Treatment Duration Increment and Pharmacodynamic Study of CX-4945 in Patients With Basal Cell Carcinoma (BCC)

NCT03897036 | Completed Phase 1 Results FDA Drug

• 1 other identifier: CX-4945-07
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 6

Brief Summary

This study is to determine the recommended phase II dose (RP2D) and schedule of CX-4945 when administered orally twice daily for 28 consecutive days, in a 4-week (28 days) cycle, in patients with locally advanced or metastatic basal cell carcinoma (BCC). The safety and tolerability of CX-4945, preliminary evidence of antitumor effect, and the effect of CX-4945 treatment on the Hh signaling pathway will also be evaluated in this study.

Conditions

Carcinoma, Basal Cell

Keywords

Advanced Basal Cell Carcinoma; laBCC; Locally Advanced Basal Cell Carcinoma; mBCC; Metastatic Basal Cell Carcinoma

Outcome Measures

Primary Outcomes (1)

• Determination of RP2D

  Determination of RP2D for the expansion cohorts

  Cycle 1, twenty-eight (28) day continuous dosing schedule

Study Arms (2)

Treatment-Duration-Increment

EXPERIMENTAL

CX-4945 capsules at 1000mg BID x 28 days/cycle

Drug: CX-4945

Expansion

EXPERIMENTAL

CX-4945 capsules at 1000mg BID x 28 days/cycle

Drug: CX-4945

Interventions

CX-4945 DRUG

API powder-in-capsule in 200 mg strength

Expansion; Treatment-Duration-Increment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed, written IRB-approved informed consent.
• Men and women age ≥ 18 years
• ECOG Performance status 0 or 1
• For patients with mBCC, histologic confirmation of distant BCC metastasis (e.g., lung, liver, lymph nodes, or bone), with metastatic disease that is RECIST measurable using CT or MRI
• Phase I Expansion:
• If a patient with locally advanced BCC also has a tumor that is not contiguous with cutaneous BCC, e.g., regional lymph nodes (if confirmed on biopsy as BCC and RECIST measurable), the patients should be considered as having mBCC and should be enrolled in the mBCC cohort
• For patients with locally advanced BCC, histologically confirmed disease with at least one lesion that was 10 mm or more in at least 1 dimension by color photograph that is considered to be inoperable or medical contraindication to surgery (see below), in the opinion of a Mohs dermatologic surgeon, head and neck surgeon, or plastic surgeon
• Acceptable medical contraindications to surgery include:
• BCC that has recurred in the same location after two or more surgical procedures and curative resection is deemed unlikely
• Anticipated substantial morbidity and/or deformity from surgery (e.g., removal of all or part of a facial structure, such as nose, ear, eyelid, eye; or requirement for limb amputation)
• Other conditions considered to be medically contraindicating must be discussed with the Medical Monitor before enrolling the patient.
• For all patients, smoothened inhibitor must have been previously administered for their locally advanced or metastatic BCC, unless smoothened inhibitor is inappropriate (e.g., patient has received a smoothened inhibitor but became intolerant to the therapy). For patients whose BCC has been treated with smoothened inhibitor, disease must have progressed after treatment.
• For patients with locally advanced BCC, radiotherapy must have been previously administered for their locally advanced BCC, unless radiotherapy is contraindicated or inappropriate (e.g., hypersensitivity to radiation due to genetic syndrome such as Gorlin syndrome, limitations because of location of tumor, or cumulative prior radiotherapy dose). For patients whose locally advanced BCC has been irradiated, disease must have progressed after radiation.
• Previous Therapy
• Surgery: Previous surgery is permitted provided that a minimum of 28 days (4 weeks) have elapsed between any major surgery and date of registration, and that wound healing has occurred.

You may not qualify if:

• Tumor histology consistent with basosquamous carcinoma (basal cell carcinoma with squamous differentiation or metatypical carcinoma).
• Pregnant or nursing women. NOTE: Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; or abstinence) prior to study entry and for the duration of study participation. Should a man father a child, or a woman become pregnant or suspect she is pregnant while participating in this study, he or she should inform the treating physician immediately.
• Concurrent non-protocol-specified anti-tumor therapy (e.g., chemotherapy, other targeted therapy, radiation therapy, or photodynamic therapy)
• For patients with multiple cutaneous BCCs at baseline that are not designated by the investigator as target lesions, treatment of these non-target BCCs with surgery may be permitted but must be discussed with the Medical Monitor prior to any surgical procedure.
• For patients with locally advanced BCC whose target lesion(s) is/are inoperable at baseline but is/are later deemed potentially operable because of tumor response to CX-4945, surgery with curative intent may be permitted but must be discussed with the Medical Monitor prior to any surgical procedure.
• History of other malignancies within 3 years of Day 1, except for tumors with a negligible risk for metastasis or death, such as adequately treated squamous-cell carcinoma of the skin, ductal carcinoma in situ of the breast, or carcinoma in situ of the cervix
• Active or uncontrolled infections or with serious illnesses or medical conditions which would not permit the patient to be managed according to the protocol.
• History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or renders the patient at high risk from treatment complications
• Difficulty with swallowing oral medications
• Chronic diarrhea (excess of 2-3 stools/day above normal frequency)

Sponsors & Collaborators

• Senhwa Biosciences, Inc.: lead

Study Sites (6)

HonorHealth Research & Innovation Institute

Scottsdale, Arizona, 85258, United States

Location

University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

H. Lee Moffitt Cancer Center & Research Institute, Inc.

Tampa, Florida, 33612, United States

Location

Texas Oncology, P.A.

Dallas, Texas, 75246, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Inova Schar Cancer Institute

Fairfax, Virginia, 22031, United States

Location

MeSH Terms

Conditions

Carcinoma, Basal Cell

Interventions

silmitasertib

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Basal Cell

Results Point of Contact

• Title: Jason Huang, MD / Chief Medical Officer and Acting Chief Executive Officer
• Organization: Senhwa Biosciences

jasonhuang@senhwabio.com

(858) 552-6808

Study Officials

• Jason Huang, MD

  Senhwa Biosciences

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2019
• First Posted: April 1, 2019
• Study Start: April 16, 2019
• Primary Completion: January 25, 2024
• Study Completion: January 25, 2024
• Last Updated: May 20, 2025
• Results First Posted: May 20, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)