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

Phase 1

Terminated

• Conditions: Basal Cell Carcinoma
• Interventions: Drug: 100 µg doxorubicin-containing MNA; Drug: Placebo-containing MNA; Drug: 200 µg doxorubicin-containing MNA; Drug: 25 µg doxorubicin-containing MNA; Drug: 50 µg doxorubicin-containing MNA

• Registration Number: NCT03646188
• Lead Sponsor: SkinJect, Inc.

Brief Summary

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).

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 Timeline

• Study First Submitted: 2018-08-22
• Study First Submitted QC: 2018-08-22
• Study First Posted: 2018-08-24
• Study Start: 2020-06-10
• Primary Completion: 2021-03-01
• Study Completion: 2021-05-04
• Results First Submitted: 2022-01-26
• Results First Submitted QC: 2022-03-30
• Results First Posted: 2022-08-17
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-14
• Last Update Posted: 2024-03-12

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 13

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
100 µg Doxorubicin-containing MNA	100 µg doxorubicin-containing MNA	D-MNA's containing 100 µg of doxorubicin hydrochloride
Placebo-containing MNA	Placebo-containing MNA	Placebo
200 µg Doxorubicin-containing MNA	200 µg doxorubicin-containing MNA	D-MNA's containing 200 µg of doxorubicin hydrochloride
25 µg Doxorubicin-containing MNA	25 µg doxorubicin-containing MNA	D-MNA's containing 25 µg of doxorubicin hydrochloride
50 µg Doxorubicin-containing MNA	50 µg doxorubicin-containing MNA	D-MNA's containing 50 µg of doxorubicin hydrochloride

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

The Center for Clinical and Cosmetic Research; 🇺🇸 Aventura, Florida, United States