Open-Label Proof of Concept Study of VP-315 in Basal Cell Carcinoma

NCT05188729 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: VP-315-201
• Study Type: interventional
• Target: 92
• Locations: 1 country
• Sites: 13

Brief 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). 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).

Conditions

Basal Cell Carcinoma; Skin Cancer; Cancer of the Skin, Basal Cell; Cancer of the Skin; Carcinoma

Keywords

Skin Cancer; BCC; Neoplasm; Epithelial

Outcome Measures

Primary Outcomes (7)

• Part 1: Percentage of Subjects With Discontinuations Due to Adverse Events

  Part 1: Percentage of subjects that discontinued the study due to adverse event

  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)

  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.

  Up to 9 weeks

• Part 2: Percent of Subjects With Adverse Events

  Part 2: Percent of subjects with adverse events, treatment-related AEs

  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.

  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)

  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

  Up to 105 days

Secondary Outcomes (4)

• Part 2: Percentage of Subjects With Clinical Clearance of Treated Lesion(s) at Excision

  Day 84-91

• Part 2: Percentage of Subjects With Histological Clearance of Treated Lesion(s) at Excision

  Day 84-91

• Part 2: Mean Estimated Remaining Tumor Volume at Excision

  Day 84-91

• Part 2 (Cohorts 4 and 5 Expansion Groups): Plasma Concentrations of VP-315

  Day 1-2

Study Arms (11)

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.

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.

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.

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.

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.

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.

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.

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).

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).

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.

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.

Drug: Part 2: VP-315 3 Day Dosing/Week - Split Dose

Interventions

Part 1: VP-315 3 Day Dosing/Week DRUG

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 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 Dose DRUG

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 - 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 Dose DRUG

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 - Cohort 2: Optimal Dosing Regimen of 3 daily doses of VP-315 no loading dose

Part 2: VP-315 2 Day Dosing/Week - Split Dose DRUG

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 - 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 Dose DRUG

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 - Cohort 5: Optimal Dosing Regimen of 3 daily doses of VP-315 8 mg (split dose)

Eligibility Criteria

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

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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:

Sponsors & Collaborators

• Verrica Pharmaceuticals Inc.: lead
• Instat Clinical Research: collaborator
• HeartcoR Solutions: collaborator
• Myonex: collaborator
• Vial Health Technology, Inc: collaborator
• OncoBay Clinical: collaborator
• Q2 Solutions: collaborator
• Canfield Scientific: collaborator
• Veristat: collaborator

Study Sites (13)

Therapeutics Clinical Research

San Diego, California, 92123, United States

Location

ClearlyDerm

Boca Raton, Florida, 33428, United States

Location

Life Clinical Trials

Coral Springs, Florida, 33071, United States

Location

Florida Center for Dermatology

Saint Augustine, Florida, 32080, United States

Location

Gwinnett Dermatology

Snellville, Georgia, 30078, United States

Location

Affinity Health

Oakbrook Terrace, Illinois, 60181, United States

Location

DermAssociates

Rockville, Maryland, 20850, United States

Location

Lawrence J Green, MD LLC

Rockville, Maryland, 20850, United States

Location

ActivMed Research - Borthwick

Portsmouth, New Hampshire, 03801, United States

Location

UPMC St. Margaret

Pittsburgh, Pennsylvania, 15213, United States

Location

Austin Institute of Clinical Research - Dripping Springs

Dripping Springs, Texas, 78620, United States

Location

Austin Institute of Clinical Research - Houston

Houston, Texas, 77056, United States

Location

Austin Institute of Clinical Research - Pflugerville

Pflugerville, Texas, 78660, United States

Location

MeSH Terms

Conditions

Carcinoma, Basal Cell; Skin Neoplasms; Carcinoma; Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Basal Cell; Neoplasms by Site; Skin Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Susan Cutler, VP Medical Affairs
• Organization: Verrica Pharmaceuticals

scutler@verrica.com

484-773-089

Study Officials

• Neal Bhatia, MD

  Therapeutics Clinical Research

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is an open-label, multicenter, dose-escalating study. Eligible subjects will be enrolled sequentially.

Study Record Dates

• First Submitted: December 13, 2021
• First Posted: January 12, 2022
• Study Start: February 1, 2022
• Primary Completion: April 15, 2024
• Study Completion: April 15, 2024
• Last Updated: June 18, 2025
• Results First Posted: June 18, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (13)