This Study is to Evaluate the Safety and Pharmacokinetics of SBS-101 in Patients With Oral Premalignant Lesions

NCT03939364 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: SBS-101-CL-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 5

Brief Summary

This study will be the first study investigating the safety, pharmacokinetics (PK), and efficacy of SBS-101 on oral pre-malignant lesions. As such, no clinical data has yet been generated using SBS-101 oral adhesive film.

Conditions

Leukoplakia, Oral

Keywords

Erythroplakia, oral; Erythro-leukoplakia, oral

Outcome Measures

Primary Outcomes (2)

• Percentages of patients with overall response will be summarized by treatment

  Overall response is defined as complete or partial response in either clinical or histological outcome without worsening of either.

  13 weeks

• The percentages of patients reporting any treatment-emergent adverse event (AE) will be tabulated by system organ class and preferred term for each treatment

  13 weeks

Secondary Outcomes (1)

• Plasma concentrations of isotretinoin will be summarized by treatment using descriptive statistics

  13 weeks

Study Arms (4)

0.1% SBS-101

EXPERIMENTAL

Drug: SBS-101

0.3% SBS-101

EXPERIMENTAL

Drug: SBS-101

0.2% SBS-101

EXPERIMENTAL

Drug: SBS-101

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

SBS-101 DRUG

Isotretinoin Oral-Adhesive Film

0.1% SBS-101; 0.2% SBS-101; 0.3% SBS-101

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient is able to comprehend and willing to sign an Informed Consent Form.
• Patient is willing and able to follow all study instructions and to attend all study visits
• Patient is male or female aged 18 years or older at Screening.
• Patient has oral leukoplakia or erythroplakia at least 5mm in length on its longest axis with histopathology showing mild, moderate, or severe dysplasia (index lesion histology score of ≥2) with incisional (punch) biopsy at Screening (or within 30 days prior to Screening).
• Patient agrees to not use any topical therapies (i.e. use of over the counter and medically prescribed topical creams, ointments, or oral rinses) on the index lesion other than the study drug that, in the Investigator's opinion, might influence the status of the index lesion for the duration of the study.
• Patient agrees to not apply any other topical products or use potential irritants (i.e. alcohol containing mouthwash) to the oral premalignant lesions throughout the study. Note: mouthwash that includes non-alcohol containing formulations of Listerine and Crest is acceptable to use during the study.
• If female, patient is non-pregnant, non-lactating and is not planning for pregnancy during the study period or for 1 month after the last dose of study drug.
• Female patients of non-childbearing potential must meet the following requirements:
• Pre-menopausal with documentation of surgical sterilization (i.e., hysterectomy, bilateral tubal ligation, bilateral oophorectomy, or bilateral salpingectomy) at least 3 months prior to study entry.
• Post-menopausal defined as amenorrhea for at least 12 months following cessation of all exogenous hormonal treatments and with follicle-stimulating hormone (FSH) level ≥40 milli-international units per milliliter (mIU/mL) at Screening.
• Female patients of child-bearing potential must select and commit to use 2 forms of effective contraception simultaneously, at least 1 of which must be a primary form, unless absolute abstinence is the chosen method, or the patient has undergone a hysterectomy. Patients must use 2 forms of effective contraception at least 1 month prior to study initiation (Screening), during the study, and for 1 month after the final dose of study drug.
• Primary (hormonal or surgical)
• Tubal ligation
• Partner's vasectomy
• Intrauterine devices

You may not qualify if:

• Patient has oral lesions to be treated that are infected (e.g. lesions that require local or systemic antimicrobial, antifungal, or antiviral therapy to treat an infection) within 14 days of Randomization.
• Patients must not use any systemic (e.g., oral or injectable) corticosteroid therapy during the study or within 30 days prior to Screening. However, use of topical, inhaled, ophthalmic, intraarticular and intralesional steroids is permitted.
• Patient has used any topical or systemic chemotherapeutic product within 30 days prior to Randomization.
• Use of smokeless tobacco within 4 weeks prior to Screening or during the course of the study.
• Patient has been diagnosed with hairy leukoplakia.
• Patient has evidence of a systemic infection or has used systemic antibiotics within 14 days prior to start of dosing for males and females of non-childbearing potential or within 30 days prior to start of dosing for Women of Childbearing Potential (WOCBP).
• Patient is currently taking high doses of vitamin A (\> 25,000 USP units per day) or has previously used isotretinoin for the treatment of another condition within 6 months of Screening.
• Patient is currently taking phenytoin or other prohibited medication
• Patient's index lesion is a recurrent dysplastic lesion in a previously treated area for oral cancer (previous treatment, bed/excision, etc.).
• The patient has received radiation or chemoradiation therapy to oral cavity within 2 years of Randomization.
• The patient has any immune compromising conditions at Screening (i.e. HIV, SLE, etc.) or has been treated with immunomodulating medications within 6 months of Screening.
• Patient currently has poorly controlled diabetes mellitus (uninterrupted hemoglobin A1c \>9% for ≥1 year despite standard care).
• Patient has a history of cardiac, hepatic (ALT and or AST \>2x ULN, Total bilirubin \>1.5x ULN at Visit 1), or renal disease (eGFR\<45 ml/min/1.73 m2) that, in the opinion of the Investigator, might put the patient at undue risk by participating in the study or interferes with the study drug application or the study assessments.
• Patient has a history of sensitivity to any of the active ingredients in the study drug (e.g. isotretinoin, propylene glycol, natural mint), or retinoids, or vitamin A.
• Patient has used oral isotretinoin within 14 days of Screening. Patients must agree to abstain from using oral isotretinoin throughout the duration in the study.

Sponsors & Collaborators

• Skyline Biosciences: lead

Study Sites (5)

University of Alabama at Birmingham (UAB) - The Kirklin Clinic (TKC)

Birmingham, Alabama, 35233, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02120, United States

Location

Carolinas Center for Oral Health

Charlotte, North Carolina, 28209, United States

Location

PennState Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

University of Texas Southwestern Medical Center - Oral & Maxillofacial Surgery Clinic

Dallas, Texas, 75390, United States

Location

MeSH Terms

Conditions

Leukoplakia, Oral; Erythroplasia

Condition Hierarchy (Ancestors)

Mouth Neoplasms; Head and Neck Neoplasms; Neoplasms by Site; Neoplasms; Leukoplakia; Precancerous Conditions; Mouth Diseases; Stomatognathic Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Joy Schleyer

  Skyline Biosciences

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 18, 2019
• First Posted: May 6, 2019
• Study Start: January 1, 2022
• Primary Completion: April 1, 2023
• Study Completion: May 1, 2023
• Last Updated: October 19, 2021

Record last verified: 2021-10

Locations

US (5)