Dose Escalation Trial to Evaluate Dose Limiting Toxicity/Maximum Tolerated Dose of Microneedle Arrays Containing Doxorubicin (D-MNA) in Basal Cell Carcinoma (BCC)

An Open-Label Dose Escalation Trial to Evaluate Dose Limiting Toxicity (DLT), Maximum Tolerated Dose (MTD), Safety, and Tolerability of Microneedle Arrays Containing Doxorubicin (D-MNA) in Participants With Basal Cell Carcinoma (BCC)

This is a Phase I study in participants with superficial or nodular Basal Cell Carcinoma (BCC), designed to assess dose limiting toxicities and maximum tolerated dose, efficacy, safety, and tolerability of dissolvable, tip-loaded, microneedle arrays containing doxorubicin (D-MNA).

Study Overview

• Status: Terminated
• Conditions: Basal Cell Carcinoma
• Intervention / Treatment: Drug: Placebo-containing MNA; Drug: 25 µg doxorubicin-containing MNA; Drug: 50 µg doxorubicin-containing MNA; Drug: 100 µg doxorubicin-containing MNA; Drug: 200 µg doxorubicin-containing MNA

Detailed Description

This is a Phase I study in participants with superficial or nodular Basal Cell Carcinoma (BCC), designed to assess dose limiting toxicities and maximum tolerated dose, efficacy, safety, and tolerability of dissolvable, tip-loaded, microneedle arrays containing doxorubicin (D-MNA). Doxorubicin is a cytotoxic anthracycline antibiotic and is currently approved for the treatment of a broad range of cancers, including but not limited to: breast, bladder, gastric, and ovarian cancers; small cell lung cancer; acute lymphoblastic leukemia; and acute myelo blastic leukemia. SkinJect, Inc. has developed a novel delivery system in the form of a tip-loaded dissolvable microneedle array (MNA) which will allow for topical delivery of doxorubicin directly to the lesion at concentrations that are far below standard systemic dosing, thereby reducing the adverse events associated with systemic delivery. The primary objective of this investigation is to establish the highest safe and tolerable dose of single applications of D-MNA, one application per week for three weeks in placebo, 25 µg, 50 µg, 100 µg, and 200 µg dose groups in participants with BCC.

• Study Type: Interventional
• Enrollment (Actual): 13
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Aventura, Florida, United States, 33180
      • The Center for Clinical and Cosmetic Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Adult males and females, 40+ years in general good health as assessed by the investigator.
2. BCC (subtype: superficial or nodular) confirmed histologically by diagnostic shave biopsy at the Screening Visit
3. Primary BCC (i.e., no previous treatment)
4. Lesion size ≥ 4 mm2 or 2 x 2 mm and ≤ 169 mm2 or 13 x 13 mm
5. Participant must have no other "clinically significant" abnormal findings in his/her medical history, physical examination or clinical laboratory test results as assessed by the investigator
6. Negative urine pregnancy at study entry for female of child bearing potential
7. Men and women of child-producing potential must agree to use adequate contraception according to standard instructions by the investigator until the completion of the study
8. Participant must to be willing to adhere to the instructions of the investigator and his or her research team
9. Participant must sign an Informed Consent Form prior to any study specific procedures and entry into the study

Exclusion Criteria:

1. Evidence of clinically significant, unstable medical conditions as assessed by the investigator
2. Excisional biopsy performed on the lesion to be treated in this study
3. Recent therapy(ies) to the BCC treatment area
4. Recurrent BCC (previously treated) at the site presented for treatment
5. BCC lesion to be treated is located in an area where excisional biopsy is undesired or aesthetically unacceptable to the participant
6. Previously demonstrated sensitivity to doxorubicin or carboxymethyl cellulose.
7. Participant with other active malignancies with the exception of non-metastatic prostate cancer and carcinoma in situ of the skin and cervix
8. Concomitant disease requiring systemic immunosuppressive treatment
9. Genetic skin cancer disorder, e.g., basal cell nevus syndrome
10. Participant is pregnant or breastfeeding
11. Treatment with another investigational drug, device, or other intervention within 3 months prior to the Screening Visit
12. Existing condition or treatment within 3 months prior to the Screening Visit that may have impact on clinical outcome for the treatment of BCC or delay in wound healing from the elliptical excision

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

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo-containing MNA; Placebo	Drug: Placebo-containing MNA; A 15 x 15 mm array containing 400 tip-loaded, dissolvable microneedles delivering a placebo.; Other Names: Placebo
Experimental: 25 µg Doxorubicin-containing MNA; D-MNA's containing 25 µg of doxorubicin hydrochloride	Drug: 25 µg doxorubicin-containing MNA; A 15 x 15 mm array containing 400 tip-loaded, dissolvable microneedles delivering 25 µg of doxorubicin hydrochloride.; Other Names: 25 µg D-MNA
Experimental: 50 µg Doxorubicin-containing MNA; D-MNA's containing 50 µg of doxorubicin hydrochloride	Drug: 50 µg doxorubicin-containing MNA; A 15 x 15 mm array containing 400 tip-loaded, dissolvable microneedles delivering 50 µg of doxorubicin hydrochloride.; Other Names: 50 µg D-MNA
Experimental: 100 µg Doxorubicin-containing MNA; D-MNA's containing 100 µg of doxorubicin hydrochloride	Drug: 100 µg doxorubicin-containing MNA; A 15 x 15 mm array containing 400 tip-loaded, dissolvable microneedles delivering 100 µg of doxorubicin hydrochloride.; Other Names: 100 µg D-MNA
Experimental: 200 µg Doxorubicin-containing MNA; D-MNA's containing 200 µg of doxorubicin hydrochloride	Drug: 200 µg doxorubicin-containing MNA; A 15 x 15 mm array containing 400 tip-loaded, dissolvable microneedles delivering 200 µg of doxorubicin hydrochloride.; Other Names: 200 µg D-MNA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Dose Limiting Toxicities as Assessed by Local Skin Response (LSR) Grading Scale	Dose Limiting Toxicity (DLT) in Trial Subjects Assessed by Local Skin Response Grading Scale, 0-4, 4 being the worst dermal response	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Complete Response (CR) of Eradicated Basal Cell Carcinoma as Measured by Histological Analysis	Complete Response (CR), defined as histological confirmation by central reading of basal cell carcinoma excision in all study participants	4 weeks

Collaborators and Investigators

• Sponsor: SkinJect, Inc.
• Investigators: Study Director: Gregory Suplick, InClinica, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2020
• Primary Completion (Actual): March 1, 2021
• Study Completion (Actual): May 4, 2021

Study Registration Dates

• First Submitted: August 22, 2018
• First Submitted That Met QC Criteria: August 22, 2018
• First Posted (Actual): August 24, 2018

Study Record Updates

• Last Update Posted (Actual): March 12, 2024
• Last Update Submitted That Met QC Criteria: February 14, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Basal Cell; Carcinoma; Carcinoma, Basal Cell; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Topoisomerase II Inhibitors; Topoisomerase Inhibitors; Antibiotics, Antineoplastic; Doxorubicin; Liposomal doxorubicin
• Other Study ID Numbers: SKNJCT-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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