Carcinoma, Basal CellPhase 1NCT03897036
Treatment Duration Increment and Pharmacodynamic Study of CX-4945 in Patients With Basal Cell Carcinoma (BCC)
A Phase 1 study testing CX-4945 in people with Carcinoma, Basal Cell, run by Senhwa Biosciences, Inc. at 6 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Carcinoma, Basal Cell
- What is being tested?
- CX-4945
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 6 sitesUnited States
- Study dates
- 16 Apr 2019to 25 Jan 2024
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
- Cytotoxic Chemotherapy: There is no limit to the number of prior regimens received.
- Other Systemic Therapy: Previous treatment with Hh pathway antagonists is not allowed (except for Smoothened inhibitors). There is no limit to the other prior therapies received
- Patients must have recovered (to baseline or ≤ grade 1) from all reversible toxicity related to prior chemotherapy or systemic therapy and have adequate washout as follows:
- Longest of one of the following:
- Two weeks,
- 5 half-lives for investigational agents,
- For anti-cancer therapies with half-lives > 8 days, a washout period of at least 28 days will be acceptable,
- Standard cycle length of standard therapies.
- Patients with nevoid BCC syndrome (Gorlin syndrome) may enroll in this study but must meet the criteria for locally advanced or metastatic disease listed above.
- For patients with locally advanced BCC, willingness to consent to biopsy of tumor(s) at baseline and during the study, as mandated by the protocol
- Adequate hematopoietic capacity, as defined by the following:
- Hemoglobin ≥ 9.0 g/dL and not transfusion dependent
- Platelets ≥ 100,000/mm3
- Absolute neutrophil count ≥ 1500 cells/mm3
- Adequate hepatic function, as defined by the following:
- AST and ALT ≤ 2.5 times upper limit of normal (ULN) or ≤ 5 times ULN if liver metastases are present
- Total bilirubin ≤ 1.5 x ULN or within 3x the ULN for patients with Gilbert disease
- Albumin ≥ 3.0 g/dL
- Adequate renal function, as defined by the following:
- Renal: calculated creatinine clearance >45 mL/min for patients with abnormal, increased, creatinine levels (Cockcroft-Gault formula).
- Women/men of childbearing potential must have agreed to use two effective contraceptive methods while on study and for 6 months after the last dose of CX-4945 (see Appendix D for definition of women of childbearing potential and acceptable and unacceptable methods of contraception)
Who cannot join
- 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
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- 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)
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
Texas Oncology, P.A.
Dallas, Texas, 75246
Inova Schar Cancer Institute
Fairfax, Virginia, 22031
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
HonorHealth Research & Innovation Institute
Scottsdale, Arizona, 85258
H. Lee Moffitt Cancer Center & Research Institute, Inc.
Tampa, Florida, 33612
About this study
Official title: A Phase I, Multi-Center, Open-Label, Treatment Duration Increment, Expansion, Safety, and Pharmacodynamic Study of CX-4945 Administered Orally Twice Daily to Patients With Advanced Basal Cell Carcinoma
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.
Read the detailed description
Basal cell carcinomas require the hedgehog (Hh) pathway for growth. Hh binding relieves the inhibitory effect of PTCH1 on Smoothened (SMO). Signal transduction by SMO then leads to the activation and nuclear localization of GLI1 transcription factors and induction of Hh target genes, many of which are involved in proliferation, survival, and angiogenesis. Hedgehog pathway inhibitors, such as vismodegib6 and sonidegib phosphate, target the G-protein-coupled receptor Smoothened (SMO) and are recommended as first-line treatment for advanced BCC or mBCC by the National Comprehensive Cancer Network. CK2 affects the terminal-most Hh signaling components. Given the roles of CK2 on the terminal step of the hedgehog signaling pathway, CK2 inhibition is unlikely to be overcome by downstream mutations within this pathway. These data thus suggest an immediately practical application of CX-4945 in Hh-driven tumors and possibly tumors resistant to SMO inhibitors.
- Conditions
- Carcinoma, Basal Cell
- Being tested
- CX-4945
- Keywords
- Advanced Basal Cell Carcinoma, laBCC, Locally Advanced Basal Cell Carcinoma, mBCC, Metastatic Basal Cell Carcinoma
- Medical subject headings
- Carcinoma, Basal Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CX-4945Drug
API powder-in-capsule in 200 mg strength
| Study group | Role | What they receive |
|---|---|---|
| Treatment-Duration-Increment | Experimental | CX-4945 capsules at 1000mg BID x 28 days/cycle Receives: Drug: CX-4945 |
| Expansion | Experimental | CX-4945 capsules at 1000mg BID x 28 days/cycle Receives: Drug: CX-4945 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of RP2D
Determination of RP2D for the expansion cohorts
Measured: Cycle 1, twenty-eight (28) day continuous dosing schedule
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2019
- Study start16 Apr 2019
- Primary completion25 Jan 2024
- Study completion25 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Carcinoma, Basal CellStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Senhwa Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jason Huang, MD, Study director, Senhwa Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03897036
- Sponsor's protocol ID
- CX-4945-07
- Status last verified
- May 2025
- First submitted
- 28 Mar 2019
- Last updated
- 20 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multi-Center, Open-Label, Treatment Duration Increment, Expansion, Safety, and Pharmacodynamic Study of CX-4945 Administered Orally Twice Daily to Patients With Advanced Basal Cell Carcinoma
- ClinicalTrials.gov ID
- NCT03897036
- Acronym / sponsor ID
- CX-4945-07
- Conditions
- Carcinoma, Basal Cell
- Keywords
- Advanced Basal Cell Carcinoma, laBCC, Locally Advanced Basal Cell Carcinoma, mBCC, Metastatic Basal Cell Carcinoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Senhwa Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Apr 2019 (actual)
- Primary completion
- 25 Jan 2024 (actual)
- Study completion
- 25 Jan 2024 (actual)
- First posted
- 1 Apr 2019 (actual)
- Results
- Posted 20 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Basal cell carcinomas require the hedgehog (Hh) pathway for growth. Hh binding relieves the inhibitory effect of PTCH1 on Smoothened (SMO). Signal transduction by SMO then leads to the activation and nuclear localization of GLI1 transcription factors and induction of Hh target genes, many of which are involved in proliferation, survival, and angiogenesis. Hedgehog pathway inhibitors, such as vismodegib6 and sonidegib phosphate, target the G-protein-coupled receptor Smoothened (SMO) and are recommended as first-line treatment for advanced BCC or mBCC by the National Comprehensive Cancer Network. CK2 affects the terminal-most Hh signaling components. Given the roles of CK2 on the terminal step of the hedgehog signaling pathway, CK2 inhibition is unlikely to be overcome by downstream mutations within this pathway. These data thus suggest an immediately practical application of CX-4945 in Hh-driven tumors and possibly tumors resistant to SMO inhibitors.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment-Duration-Increment | Experimental | Drug: CX-4945CX-4945 capsules at 1000mg BID x 28 days/cycle |
| Expansion | Experimental | Drug: CX-4945CX-4945 capsules at 1000mg BID x 28 days/cycle |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (36 listed)
- 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
- 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
Key exclusion criteria (10 listed)
- 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)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CX-4945API powder-in-capsule in 200 mg strength | Drug | Expansion, Treatment-Duration-Increment |
Primary outcome
Determination of RP2D
Time frame: Cycle 1, twenty-eight (28) day continuous dosing schedule
Determination of RP2D for the expansion cohorts
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jason Huang, MD study director
Senhwa Biosciences
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CX-4945 in people with Carcinoma, Basal Cell, run by Senhwa Biosciences, Inc. at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 36 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determination of RP2D (time frame: Cycle 1, twenty-eight (28) day continuous dosing schedule). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2025; AzTrials last refreshed its copy on 7 Nov 2026.