Safety Study of Sonidegib in Adult Patients With Locally Advanced Basal Cell Carcinoma (laBCC) in China

This is a Phase IV, single arm, multicenter study designed to evaluate the efficacy and safety profile of Sonidegib in chinese participants with locally advanced basal cell carcinoma (laBCC) who are not amenable to radiation therapy, curative surgery or other local therapies.

Study Overview

• Status: Completed
• Conditions: Carcinoma, Basal Cell (BCC)
• Intervention / Treatment: Drug: Sonidegib Phosphate Capsules

• Study Type: Interventional
• Enrollment (Actual): 160
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • China
    • Beijing, China, China, 100034
      • Peking University First Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age 18 years or older.
2. Patients with a histologically confirmed diagnosis of laBCC that is not amenable to radiation therapy, curative surgery, or other local therapies. Patients with laBCC must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension as ≥ 10 mm, with MRI/CT scan or on color photographs.
3. ECOG PS Score ≤ 2.
4. Patients with adequate bone marrow, liver and renal function.
5. Written informed consent obtained prior to any screening procedures.
6. Patients are willing and able to comply with the scheduled visits, treatment plan, laboratory testing, study procedures, and restrictions.
7. Females of childbearing potential agree to use contraception during treatment and for 20 months following the last dose; male patients (even after vasectomy) agree to use contraception during treatment and for 8 months after the last dose.

Exclusion Criteria:

1. Subjects in the opinion of the investigator are immunosuppressed (e.g., organ transplant recipients, HIV, systemic invasive malignancy within past 5 years excluding Stage I cervical cancer, ductal carcinoma in situ of the breast or CLL stage 0, graft vs. host disease, etc.).
2. LaBCC patients with skin lesions in multiple body locations will not be considered metastatic patients. LaBCC patients with positive (histological confirmation of BCC) regional nodal disease will be considered to have metastatic disease.
3. Poorly controlled diabetes mellitus (as per Investigator's discretion in consultation with Sponsor's medical monitor).
4. Major surgery within 4 weeks prior to study treatment.
5. Patients with concurrent uncontrolled medical conditions that may interfere with their participation in the study or potentially affect the interpretation of the study data.
6. Patients unable to take oral drugs or with lack of physical integrity of the upper gastrointestinal tract or known malabsorption syndromes.
7. Patients who have previously been treated with systemic Sonidegib or with other Hh pathway inhibitors.
8. Patients who have neuromuscular disorders (e.g. inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis and spinal muscular atrophy).
9. Patients who are planning on embarking on a new strenuous exercise regimen after initiation of study treatment.
10. Have participated in other clinical trials within 4 weeks prior to the first dose of sonidegib.
11. Have received other anti-neoplastic therapy within 4 weeks prior to study treatment.
12. Patients who are receiving medications known to be moderate and strong inhibitors or inducers of cytochrome P450 (CYP) 3A4/5 or drugs metabolized by CYP2B6 or CYP2C9 that have narrow therapeutic index, and that cannot be discontinued before starting treatment with sonidegib. Medications that are strong CYP3A4/5 inhibitors should be discontinued at least 7 days and strong CYP3A/5 inducers for at least 2 weeks prior to starting treatment with sonidegib.
13. Pregnant or nursing (lactating) women, confirmed by a positive hCG laboratory test (> 5mIU/mL).
14. Unwilling or unable to comply with the protocol.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Sonidegib Phosphate Capsules; Participants will receive continuous treatment with oral administration once a day until conditions for treatment termination are met.	Drug: Sonidegib Phosphate Capsules; Sonidegib should be taken 1 hour before or 2 hours after a breakfast

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR)	Objective response rate (ORR) is defined as the percentage of participants with a confirmed complete or partial response (CR or PR) , per modified Response Evaluation Criteria in Solid Tumors (mRECIST) as assessed by central review.	up to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Duration of Response (DoR)	The DOR for a responder is defined as the time from the participant's initial CR or PR to the first date of either disease progression or death, whichever occurs first. CR, PR and PD will be determined per mRECIST by investigator and central review, respectively	up to 1 year
Complete response rate (CRR) per central and investigator review	CRR is defined as the percentage of participants who achieved a complete response per mRECIST	up to 1 year
Progression-free Survival (PFS)	PFS is defined as the time from the participant's first dose of study treatment to the first date of either disease progression or death, whichever occurs first. Disease progression will be determined per mRECIST by investigator and central review, respectively	up to 1 year
Time to tumor response (TTR)	TTR is defined as the time from the participant's first dose of study treatment to the date of initial CR or PR. CR and PR will be determined per mRECIST by investigator and central review, respectively.	up to 1 year
Adverse event (AE)/Serious adverse event (SAE)/Treatment emergent adverse event (TEAE)	Adverse events, and changes from baseline in vital signs,etc	up to 1 year

Collaborators and Investigators

• Sponsor: Jemincare
• Collaborators: Sun Pharmaceutical Industries Limited; Zhejiang Hangyu Pharmaceutical Co., Ltd.
• Investigators: Principal Investigator: Hang Li, Peking University First Hospital

Study record dates

Study Major Dates

• Study Start (Actual): June 21, 2023
• Primary Completion (Actual): August 26, 2025
• Study Completion (Actual): August 26, 2025

Study Registration Dates

• First Submitted: March 12, 2025
• First Submitted That Met QC Criteria: March 12, 2025
• First Posted (Actual): March 18, 2025

Study Record Updates

• Last Update Posted (Estimated): September 24, 2025
• Last Update Submitted That Met QC Criteria: September 23, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: BCC; Basal Cell Carcinoma
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Basal Cell; Carcinoma; Carcinoma, Basal Cell
• Other Study ID Numbers: AM20220301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No