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

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ClinicalTrials.gov Identifier: NCT04669808
Recruitment Status : Recruiting
First Posted : December 17, 2020
Last Update Posted : March 26, 2021
Sponsor:
Collaborator:
Amarex Clinical Research
Information provided by (Responsible Party):
Sirnaomics

Tracking Information
First Submitted Date  ICMJE December 9, 2020
First Posted Date  ICMJE December 17, 2020
Last Update Posted Date March 26, 2021
Actual Study Start Date  ICMJE December 28, 2020
Estimated Primary Completion Date April 15, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 9, 2020)
Proportion of participants with histological clearance of treated basal cell carcinoma lesion at the End of Treatment (EOT) [ Time Frame: 6 weeks ]
Histological clearance (HC) will be defined as the absence of detectable evidence of BCC tumor cell nests as determined by central pathology review.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 9, 2020)
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 ]
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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open Label, Dose Escalation Study for the Safety and Efficacy of STP705 in Adult Patients With Basal Cell Carcinoma
Official Title  ICMJE An Open Label, Dose Escalation Study to Evaluate the Safety and Efficacy of Localized Injection of STP705 in Adult Patients With Basal Cell Carcinoma
Brief Summary

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

Participants in the first cohort will attend the study center once weekly for an injection of STP705 into the BCC lesion. The participants will receive injections of STP705 once a week for 6 weeks. 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.

In the absence of dose limiting toxicities (DLT), the subsequent cohorts will receive increasing doses of STP705, following the same schedule of administration as the first cohort.

Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Basal Cell Carcinoma
Intervention  ICMJE Drug: STP705
Dry powder for intra-and peri-lesional injection.
Study Arms  ICMJE
  • Experimental: Cohort A: STP705 30 μg dose
    Cohort A: STP705 30 μg dose, localized injection, given once a week for 6 weeks
    Intervention: Drug: STP705
  • Experimental: Cohort B: STP705 60 μg dose
    Cohort B: STP705 60 μg dose, localized injection, given once a week for 6 weeks
    Intervention: Drug: STP705
  • Experimental: Cohort C: STP705 90 μg dose
    Cohort C: STP705 90 μg dose, localized injection, given once a week for 6 weeks
    Intervention: Drug: STP705
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 9, 2020)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 30, 2021
Estimated Primary Completion Date April 15, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Kush Dhody, MBBS, MS, CCRA (301) 956-2536 kushd@amarexcro.com
Contact: Michael Molyneaux,, MD, MBA MichaelMolyneaux@sirnaomics.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04669808
Other Study ID Numbers  ICMJE SRN-705-006
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Sirnaomics
Study Sponsor  ICMJE Sirnaomics
Collaborators  ICMJE Amarex Clinical Research
Investigators  ICMJE Not Provided
PRS Account Sirnaomics
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP