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

Status

Completed

Phase

Phase 2

Enrollment

92 participants (actual)

Sponsor

Verrica Pharmaceuticals Inc. · Industry

Design

Not randomized · Open label (no masking)

Countries

1 country

United States

Start

1 Feb 2022

Primary completion

15 Apr 2024

Completion

15 Apr 2024

First posted

12 Jan 2022

Last update posted

18 Jun 2025

Conditions

Basal Cell Carcinoma · Skin Cancer · Cancer of the Skin, Basal Cell · Cancer of the Skin · 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).

• Experimental

  Part 1 - Cohort 1: Dose Escalation VP-315 2 mg

  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.

• Experimental

  Part 1 - Cohort 2: Dose Escalation VP-315 3 mg

  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.

• Experimental

  Part 1 - Cohort 3: Dose Escalation VP-315 4 mg

  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.

• Experimental

  Part 1 - Cohort 4: Dose Escalation VP-315 5 mg

  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.

• Experimental

  Part 1 - Cohort 5: Dose Escalation VP-315 6 mg

  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.

• Experimental

  Part 1 - Cohort 6: Dose Escalation VP-315 7 mg

  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.

• Experimental

  Part 1 - Cohort 7: Dose Escalation VP-315 8 mg

  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.

• Experimental

  Part 2 - Cohort 1: Optimal Dosing Regimen of 3 daily doses of VP-315, 4 mg loading dose

  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).

• Experimental

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

  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).

• Experimental

  Part 2 - Cohort 4: Optimal Dosing Regimen of 2 daily doses of VP-315 8 mg (split dose)

  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.

• Experimental

  Part 2 - Cohort 5: Optimal Dosing Regimen of 3 daily doses of VP-315 8 mg (split dose)

  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.

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

   Time frame · Up to 9 weeks

   Registry description

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

2. Part 1: Percentage of Subjects With Dose-limiting Toxicities (DLTs)

   Time frame · Day 4 (Safety Assessment)

   Registry description

   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)

3. Part 1: Percentage of Subjects With Cutaneous Reaction by Maximum Severity

   Time frame · Up to 9 weeks

   Registry description

   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.

4. Part 2: Percent of Subjects With Adverse Events

   Time frame · Up to 15 weeks

   Registry description

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

5. Part 2: Percentage of Subjects With Study Discontinuations Due to Adverse Events

   Time frame · Up to 15 weeks

   Registry description

   Part 2: Percentage of subjects with study discontinuations due to adverse events.

6. Part 2: Percent of Subjects With Treatment Related Adverse Events of Special Interest (TRAEs SI)

   Time frame · Treatment Days up to 2 weeks

   Registry description

   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)

7. Part 2: Percentage of Subjects With Cutaneous Reaction by Maximum Severity

   Time frame · Up to 105 days

   Registry description

   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

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