Clinical Trials /

Open Label, Dose Escalation Study for the Safety and Efficacy of STP705 in Adult Patients With Basal Cell Carcinoma

NCT04669808

Description:

This phase 2, open label, dose escalation study is designed to evaluate the safety, tolerability and efficacy of various doses of STP705 administered as localized injection in patients with Basal Cell Carcinoma (BCC). Goals: - To determine the safe and effective recommended dose of STP705 for the treatment of basal cell carcinoma. - Analysis of biomarkers common to BCC formation pathway including TGF-β1 and COX-2.

Related Conditions:
  • Basal Cell Carcinoma
Recruiting Status:

Recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: Open Label, Dose Escalation Study for the Safety and Efficacy of STP705 in Adult Patients With Basal Cell Carcinoma
  • Official Title: An Open Label, Dose Escalation Study to Evaluate the Safety and Efficacy of Localized Injection of STP705 in Adult Patients With Basal Cell Carcinoma

Clinical Trial IDs

  • ORG STUDY ID: SRN-705-006
  • NCT ID: NCT04669808

Conditions

  • Basal Cell Carcinoma

Interventions

DrugSynonymsArms
STP705Cohort A: STP705 30 μg dose

Purpose

This phase 2, open label, dose escalation study is designed to evaluate the safety, tolerability and efficacy of various doses of STP705 administered as localized injection in patients with Basal Cell Carcinoma (BCC). Goals: - To determine the safe and effective recommended dose of STP705 for the treatment of basal cell carcinoma. - Analysis of biomarkers common to BCC formation pathway including TGF-β1 and COX-2.

Detailed Description

      Basal cell carcinoma occurs most often on areas of the skin that are exposed to the sun, such
      as head and neck. The most commonly found clinical feature of Basal Cell Carcinoma (BCC) is
      an elevated tumor with a pearly and translucent margin and telangiectasia. The color may vary
      widely from nearly normal skin color to erythematous to violaceous and may also be pigmented.
      BCC may also resemble noncancerous skin conditions such as eczema or psoriasis. The majority
      of these cancers occur on areas of skin that are regularly exposed to sunlight or other
      ultraviolet radiation.

      The STP705 drug substance (STP705) is composed of two siRNA oligonucleotides, targeting the
      expression of TGF-β1 and Cox-2 mRNA respectively. Along with the HKP-enhanced delivery
      system, the combination is expected to downregulate TGF-β1 and COX-2 expression resulting in
      the inhibition of tumor growth and provide an alternative non-invasive approach for the
      treatment of BCC.

      This phase 2, open label, dose escalation study is designed to evaluate the safety,
      tolerability and efficacy of various doses of STP705 administered as localized injection in
      patients with BCC. This study seeks to establish a safe and effective recommended dose of
      STP705 for the treatment of BCC. The clinician will evaluate the change in tumor size at each
      treatment visit. At the End of Treatment visit, the residual tumor, or former tumor location,
      will be excised for analysis. Expression of biomarkers common to the BCC formation pathway,
      including TGF-β1 and COX-2, will be evaluated.

      Safety and tolerability will be assessed by the number of incidence of adverse events (AEs)
      and serious adverse events (SAEs); the incidence of AEs and SAEs leading to discontinuation
      of trial medication; the incidence and severity of Local Skin Response (LSR);
      hypopigmentation and hyperpigmentation following treatment; and the tolerability of repeated
      localized administration of STP705 as assessed by investigator-evaluation of injection site
      reactions for all patients and within each cohort.

      The study plans to enroll approximately 15 adult patients at up to 3 clinical sites in the
      United States. The 15 patients will be divided equally among 3 cohorts (30, 60 and 90 μg dose
      level) of 5 patients each.
    

Trial Arms

NameTypeDescriptionInterventions
Cohort A: STP705 30 μg doseExperimentalCohort A: STP705 30 μg dose, localized injection, given once a week for 6 weeks
  • STP705
Cohort B: STP705 60 μg doseExperimentalCohort B: STP705 60 μg dose, localized injection, given once a week for 6 weeks
  • STP705
Cohort C: STP705 90 μg doseExperimentalCohort C: STP705 90 μg dose, localized injection, given once a week for 6 weeks
  • STP705

Eligibility Criteria

        Inclusion Criteria: Subjects are required to meet all of the following criteria for
        enrollment into the study:

          1. Male or female adult ≥ 18 years of age.

          2. Primary, histologically confirmed trunk or extremity
             (non-peri-orbital/-anogenital/-facial/-scalp) basal cell carcinoma lesion suitable for
             excision with a minimum diameter of 0.5 cm and with a maximum diameter of 2.0 cm.

          3. Histological diagnosis made no more than 6 months prior to the screening visit.

          4. Histological biopsy removed ≤25% of the original volume of the target lesion.

          5. No other dermatological disease in the BCC target site or surrounding area, which in
             the opinion of the investigator, could interfere with the study.

          6. Willing to refrain from using non-approved lotions or creams on the target site and
             surrounding area during the treatment period.

          7. Willing to refrain from exposure to excessive direct sunlight or ultraviolet light and
             to avoid the use of tanning parlors for the duration of the study.

          8. Laboratory values for the tests (listed in the Study Schedule) within the reference
             ranges as defined by the central laboratory, or "out of range" test results that is
             clinically acceptable to the investigator. Ability to follow study instructions and
             likely to complete all study requirements.

          9. Written informed consent obtained, including consent for tissue to be examined and
             stored by the Central Histology Lab.

         10. Written consent to allow photographs of the target BCC lesion to be used as part of
             the study data and documentation.

         11. For females of childbearing potential, a negative pregnancy test at screening and
             using an acceptable form of birth control (oral / implant/ injectable/ transdermal
             contraceptives, intrauterine device, condom, diaphragm, abstinence, or a monogamous
             relationship with a partner who has had a vasectomy).

        Exclusion Criteria:

          1. Pregnant or lactating.

          2. Presence of known or suspected systemic cancer.

          3. Histological evidence of SCC, or any other non-BCC tumor in the biopsy specimen.

          4. Histological evidence of infiltrative or other aggressive histological subtype growth
             patterns in the biopsy specimen.

          5. History of recurrence of the target BCC lesion.

          6. Evidence of dermatological disease or confounding skin condition with in 2 cm margin
             of the target BCC lesion, e.g., SCC, actinic keratosis, rosacea, psoriasis, atopic
             dermatitis, eczema, xeroderma pigmentosa.

          7. Concurrent disease or treatment that suppresses the immune system;

          8. Patients with baseline QTC > 480 msec using Frederica's formula.

          9. Chronic medical condition that in the judgment of the investigator(s) would interfere
             with the performance of the study or would place the patient at undue risk.

         10. Known sensitivity to any of the ingredients in the study medication.

         11. Use of a tanning beds or other excessive or prolonged exposure to ultraviolet light or
             direct sunlight during the study.

         12. Treatment with systemic chemotherapeutic agents within the 6 months prior to the
             screening visit.

         13. Use of systemic retinoids within the 6 months prior to the screening period.

         14. Treatment with systemic immunomodulators or immunosuppressants within the 6 months
             prior to the screening period.

         15. Use of topical immunomodulators within 2 cm of the target BCC lesion within the 4
             weeks prior to the screening period.

         16. Treatment with the following topical agents within 2 cm of the target BCC lesion
             within the 4 weeks prior to the screening visit: amino-levulinic acid, 5-fluorouracil,
             corticosteroids, retinoids, diclofenac, ingenol mebutate, or imiquimod.

         17. Treatment with liquid nitrogen, surgical excision (excluding diagnostic incisional
             biopsy) or curettage within 2 cm of the target BCC lesion during the 4 weeks prior to
             the screening visit.

         18. Evidence of current chronic alcohol or drug abuse.

         19. Current enrollment in an investigational drug or device study or participation in such
             a study within 4 weeks of the screening visit.

         20. In the investigator's opinion, evidence of unwillingness, or inability to follow the
             restrictions and requirements of the protocol and complete the study.

         21. Taking any other investigational product within 1 month of first dose of STP705.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Proportion of participants with histological clearance of treated basal cell carcinoma lesion at the End of Treatment (EOT)
Time Frame:6 weeks
Safety Issue:
Description:Histological clearance (HC) will be defined as the absence of detectable evidence of BCC tumor cell nests as determined by central pathology review.

Secondary Outcome Measures

Measure:Change in clinical diameter of the treated basal cell carcinoma lesion over the 6 week treatment period
Time Frame:over the 6 week treatment period
Safety Issue:
Description:A base line assessment of lesion clinical diameter will be made by investigator at T1 (first visit). The change in size will be assessed every week until the surgical excision of BCC at the End of Treatment (EOT) visit.

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Not yet recruiting
Lead Sponsor:Sirnaomics

Trial Keywords

  • BCC
  • STP705
  • Basal Cell Carcinoma

Last Updated

December 17, 2020