Basal Cell CarcinomaPhase 2NCT05188729
Open-Label Proof of Concept Study of VP-315 in Basal Cell Carcinoma
A Phase 2 study testing Part 1: VP-315 3 Day Dosing/Week, Part 2: VP-315 3 Day Dosing/Week - Loading Dose and Part 2: VP-315 3 Day Dosing/Week - No Loading Dose in people with Basal Cell Carcinoma and Skin Cancer, run by Verrica Pharmaceuticals Inc. at 13 sites in United States.
Trial summary
This is a 2-part, open-label, multicenter, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, tolerability, MTD, and objective antitumor efficacy of ascending dose strengths of VP-315 when administered intratumorally to adults with biopsy proven basal cell carcinoma (BCC). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Basal Cell Carcinoma, Skin Cancer, Cancer of the Skin, Basal Cell
- What is being tested?
- Part 1: VP-315 3 Day Dosing/Week, Part 2: VP-315 3 Day Dosing/Week - Loading Dose and Part 2: VP-315 3 Day Dosing/Week - No Loading Dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 92 participants
- Where
- 13 sitesUnited States
- Study dates
- 1 Feb 2022to 15 Apr 2024
- Phase
- Phase 2
- 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
- Adults ≥18 years of age
- Clinically suspected BCC with at least 1 and up to 5 eligible lesion(s) suitable for biopsy and excision
- Willing to refrain from using nonapproved topical agents on, or within 2 cm of, the target BCC lesions and surrounding areas during the treatment period. Subjects should use topical agents that are gentle (eg, Aquaphor, CeraVe) and will not irritate the skin in these areas.
- Willing to refrain from exposure to direct sunlight or ultraviolet light and to avoid the use of tanning parlors for the duration of the study
- Written informed consent obtained, including consent for tissue to be examined by the central dermatopathologist and stored by the Sponsor or designee
- Willing to undergo BCC surgical excision procedure of target and nontarget BCC lesions after study treatment
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- Willing to delay surgical excision of target and nontarget BCC lesions until the end of treatment (EOT) visit
- Provides written consent to allow photographs of the target and nontarget BCC lesion to be used as part of the study data
- Willing to practice a highly effective method of birth control while on study and until 4 weeks after the last treatment. Highly effective birth control includes sexual abstinence, vasectomy, bilateral tubal ligation/occlusion, or a condom with spermicide (men) combined with hormonal birth control or intrauterine device in women.
- BCC Lesion Eligibility
- Eligible lesions are those that meet the BCC lesion eligibility specifications described herein, from samples that are either from:
- HISTORICAL punch or shave biopsies (i.e., samples collected according to clinical standard of care collected within the 90 days prior to W1D1);
- A 2-mm punch biopsy collected within 90 days of W1D1 for suspected BCC ≥0.5 cm to 1.0 cm, and 3-mm punch biopsy for suspected BCC >1.0 cm to 2.0 cm; or
- A shave biopsy performed according to standard of care to include superficial or middle papillary dermis collected within 90 days of W1D1.
- Lesions must meet the following criteria to be eligible for treatment
- BCC Lesion Inclusion Criteria
- For punch biopsies: the size of the lesion(s) must be ≥0.5 cm and </=2 cm in the longest diameter prior to punch biopsy.
- Histological diagnosis of nodular, micronodular, or superficial BCC, as confirmed by punch or shave biopsy performed within 90 days of W1D1. (NOTE: HISTORICAL punch or shave biopsies are acceptable, provided that the biopsy was performed according to clinical standard of care and was collected within the 90 days prior to Screening.) Subject Exclusion Criteria Subjects who meet any of the following exclusion criteria are not to be enrolled in this study.
Who cannot join
- Presence of known or suspected systemic cancer
- Treatment with systemic chemotherapeutic agents within the 6 months prior to the screening visit
- Treatment with systemic immunotherapy, immunomodulators or immunosuppressants within the 12 weeks prior to the screening period
- Genetic or nevoid conditions (eg, Gorlin / basal cell nevus syndrome, xeroderma pigmentosum)
- Clinically significant laboratory values, as assessed by the investigator, for the tests listed in the Schedule of Assessments, including:
- serum creatinine >1.5× the upper limits of normal and
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- serum tryptase concentration >11.4 ng/mL
- Chronic medical condition that in the judgment of the investigator(s) would interfere with the performance of the study or would place the subject at undue risk, such as, but not limited to:
- Uncontrolled infection or infection requiring antibiotics
- Uncontrolled cardiac failure: Classification III or IV New York Heart Association
- Subjects presenting with a systolic BP <110 mmHg and/or diastolic BP <70 mmHg at Screening or Week 1 Day 1 or a history of cerebrovascular or cardiac disorders, or subjects at particular risk of sequelae following a short hypotensive episode.
- Uncontrolled systemic or gastrointestinal inflammatory conditions
- Known bone marrow dysplasia
- History of positive tests for human immunodeficiency virus/acquired immunodeficiency syndrome or active hepatitis B or C
- History of systemic autoimmune disease requiring anti-inflammatory or immunosuppressive therapy within 3 months prior to Day 1, with the following exceptions:
- Subjects with a history of autoimmune thyroiditis are eligible provided the subject requires only thyroid hormone replacement therapy and disease has been stable for ≥1 year
- Subjects with well-controlled type I diabetes (in the opinion of the investigator) are eligible
- Known mast cell activation syndrome, mastocytosis, or chronic idiopathic urticaria
- Known sensitivity to any of the ingredients in the study medication
- Elective surgery within 4 weeks prior to the screening visit, during the study, or 4 weeks after the treatment period
- Evidence of current chronic alcohol or drug abuse
- Current enrollment in an investigational drug or device study or participation in such a study within 4 weeks of the screening visit
- In the investigator's opinion, evidence of unwillingness, or inability to follow the restrictions of the protocol and complete the study
- Females who are pregnant or breastfeeding
- BCC Lesion Exclusion Criteria
- Recurrent or previously treated lesions
- Lesions within 1 cm of the eyelids or lips, or on the hands, feet, ears, nose, and genitalia
- Histological evidence of any other tumor in the biopsy specimen
- Histological evidence of infiltrative, desmoplastic, sclerosing, or morpheaform BCC subtypes in the biopsy specimen
- Medium- and high-risk basal cell carcinomas as defined by the National Comprehensive Cancer Network (NCCN) or Mohs Appropriate Use Criteria (ie, BCCs eligible for Mohs surgery).
- TARGET LESION EXCLUSION ONLY:
- For subjects with severe stasis dermatitis, target BCC lesions may not be on the lower extremities
- Within 2 cm of the target BCC lesion(s):
- Treatment with the following topical agents within the 12 weeks prior to the screening visit: aminolevulinic acid, 5-fluorouracil, corticosteroids, diclofenac, imiquimod, ingenol mebutate
- Treatment with surgical excision, or curettage within the 2 weeks prior to the screening visit
- Evidence of dermatological disease or confounding skin condition that may interfere with clinical evaluation (ie, psoriasis, atopic dermatitis, eczema, propensity to form keloids or hypertrophic scarring)
- Use of topical immunomodulators during study
- Within 5 cm of the target BCC lesion(s): history of any skin cancer, except for other currently identified target and nontarget BCC lesions
- Target BCC lesion is in the area of prior resurfacing procedure with CO2 laser or any photodynamic and phototherapy treatment within the 3 months prior to the screening visit
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
ClearlyDerm
Boca Raton, Florida, 33428
Life Clinical Trials
Coral Springs, Florida, 33071
Austin Institute of Clinical Research - Dripping Springs
Dripping Springs, Texas, 78620
Austin Institute of Clinical Research - Houston
Houston, Texas, 77056
Affinity Health
Oakbrook Terrace, Illinois, 60181
Austin Institute of Clinical Research - Pflugerville
Pflugerville, Texas, 78660
UPMC St. Margaret
Pittsburgh, Pennsylvania, 15213
ActivMed Research - Borthwick
Portsmouth, New Hampshire, 03801
DermAssociates
Rockville, Maryland, 20850
Lawrence J Green, MD LLC
Rockville, Maryland, 20850
Florida Center for Dermatology
Saint Augustine, Florida, 32080
Therapeutics Clinical Research
San Diego, California, 92123
Gwinnett Dermatology
Snellville, Georgia, 30078
About this study
Official title: A Phase 2, Multicenter, Open-label, Proof-of-concept Study With Safety Run-in to Evaluate the Safety, Pharmacokinetics, and Efficacy of VP-315 in Adult Subjects With Basal Cell Carcinoma
This is a 2-part, open-label, multicenter, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, tolerability, MTD, and objective antitumor efficacy of ascending dose strengths of VP-315 when administered intratumorally to adults with biopsy proven basal cell carcinoma (BCC).
The study is expected to enroll approximately 86 subjects with a histological diagnosis of BCC in at least 1 eligible target lesion (confirmed by punch or shave biopsy).
Read the detailed description
This is a 2-part, open-label, multicenter, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, tolerability, maximum tolerated dose (MTD), and objective antitumor efficacy of ascending dose strengths of VP-315 when administered intratumorally to adults with biopsy proven BCC.
The study is expected to enroll approximately 86 subjects with a histological diagnosis of BCC in at least 1 eligible target lesion (confirmed by punch or shave biopsy).
All enrolled subjects will receive VP-315 intradermal injection on an outpatient basis into up to 2 target lesions. In all Parts of the study (1 or 2, as below), each 7-day treatment week comprises up to 3 consecutive treatment days followed by a no-treatment period of at least 4 days. Dosing will commence in a single target lesion. Once a lesion is observed to be fully necrotic (Part 1, Part 2; Cohorts 1-2 only), treatment of that lesion stops, and treatment of subsequent target lesions (up to 2 total) may continue on Day 1 of the following week. In Part 2, Cohorts 4 and 5, treatment of a second target lesion begins on W2D1 (not based on status of necrosis of target lesion 1).
- Conditions
- Basal Cell Carcinoma, Skin Cancer, Cancer of the Skin, Basal Cell, Cancer of the Skin, Carcinoma
- Being tested
- Part 1: VP-315 3 Day Dosing/Week, Part 2: VP-315 3 Day Dosing/Week - Loading Dose, Part 2: VP-315 3 Day Dosing/Week - No Loading Dose, Part 2: VP-315 2 Day Dosing/Week - Split Dose, Part 2: VP-315 3 Day Dosing/Week - Split Dose
- Keywords
- Skin Cancer, BCC, Neoplasm, Epithelial
- Medical subject headings
- Carcinoma, Basal Cell, Skin Neoplasms, Carcinoma, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Part 1: VP-315 3 Day Dosing/WeekDrug
2-8 mg of VP-315 administered via intratumor injection into a single target lesion on W1D1. Each 500-μL dose will be divided into 2 injections given at least 15 minutes and no more than 30 minutes apart, with 30% (150 μL) administered in the first injection and the remaining 70% (350 μL) with the second injection. In all parts of the study, the targeted total volume of delivery is 500 μL daily.
Part 2: VP-315 3 Day Dosing/Week - Loading DoseDrug
4mg (halt the target dose) loading dose on W1D1 administered via intratumor injection into a single target lesion, followed by total daily doses at the full target dose of 8 mg on the remaining days of treatment.
Part 2: VP-315 3 Day Dosing/Week - No Loading DoseDrug
8 mg of VP-315 administered daily via intratumor injection into a single target lesion up to 3 consecutive daily doses/week for up to 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses).
Part 2: VP-315 2 Day Dosing/Week - Split DoseDrug
8 mg of VP-315 administered daily via intratumor injection into a single target lesion up to 2 consecutive daily doses/week for up to 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses).
Part 2: VP-315 3 Day Dosing/Week - Split DoseDrug
8 mg of VP-315 administered daily via intratumor injection into a single target lesion up to 3 consecutive daily doses/week for up to 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses).
| Study group | Role | What they receive |
|---|---|---|
| Part 1 - Cohort 1: Dose Escalation VP-315 2 mg | Experimental | Cohort 1: Starting total daily dose of VP-315 will be 2 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 2 mg on Day 1, 3 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. Receives: Drug: Part 1: VP-315 3 Day Dosing/Week |
| Part 1 - Cohort 2: Dose Escalation VP-315 3 mg | Experimental | Cohort 2: Starting total daily dose of VP-315 will be 3 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 3 mg on Day 1, 4 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. Receives: Drug: Part 1: VP-315 3 Day Dosing/Week |
| Part 1 - Cohort 3: Dose Escalation VP-315 4 mg | Experimental | Cohort 3: Starting total daily dose of VP-315 will be 4 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 4 mg on Day 1, 5 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. Receives: Drug: Part 1: VP-315 3 Day Dosing/Week |
| Part 1 - Cohort 4: Dose Escalation VP-315 5 mg | Experimental | Cohort 4: Starting total daily dose of VP-315 will be 5 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 5 mg on Day 1, 6 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. Receives: Drug: Part 1: VP-315 3 Day Dosing/Week |
| Part 1 - Cohort 5: Dose Escalation VP-315 6 mg | Experimental | Cohort 5: Starting total daily dose of VP-315 will be 6 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 6 mg on Day 1, 7 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. Receives: Drug: Part 1: VP-315 3 Day Dosing/Week |
| Part 1 - Cohort 6: Dose Escalation VP-315 7 mg | Experimental | Cohort 6: Starting total daily dose of VP-315 will be 7 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 7 mg on Day 1, 8 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. Receives: Drug: Part 1: VP-315 3 Day Dosing/Week |
| Part 1 - Cohort 7: Dose Escalation VP-315 8 mg | Experimental | Cohort 7: Starting total daily dose of VP-315 will be 8 mg. Subjects will receive once daily doses of 8 mg for up to 3 days in a 7-day treatment week (e.g., 8 mg on Day 1, 8 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. Receives: Drug: Part 1: VP-315 3 Day Dosing/Week |
| Part 2 - Cohort 1: Optimal Dosing Regimen of 3 daily doses of VP-315, 4 mg loading dose | Experimental | VP-315 once-daily dosing of 8 mg with a loading dose of half the target dose of 8 mg (i.e. 4 mg) only on W1D1; all remaining doses will be the full target dose without a loading dose. Subjects will be treated until the lesion is necrosed, for a maximum of 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses). Receives: Drug: Part 2: VP-315 3 Day Dosing/Week - Loading Dose |
| Part 2 - Cohort 2: Optimal Dosing Regimen of 3 daily doses of VP-315 no loading dose | Experimental | VP-315 once-daily dosing of 8 mg on all treatment days (i.e., NO LOADING dose on W1D1) for up to 3 consecutive daily doses/week until the lesion is necrosed, for a maximum of 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses). Receives: Drug: Part 2: VP-315 3 Day Dosing/Week - No Loading Dose |
| Part 2 - Cohort 4: Optimal Dosing Regimen of 2 daily doses of VP-315 8 mg (split dose) | Experimental | VP-315 once-daily dosing of 8 mg, administered on 2 consecutive days in one week (W1D1, W1D2). The planned dosing regimen will be a split dose of VP-315 for all treatments. The 500μL (8 mg) dose will be divided into 2 injections given at least 15 minutes and no more than 30 minutes apart, with 30% (150 μL) administered in the first injection and the remaining 70% (350 μL) with the second injection. Treatment for a second Target Lesion may begin on D1 of the next week (W2D1, W2D2). Each individual target lesion is treated for the assigned 2 days only (regardless of necrosis status). Up to 2 target lesions may be treated - up to 4 total doses. Receives: Drug: Part 2: VP-315 2 Day Dosing/Week - Split Dose |
| Part 2 - Cohort 5: Optimal Dosing Regimen of 3 daily doses of VP-315 8 mg (split dose) | Experimental | VP-315 once-daily dosing of 8 mg, administered on 3 consecutive days in one week (W1D1, W1D2, W1D3). The planned dosing regimen will be a split dose of VP-315 for all treatments. The 500μL (8 mg) dose will be divided into 2 injections given at least 15 minutes and no more than 30 minutes apart, with 30% (150 μL) administered in the first injection and the remaining 70% (350 μL) with the second injection. Treatment for a second target lesion may begin on D1 of the next week (W2D1, W2D2, W2D3). Each individual target lesion is treated for the assigned 3 days only (regardless of necrosis status). Up to 2 target lesions may be treated - up to 6 total doses. Receives: Drug: Part 2: VP-315 3 Day Dosing/Week - Split Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Percentage of Subjects With Discontinuations Due to Adverse Events
Part 1: Percentage of subjects that discontinued the study due to adverse event
Measured: Up to 9 weeks
Part 1: Percentage of Subjects With Dose-limiting Toxicities (DLTs)
Subjects with pre-determined dose-limiting toxicities such as hypotension (specific criteria); significant elevation of serum tryptase; Grade 2 or higher adverse event (with specific criteria)
Measured: Day 4 (Safety Assessment)
Part 1: Percentage of Subjects With Cutaneous Reaction by Maximum Severity
Cutaneous injection site reactions are defined as the following preferred terms: Injection site erythema, Injection site induration, Injection site swelling, Injection site vesicles, Injection site exfoliation, Injection site erosion, Injection site ulcer, Injection site necrosis.
The intended scale for cutaneous injection site reactions is mild, moderate, severe from CRA.
Subjects having more than one event may appear in more than one System Organ Class or Preferred Term but are counted at most once per each SOC and PT at the maximum severity.
Cutaneous injection site reactions are types of reactions that can be expected to occur.
Measured: Up to 9 weeks
Part 2: Percent of Subjects With Adverse Events
Part 2: Percent of subjects with adverse events, treatment-related AEs
Measured: Up to 15 weeks
Part 2: Percentage of Subjects With Study Discontinuations Due to Adverse Events
Part 2: Percentage of subjects with study discontinuations due to adverse events.
Measured: Up to 15 weeks
Part 2: Percent of Subjects With Treatment Related Adverse Events of Special Interest (TRAEs SI)
Subjects with pre-determined TRAEs of SI such as hypotension (specific criteria); significant elevation of serum tryptase; Grade 2 or higher adverse event (with specific criteria)
Measured: Treatment Days up to 2 weeks
Part 2: Percentage of Subjects With Cutaneous Reaction by Maximum Severity
Evaluation of the tissue condition at the treatment site for the presence and severity of each of the following cutaneous reactions; Erythema, Induration, Swelling, Blister Formation, Desquamation, Erosion, Ulceration, Necrosis by a scale of None; Mild; Moderate; Severe. Subjects having more than one event may appear in more than one SOC or PT but are counted at most once per each SOC and PT at the maximum severity.
Cutaneous injection site reactions are types of reactions that can be expected to occur. The intended scale for cutaneous injection site reactions is mild, moderate, severe from CRA
Measured: Up to 105 days
Secondary outcomes (4)
Part 2: Percentage of Subjects With Clinical Clearance of Treated Lesion(s) at Excision
Clinical clearance of Target Lesion at excision as determined by visual assessment (no residual tumor seen on visual inspection). Clinical assessment using Physician Global Assessment (PGA). PGA scale: 100% Improvement, no visible tumor; 75% to less than 100% improvement, 50% to less than 75% improvement, 25% to less than 50% improvement, Up to 25% improvement, No change, Worse.
Measured: Day 84-91
Part 2: Percentage of Subjects With Histological Clearance of Treated Lesion(s) at Excision
Percentage of Subjects with histological clearance of treated lesion(s) at excision. Histologic clearance confirmed by central dermatopathologist.
Percentage is calculated using the number of subjects with non-missing responses within lesion as the denominator.
*Scar indicates complete histologic clearance
Measured: Day 84-91
Part 2: Mean Estimated Remaining Tumor Volume at Excision
Estimate of remaining tumor volume (necrotic cells:tumor cells) at excision by central dermatopathologist
Scale: 0 = None Remaining to 100 = All Remaining.
Measured: Day 84-91
Part 2 (Cohorts 4 and 5 Expansion Groups): Plasma Concentrations of VP-315
Pharmacokinetics (PK) of an 8 mg dose of VP-315 administered with the optimal dosing regimen
Measured: Day 1-2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2022
- Study start1 Feb 2022
- Primary completion15 Apr 2024
- Study completion15 Apr 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 Basal Cell CarcinomaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- This is an open-label, multicenter, dose-escalating study. Eligible subjects will be enrolled sequentially.
- Masking
- None (open label)
- Enrolment
- 92 participants (actual)
Sponsor and investigators
- Lead sponsor
- Verrica Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Instat Clinical Research, HeartcoR Solutions, Myonex, Vial Health Technology, Inc, OncoBay Clinical, Q2 Solutions, Canfield Scientific Inc., Veristat
- Responsible party
- Sponsor
- Investigators
- Neal Bhatia, MD, Principal investigator, Therapeutics Clinical Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05188729
- Sponsor's protocol ID
- VP-315-201
- Status last verified
- May 2025
- First submitted
- 13 Dec 2021
- Last updated
- 18 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- 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 18 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Open-label, Proof-of-concept Study With Safety Run-in to Evaluate the Safety, Pharmacokinetics, and Efficacy of VP-315 in Adult Subjects With Basal Cell Carcinoma
- ClinicalTrials.gov ID
- NCT05188729
- Acronym / sponsor ID
- VP-315-201
- Conditions
- Basal Cell Carcinoma, Skin Cancer, Cancer of the Skin, Basal Cell, Cancer of the Skin, Carcinoma
- Keywords
- Skin Cancer, BCC, Neoplasm, Epithelial
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 92 participants (actual)
- Lead sponsor
- Verrica Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Collaborators
- Instat Clinical Research, HeartcoR Solutions, Myonex, Vial Health Technology, Inc, OncoBay Clinical, Q2 Solutions, Canfield Scientific Inc., Veristat
- Study start
- 1 Feb 2022 (actual)
- Primary completion
- 15 Apr 2024 (actual)
- Study completion
- 15 Apr 2024 (actual)
- First posted
- 12 Jan 2022 (actual)
- Results
- Posted 18 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a 2-part, open-label, multicenter, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, tolerability, MTD, and objective antitumor efficacy of ascending dose strengths of VP-315 when administered intratumorally to adults with biopsy proven basal cell carcinoma (BCC).
The study is expected to enroll approximately 86 subjects with a histological diagnosis of BCC in at least 1 eligible target lesion (confirmed by punch or shave biopsy).
Detailed description
This is a 2-part, open-label, multicenter, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, tolerability, maximum tolerated dose (MTD), and objective antitumor efficacy of ascending dose strengths of VP-315 when administered intratumorally to adults with biopsy proven BCC.
The study is expected to enroll approximately 86 subjects with a histological diagnosis of BCC in at least 1 eligible target lesion (confirmed by punch or shave biopsy).
All enrolled subjects will receive VP-315 intradermal injection on an outpatient basis into up to 2 target lesions. In all Parts of the study (1 or 2, as below), each 7-day treatment week comprises up to 3 consecutive treatment days followed by a no-treatment period of at least 4 days. Dosing will commence in a single target lesion. Once a lesion is observed to be fully necrotic (Part 1, Part 2; Cohorts 1-2 only), treatment of that lesion stops, and treatment of subsequent target lesions (up to 2 total) may continue on Day 1 of the following week. In Part 2, Cohorts 4 and 5, treatment of a second target lesion begins on W2D1 (not based on status of necrosis of target lesion 1).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 11
- Model description
- This is an open-label, multicenter, dose-escalating study. Eligible subjects will be enrolled sequentially.
| Arm | Type | Receives |
|---|---|---|
| Part 1 - Cohort 1: Dose Escalation VP-315 2 mg | Experimental | Drug: Part 1: VP-315 3 Day Dosing/WeekCohort 1: Starting total daily dose of VP-315 will be 2 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 2 mg on Day 1, 3 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. |
| Part 1 - Cohort 2: Dose Escalation VP-315 3 mg | Experimental | Drug: Part 1: VP-315 3 Day Dosing/WeekCohort 2: Starting total daily dose of VP-315 will be 3 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 3 mg on Day 1, 4 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. |
| Part 1 - Cohort 3: Dose Escalation VP-315 4 mg | Experimental | Drug: Part 1: VP-315 3 Day Dosing/WeekCohort 3: Starting total daily dose of VP-315 will be 4 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 4 mg on Day 1, 5 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. |
| Part 1 - Cohort 4: Dose Escalation VP-315 5 mg | Experimental | Drug: Part 1: VP-315 3 Day Dosing/WeekCohort 4: Starting total daily dose of VP-315 will be 5 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 5 mg on Day 1, 6 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. |
| Part 1 - Cohort 5: Dose Escalation VP-315 6 mg | Experimental | Drug: Part 1: VP-315 3 Day Dosing/WeekCohort 5: Starting total daily dose of VP-315 will be 6 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 6 mg on Day 1, 7 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. |
| Part 1 - Cohort 6: Dose Escalation VP-315 7 mg | Experimental | Drug: Part 1: VP-315 3 Day Dosing/WeekCohort 6: Starting total daily dose of VP-315 will be 7 mg. Subjects will receive ascending once daily doses increasing in 1 mg increments for up to 3 days in a 7-day treatment week (e.g., 7 mg on Day 1, 8 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. |
| Part 1 - Cohort 7: Dose Escalation VP-315 8 mg | Experimental | Drug: Part 1: VP-315 3 Day Dosing/WeekCohort 7: Starting total daily dose of VP-315 will be 8 mg. Subjects will receive once daily doses of 8 mg for up to 3 days in a 7-day treatment week (e.g., 8 mg on Day 1, 8 mg on Day 2) until the first lesion is necrosed or a DLT occurs. Subjects may be treated for a maximum of 2 weeks and a maximum total daily dose of 8 mg. |
| Part 2 - Cohort 1: Optimal Dosing Regimen of 3 daily doses of VP-315, 4 mg loading dose | Experimental | Drug: Part 2: VP-315 3 Day Dosing/Week - Loading DoseVP-315 once-daily dosing of 8 mg with a loading dose of half the target dose of 8 mg (i.e. 4 mg) only on W1D1; all remaining doses will be the full target dose without a loading dose. Subjects will be treated until the lesion is necrosed, for a maximum of 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses). |
| Part 2 - Cohort 2: Optimal Dosing Regimen of 3 daily doses of VP-315 no loading dose | Experimental | Drug: Part 2: VP-315 3 Day Dosing/Week - No Loading DoseVP-315 once-daily dosing of 8 mg on all treatment days (i.e., NO LOADING dose on W1D1) for up to 3 consecutive daily doses/week until the lesion is necrosed, for a maximum of 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses). |
| Part 2 - Cohort 4: Optimal Dosing Regimen of 2 daily doses of VP-315 8 mg (split dose) | Experimental | Drug: Part 2: VP-315 2 Day Dosing/Week - Split DoseVP-315 once-daily dosing of 8 mg, administered on 2 consecutive days in one week (W1D1, W1D2). The planned dosing regimen will be a split dose of VP-315 for all treatments. The 500μL (8 mg) dose will be divided into 2 injections given at least 15 minutes and no more than 30 minutes apart, with 30% (150 μL) administered in the first injection and the remaining 70% (350 μL) with the second injection. Treatment for a second Target Lesion may begin on D1 of the next week (W2D1, W2D2). Each individual target lesion is treated for the assigned 2 days only (regardless of necrosis status). Up to 2 target lesions may be treated - up to 4 total doses. |
| Part 2 - Cohort 5: Optimal Dosing Regimen of 3 daily doses of VP-315 8 mg (split dose) | Experimental | Drug: Part 2: VP-315 3 Day Dosing/Week - Split DoseVP-315 once-daily dosing of 8 mg, administered on 3 consecutive days in one week (W1D1, W1D2, W1D3). The planned dosing regimen will be a split dose of VP-315 for all treatments. The 500μL (8 mg) dose will be divided into 2 injections given at least 15 minutes and no more than 30 minutes apart, with 30% (150 μL) administered in the first injection and the remaining 70% (350 μL) with the second injection. Treatment for a second target lesion may begin on D1 of the next week (W2D1, W2D2, W2D3). Each individual target lesion is treated for the assigned 3 days only (regardless of necrosis status). Up to 2 target lesions may be treated - up to 6 total doses. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Adults ≥18 years of age
- Clinically suspected BCC with at least 1 and up to 5 eligible lesion(s) suitable for biopsy and excision
- Willing to refrain from using nonapproved topical agents on, or within 2 cm of, the target BCC lesions and surrounding areas during the treatment period. Subjects should use topical agents that are gentle (eg, Aquaphor, CeraVe) and will not irritate the skin in these areas.
- Willing to refrain from exposure to direct sunlight or ultraviolet light and to avoid the use of tanning parlors for the duration of the study
- Written informed consent obtained, including consent for tissue to be examined by the central dermatopathologist and stored by the Sponsor or designee
Key exclusion criteria (39 listed)
- Presence of known or suspected systemic cancer
- Treatment with systemic chemotherapeutic agents within the 6 months prior to the screening visit
- Treatment with systemic immunotherapy, immunomodulators or immunosuppressants within the 12 weeks prior to the screening period
- Genetic or nevoid conditions (eg, Gorlin / basal cell nevus syndrome, xeroderma pigmentosum)
- Clinically significant laboratory values, as assessed by the investigator, for the tests listed in the Schedule of Assessments, including:
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 |
|---|---|---|
| Part 1: VP-315 3 Day Dosing/Week2-8 mg of VP-315 administered via intratumor injection into a single target lesion on W1D1. Each 500-μL dose will be divided into 2 injections given at least 15 minutes and no more than 30 minutes apart, with 30% (150 μL) administered in the first injection and the remaining 70% (350 μL) with the second injection. In all parts of the study, the targeted total volume of delivery is 500 μL daily. | Drug | Part 1 - Cohort 1: Dose Escalation VP-315 2 mg, Part 1 - Cohort 2: Dose Escalation VP-315 3 mg, Part 1 - Cohort 3: Dose Escalation VP-315 4 mg, Part 1 - Cohort 4: Dose Escalation VP-315 5 mg, Part 1 - Cohort 5: Dose Escalation VP-315 6 mg, Part 1 - Cohort 6: Dose Escalation VP-315 7 mg, Part 1 - Cohort 7: Dose Escalation VP-315 8 mg |
| Part 2: VP-315 3 Day Dosing/Week - Loading Dose4mg (halt the target dose) loading dose on W1D1 administered via intratumor injection into a single target lesion, followed by total daily doses at the full target dose of 8 mg on the remaining days of treatment. | Drug | Part 2 - Cohort 1: Optimal Dosing Regimen of 3 daily doses of VP-315, 4 mg loading dose |
| Part 2: VP-315 3 Day Dosing/Week - No Loading Dose8 mg of VP-315 administered daily via intratumor injection into a single target lesion up to 3 consecutive daily doses/week for up to 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses). | Drug | Part 2 - Cohort 2: Optimal Dosing Regimen of 3 daily doses of VP-315 no loading dose |
| Part 2: VP-315 2 Day Dosing/Week - Split Dose8 mg of VP-315 administered daily via intratumor injection into a single target lesion up to 2 consecutive daily doses/week for up to 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses). | Drug | Part 2 - Cohort 4: Optimal Dosing Regimen of 2 daily doses of VP-315 8 mg (split dose) |
| Part 2: VP-315 3 Day Dosing/Week - Split Dose8 mg of VP-315 administered daily via intratumor injection into a single target lesion up to 3 consecutive daily doses/week for up to 2 weeks (W1D1, W1D2, W1D3 and W2D1, W2D2, W2D3 - up to 6 total doses). | Drug | Part 2 - Cohort 5: Optimal Dosing Regimen of 3 daily doses of VP-315 8 mg (split dose) |
Primary outcome
Part 1: Percentage of Subjects With Discontinuations Due to Adverse Events
Time frame: Up to 9 weeks
Part 1: Percentage of subjects that discontinued the study due to adverse event
Part 1: Percentage of Subjects With Dose-limiting Toxicities (DLTs)
Time frame: Day 4 (Safety Assessment)
Subjects with pre-determined dose-limiting toxicities such as hypotension (specific criteria); significant elevation of serum tryptase; Grade 2 or higher adverse event (with specific criteria)
Part 1: Percentage of Subjects With Cutaneous Reaction by Maximum Severity
Time frame: Up to 9 weeks
Cutaneous injection site reactions are defined as the following preferred terms: Injection site erythema, Injection site induration, Injection site swelling, Injection site vesicles, Injection site exfoliation, Injection site erosion, Injection site ulcer, Injection site necrosis. The intended scale for cutaneous injection site reactions is mild, moderate, severe from CRA. Subjects having more than one event may appear in more than one System Organ Class or Preferred Term but are counted at most once per each SOC and PT at the maximum severity. Cutaneous injection site reactions are types of reactions that can be expected to occur.
Part 2: Percent of Subjects With Adverse Events
Time frame: Up to 15 weeks
Part 2: Percent of subjects with adverse events, treatment-related AEs
Part 2: Percentage of Subjects With Study Discontinuations Due to Adverse Events
Time frame: Up to 15 weeks
Part 2: Percentage of subjects with study discontinuations due to adverse events.
Part 2: Percent of Subjects With Treatment Related Adverse Events of Special Interest (TRAEs SI)
Time frame: Treatment Days up to 2 weeks
Subjects with pre-determined TRAEs of SI such as hypotension (specific criteria); significant elevation of serum tryptase; Grade 2 or higher adverse event (with specific criteria)
Part 2: Percentage of Subjects With Cutaneous Reaction by Maximum Severity
Time frame: Up to 105 days
Evaluation of the tissue condition at the treatment site for the presence and severity of each of the following cutaneous reactions; Erythema, Induration, Swelling, Blister Formation, Desquamation, Erosion, Ulceration, Necrosis by a scale of None; Mild; Moderate; Severe. Subjects having more than one event may appear in more than one SOC or PT but are counted at most once per each SOC and PT at the maximum severity. Cutaneous injection site reactions are types of reactions that can be expected to occur. The intended scale for cutaneous injection site reactions is mild, moderate, severe from CRA
4 secondary outcomes
Part 2: Percentage of Subjects With Clinical Clearance of Treated Lesion(s) at Excision
Time frame: Day 84-91
Part 2: Percentage of Subjects With Histological Clearance of Treated Lesion(s) at Excision
Time frame: Day 84-91
Part 2: Mean Estimated Remaining Tumor Volume at Excision
Time frame: Day 84-91
Part 2 (Cohorts 4 and 5 Expansion Groups): Plasma Concentrations of VP-315
Time frame: Day 1-2
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 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.
Neal Bhatia, MD principal investigator
Therapeutics Clinical Research
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Part 1: VP-315 3 Day Dosing/Week, Part 2: VP-315 3 Day Dosing/Week - Loading Dose and Part 2: VP-315 3 Day Dosing/Week - No Loading Dose in people with Basal Cell Carcinoma and Skin Cancer, run by Verrica Pharmaceuticals Inc. at 13 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 18 inclusion and 39 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 18 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 18 Jun 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?
Part 1: Percentage of Subjects With Discontinuations Due to Adverse Events (time frame: Up to 9 weeks). The study lists 7 primary outcomes in all. 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 18 Jun 2025.