An Open-Label Study to Evaluate the Safety and Efficacy of EscharEx (EX-02) in the Treatment of Basal Cell Carcinoma
Trial Snapshot
- Phase
- Phase 1
- Sponsor
- MediWound Ltd
- Enrollment
- 32
- Locations
- 3
- Primary Endpoint
- Pain assessments- Proportion of patients with clinical significant elevation in NPRS score
Study Overview
Brief Summary
This study will be a multicenter, prospective, open label, one-arm study intended to assess the Safety and Efficacy of EscharEx (EX-02) in the treatment of Basal Cell Carcinoma.
In patients with one primary superficial or nodular basal cell carcinoma lesion with a diameter of 5-10mm (Histologically confirmed BCC) located on the trunk or upper extremities (not including the hands), with well-defined borders and no previous radiation therapy.
Detailed Description
32 patients are planned to be enrolled into the study from 2-4 US sites. This study will be an Open-Label Study where all patients will be treated with the investigational product EX-02.
Enrollment will be conducted in 2 stages. In the first stage, a group of sixteen (16) patients will be enrolled and treated as detailed below for group 1. Safety results from the first group of patients will be evaluated by the sponsor and by the DSMB. Based on the safety results and DSMB recommendations, the second group of patients will include additional 16 patients that will be enrolled and treated, as detailed below for group 2. All patients will undergo complete surgical excision at the end of the 8 weeks post treatment period and will be followed up until complete clearance is confirmed by biopsy and the wound is closed.
Each patient will go through the following periods during the trial:
- Screening and enrollment period (up to 2 weeks, visit#1):
Screening and enrollment duration will be up to 2 weeks and will include the following:
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Inclusion Criteria- Patient level
- •Male or female greater than age 18,
- •Patients with one primary superficial or nodular basal cell carcinoma lesion (Histologically confirmed BCC) located on the trunk or upper extremities (not including the hands), with well-defined borders and no previous radiation therapy.
- •Lesion is present for no longer than 4 years.
- •Lesion with a diameter of 5-10mm,
- •Patient and/or legally authorized representative (LAR) understands the nature of the procedure, is able and willing to adhere to the protocol regimen, and able to provide a written informed consent prior to any study procedure.
Exclusion Criteria
- •Exclusion Criteria- Patient level
- •Evidence of Gorlin syndrome, neoplastic diseases (except actinic lesions), metastatic tumor or tumor with high probability of metastatic spread,
- •Other malignant cancers (non BCC) of the skin at the lesion's site,
- •Morphea-type basal cell carcinoma (MBCC) at the lesion's site (per biopsy report),
- •Any signs of infection at the lesion site including purulent discharge, tissue abscess, erysipelas, cellulitis, etc.,
- •Patients with any dermatological disease in the target lesion site or surrounding area (not including chronic actinic damage in the surrounding area),
- •History of allergy or atopic disease or a known sensitivity to pineapples, papaya, bromelain or papain, as well as known sensitivity to latex proteins (known as latex-fruit syndrome), bee venom or olive tree pollen,
- •Patients undergoing renal or peritoneal dialysis,
- •Any condition that would preclude safe participation in the study, e.g. evidence of significant or unstable cardiovascular, pulmonary, liver, hematological, immunological, or any immediate life threatening condition,
- •Concurrent acute injury or disease that might compromise the patient's welfare or the participation in the study,
- •Current (within last 12 months) severe alcohol or drug use disorder
- •Pregnant women (positive blood or urine pregnancy test) or nursing mothers,
- •Exposure to investigational intervention within 4 weeks prior to enrolment, or anticipated participation in another investigational drug trial or other intervention trial, while enrolled in the study.
Arms & Interventions
EscharEx 5%
The powder of EX-02 (4 g per vial) should be reconstituted with 10 ml water for injection (WFI) to obtain 5% EX-02 gel. The EX-02 powder and the WFI are to be mixed up to 15 min prior to use. EX-02 5% gel will be topically applied in a thick layer of 2-3 mm on the lesion surface including margin of 5-10 mm for 8-12 hours (preferable over-night) and covered with an occlusive dressing. A new vial should be used for each application. Each patient will be treated with 7 applications.
Intervention: EscharEx 5% (EX-02 formulation) (Drug)
Outcomes
Primary Outcomes
Pain assessments- Proportion of patients with clinical significant elevation in NPRS score
Time Frame: Up to completion of treatment period - up to 5 weeks
Assesed pre 2nd, pre 5th application and post last application compared with pre 1st application
Proportion of subjects discontinuing a treatment due to TEAEs, assessed over the entire course of the treatment.
Time Frame: Up to completion of treatment period - up to 5 weeks
This proportion will be calculated, together with its 95% confidence interval based on the binomial distribution.
Vital signs - proportion of patients with abnormal clinical significant measurements
Time Frame: Up to completion of treatment period - up to 5 weeks
Assessed pre 2nd, pre 5th application and post last application
Incidence and severity of treatment-emergent AEs (TEAEs) and serious TEAEs (STEAEs).
Time Frame: Through study completion - an average of 16 weeks
All adverse events (AEs) will be listed. Adverse events records will include notification whether the AE is local and occurs near or in the target lesion. The AEs will be listed as counts and percentages by System Organ Class (SOC), Preferred Term (PT), severity, and time of onset. Serious Adverse Events will be analyzed in the same way as AEs
Proportion of subjects that requested to discontinue the treatment
Time Frame: Up to completion of treatment period - up to 5 weeks
This will be assessed over the entire course of the treatment and will be calculated, together with its 95% confidence interval based on the binomial distribution
Secondary Outcomes
- Proportion of patients who reached complete histological clearance at the end of the post treatment period.(Up to 13 weeks)
- Proportion of patients who reached complete clearance at the end of the post treatment period, as clinically assessed prior to surgical removal.(Up to 13 weeks)