Safety and Efficacy Evaluation of AIV001 in Nonmelanoma Skin Cancer of the Low Risk Basal Cell Carcinoma Subtype (NMSC)

An Exploratory Study to Evaluate the Safety and Efficacy of AIV001 Intradermally Administered in Subjects With Biopsy-confirmed Basal Cell Carcinoma

To evaluate safety and efficacy of AIV001 treatment on low-risk Nonmelanoma Skin Caner of the basal cell carcinoma subtype.

Study Overview

• Status: Completed
• Conditions: Nodular Basal Cell Carcinoma; Superficial Basal Cell Carcinoma; Nonmelanoma Skin Cancers
• Intervention / Treatment: Drug: AIV001; Drug: AIV001 suspension

Detailed Description

AIV001 (axitinib) has been formulated to provide focal disease-modifying therapy for patients with nonmelanoma skin cancer (NMSC) of the basal cell carcinoma (BCC) subtype. AIV001 targets angiogenesis, inflammation, and fibrosis associated with various pathological skin conditions and was formulated as a simple intradermal/intratumoral injection demonstrating prolonged skin residence. Surgical excision is the standard treatment for NMSC of the basal cell carcinoma subtype for lesions of < 20 mm. Surgical removal of lesions is effective but for some patients unwilling or contraindicated for surgery a nonsurgical option is needed. A nonsurgical option will eliminate post-surgical complications and scarring experienced after lesion removal. Also, specific anatomical location of lesions present cosmesis (i.e., face) or healing challenges (i.e., lower limbs). An effective injectable will benefit patients who are averse to surgery, at risk of wound healing complications or concerned with cosmesis outcomes or fatigued from multiple surgeries. Patient populations (i.e., elderly or patients with diabetes) who are at risk of delayed wound healing would benefit from an injectable option. This study will evaluate injection methods, interval of treatment, four ascending doses, safety, histological clearance and clinical clearance of biopsy-confirmed "low-risk" BCC lesion of <20 mm located on non-facial skin.

• Study Type: Interventional
• Enrollment (Actual): 26
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Encinitas, California, United States, 92024
      • California Dermatology & Clinical Research Institute
    • Newport Beach, California, United States, 92660
      • Island Dermatology
    • San Diego, California, United States, 92117
      • Skin Surgery Medical Group
  • Texas
    • Pflugerville, Texas, United States, 78660
      • Austin Institute for Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female, aged 18 to 80 years, inclusive
2. No clinically relevant abnormalities identified by a detailed medical history and vital signs
3. Presence of a histologically confirmed low risk BCC lesion, with well-defined borders, and with a largest diameter measure before biopsy of 5 mm to 20 mm, located on arms or trunk
4. Histological diagnosis of the target lesion must have been conducted 5 to 30 days prior to Day 1
5. No other dermatological disease within 50 mm of the target lesion at Day 1
6. No prior or concurrent treatment of the target lesion (including radiation therapy)
7. Willing to undergo surgical excision approximately 63 days after first treatment.

Exclusion Criteria:

1. History or presence of systemic cancer
2. Prior radiation treatment at the lesion site or anywhere else on the body within the past 20 years
3. Concurrent disease or treatment that suppresses the immune system (eg, previous organ transplant history, etc.)
4. Clinically relevant cardiovascular, endocrine, hepatic, neurologic, renal, or other major systemic disease that could complicate execution of the protocol or interpretation of the study results.
5. History of thrombotic events, hemorrhagic events, and gastrointestinal perforation and fistula
6. History of recurrence or presence of any other tumor subtype in the target lesion
7. Concurrent presence of a malignant lesion within 100 mm of the target lesion that will require treatment during the study
8. Current enrollment in any other investigational drug or device study within 60 days of Day 1 of this study
9. Evidence of dermatological disease or confounding dermatological condition that would hinder carrying out the study or interpreting the results (eg, atopic dermatitis, eczema, psoriasis, xeroderma pigmentosa, etc.)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AIV001 Treatment Dose 1; Intradermal/intratumoral, Dose 1	Drug: AIV001; Intradermal/intratumoral; Drug: AIV001 suspension; AIV001 is administered as a aqueous suspension by an intradermal/intratumoral injection
Experimental: AIV001 Treatment Dose 2; Intradermal/intratumoral, Dose 2	Drug: AIV001; Intradermal/intratumoral; Drug: AIV001 suspension; AIV001 is administered as a aqueous suspension by an intradermal/intratumoral injection
Experimental: AIV001 Treatment Dose 3; Intradermal/intratumoral, Dose 3	Drug: AIV001; Intradermal/intratumoral; Drug: AIV001 suspension; AIV001 is administered as a aqueous suspension by an intradermal/intratumoral injection
Experimental: AIV001 Treatment Dose 4; Intradermal/intratumoral, Dose 4	Drug: AIV001; Intradermal/intratumoral; Drug: AIV001 suspension; AIV001 is administered as a aqueous suspension by an intradermal/intratumoral injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse events during study period	Incidence of adverse events	Approximately 119 days
Percentage of Histological Clearance achieved of BCC treated lesion	full clearance is no residual BCC cells by histology	Cohort 1-3 Day 63; Cohort 4 Day 105 or Day 126

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Clinical Clearance of treated basal cell carcinoma lesion on skin surface	Number of participants with no surface tumor visible in the study lesion on excision day	Cohort 1-3 Day 63; Cohort 4 Day 105 or Day 126

Collaborators and Investigators

• Sponsor: AiViva BioPharma, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 18, 2020
• Primary Completion (Actual): June 20, 2023
• Study Completion (Actual): July 28, 2023

Study Registration Dates

• First Submitted: July 10, 2020
• First Submitted That Met QC Criteria: July 10, 2020
• First Posted (Actual): July 14, 2020

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 20, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Keywords: BCC; basal cell carcinoma; Nonmelanoma Skin Cancer; Keratinocyte Carcinoma; NMSC
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Skin Diseases; Neoplasms, Basal Cell; Carcinoma; Skin Neoplasms; Carcinoma, Basal Cell
• Other Study ID Numbers: AIV001-C01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No