Investigation of the Safety and Tolerability of BSCT (Anti-nf-P2X7) 10% Ointment

Study Overview
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Brief Summary
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This is a Phase 1, open-label, single-arm, multicenter study to assess the safety and tolerability of BSCT (anti-nf-P2X7) 10% Ointment in subjects with BCC.

Detailed Description
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The purpose of the trial was to determine the safety and tolerability of BSCT (anti-nf-P2X7) 10% Ointment topically applied twice daily for 28 consecutive days in male and female patients with BCC; and to determine the steady-state pharmacokinetics (PK) of the active pharmaceutical ingredient (total sheep Immunoglobulin G [IgG]) when BSCT (anti-nf-P2X7) 10% Ointment is applied twice daily to BCC lesions. This was an open-label, single-arm, multicenter Phase 1 study that enrolled 21 BCC patients.

Official Title
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An Open-Label Phase 1 Investigation of the Safety and Tolerability of BSCT (Anti-nf-P2X7) 10% Ointment When Applied Twice Daily for 28 Days in Male and Female Patients With Basal Cell Carcinoma

Conditions
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Carcinoma, Basal Cell (BCC)

Intervention / Treatment
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* Drug: Treatment with BSCT


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female adults ≥ 18 years of age; One primary histologically confirmed BCC lesion, not located on the hand or foot, suitable for surgical excision, with a minimum area of 0.5 cm2 and a maximum diameter of 2.0 cm (histological diagnosis made no more than 4 weeks prior to the Screening visit. Willing to refrain from using non-approved lotions or creams on the BCC treatment site and surrounding area during the treatment period and from washing the treated area for at least 8 hours following each application of study medication; Ability to follow study instructions and likely to complete all study requirements; Written informed consent obtained, including consent for biopsy tissue to be examined and stored by the central dermatopathologist; Written consent to allow photographs of the BCC lesion to be used as part of the study data; For females of childbearing potential, a negative pregnancy test at Screening and use of an acceptable form of birth control. Exclusion Criteria: Pregnant, lactating, or planning pregnancy during the study; Presence of known or suspected systemic cancer; Histological evidence of squamous cell carcinoma (SCC) or any tumor other than BCC in the biopsy specimen; Histological evidence of severe squamous metaplasia, infiltrative, desmoplastic, or micronodular growth patterns in the biopsy specimen; Evidence of dermatological disease or confounding skin condition within the 25-cm2 treatment area, eg, SCC, actinic keratosis, rosacea, psoriasis, atopic dermatitis, eczema, or xeroderma pigmentosa; Concurrent disease or treatment that suppresses the immune system; Chronic medical condition that in the judgment of the investigator would interfere with the performance of the study or would place the patient at undue risk; Known sensitivity to any of the ingredients in the study medication; Treatment with systemic chemotherapeutic agents (eg, methotrexate, paclitaxel) within the 6 months prior to the Baseline visit; Use of systemic retinoids within the 6 months prior to the Baseline visit; Treatment with systemic immunomodulators or immunosuppressants within the 6 months prior to the Baseline visit; Use of topical immunomodulators within 2 cm of the target treatment area within the 4 weeks prior to the Baseline visit; Treatment with topical agents for the treatment of BCC or actinic keratosis within 2 cm of the target treatment area within the 4 weeks prior to the Baseline visit: Treatment with liquid nitrogen, surgical excision or curettage within 2 cm of the target treatment area during the 4 weeks prior to the Baseline visit; Clinically significant abnormalities as noted in the screening ECG, physical examination, or laboratory test results; Evidence of current chronic alcohol or drug abuse which, in the investigator's opinion, might interfere with the subject's adherence to protocol requirements; Current enrollment in an investigational drug or device study or participation in such a study within 4 weeks of the Baseline visit; In the investigator's opinion, evidence of unwillingness, or inability to follow the restrictions of the protocol and complete the study.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment with BSCT<br>21 patients with BCC were treated with BSCT (anti-nf-P2X7) 10% Ointment topically applied twice daily for 28 consecutive days. | Drug: Treatment with BSCT<br>* The study product BSCT (anti-nf-P2X7) 10% Ointment was anti-nf-P2X7 (highly purified sheep IgG) in an ointment formulation for topical administration. The formulation contained 10% weight by weight of the active pharmaceutical ingredient in an anhydrous ointment base. Fifty (50) to 100 mg of product (an amount the size of a small pea) was applied topically twice a day for 28 days to a 25 cm2 area of skin containing a single BCC lesion. The product was to be applied in the morning and in the evening after washing.<br>* Other names: BSCT (anti-nf-P2X7) 10% Ointment;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) | Adverse events and any changes in physical examinations will be monitored, as described in the Code of Federal Regulations (CFR) Title 21 Part 312. In particular local cutaneous irritation including erythema, peeling, dryness, itching, and burning/ stinging that first occur during the study or represent a worsening from Baseline will be recorded as AEs. | 8 weeks | 
| Pharmacokinetics - Measure Serum Concentration of Total Sheep IgG Using an ELISA. | To determine PK, blood levels of sheep IgG were measured in samples collected at Visit 2 (Baseline), Visit 5, predose at Visit 6 (EOT), and then at 1 h, 2 h, and 4 h after the last dose of study medication. | 28 days | 
| Pharmacokinetics - Measure Subject Antibody Response to the Active Pharmaceutical Ingredient Using an Indirect Fluorescent Immuno Assay. | The active ingredient of BSCT is sheep IgG which may causes an immunogenic response if it enters the systemic circulation. To monitor this response patient blood samples collected at Screening, Visit 2 (Baseline), Visit 6 (EOT), and at Visit 8 (EOS) was tested for anti-sheep IgG antibodies (indicative of immune response against API). | 8 weeks |