## Protocol Section
### Identification Module
**NCT ID:** NCT06422936

**Acronym:** SPOTLIGHT204

**Brief Title:** Clinical Trial to Evaluate BO-112 in Patients With Basal Cell Carcinoma (BCC)

**Official Title:** SPOTLIGHT 204: A Multicenter, Phase 2b, Open-label, Non-randomized, Clinical Trial to Evaluate Safety, Tolerability and Preliminary Efficacy of Intra-lesional BO-112 in Patients With Resectable Primary Low and High Risk Basal Cell Carcinoma

#### Organization Study ID Info
**ID:** BOT-112-204
#### Organization
**Class:** INDUSTRY
**Full Name:** Highlight Therapeutics
### Status Module
#### Completion Date
**Date:** 2028-06

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-31

**Type:** ESTIMATED

**Last Update Submit Date:** 2024-05-29

**Overall Status:** RECRUITING

#### Primary Completion Date
**Date:** 2026-06

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-05-27

**Type:** ACTUAL

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-21
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-13
**Study First Submit QC Date:** 2024-05-16
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Highlight Therapeutics

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** This is a multicenter, phase 2b, open-label, non-randomized, clinical trial to evaluate safety, tolerability, pharmacodynamics and preliminary efficacy of intra-lesional BO-112 in patients with resectable primary low and high risk basal cell carcinoma.

* primary endpoint is complete visual and pathological response \[at surgery\] on patient level assessed by central review
* secondary endpoints are

  1. Occurrence of adverse events (AEs), serious adverse events (SAEs), and AEs leading to discontinuation or death on patient level.
  2. Pathological response \[at surgery\] on patient level assessed by the investigator and central review, respectively, and visual response \[during the study and at surgery\] on patient level assessed by the investigator and central review, respectively.
  3. Recurrence \[at 12 and 24 months\] after surgery on patient level assessed by the investigator.

### Conditions Module

**Conditions:**

- Basal Cell Carcinoma

### Design Module

#### Design Info

**Allocation:** NON_RANDOMIZED

**Intervention Model:** PARALLEL

**Intervention Model Description:** Parallel group design with same treatment and study procedures

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 60

**Type:** ESTIMATED

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** patients with low risk nodular BCC

**Intervention Names:**

- Drug: BO-112

**Label:** Cohort I

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** patients with high risk BCC

**Intervention Names:**

- Drug: BO-112

**Label:** Cohort II

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Cohort I

- Cohort II

**Description:** Noncoding double-stranded (ds) RNA based on polyinosinic-polycytidylic acid (Poly I:C), formulated with polyethylenimine (PEI) for intra-lesional injection

**Name:** BO-112

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Complete visual and pathological response of all treated BCC lesions on participant level assessed by central review

**Measure:** Number of Participants with Complete Visual and Pathological Response

**Time Frame:** at 24 weeks

#### Secondary Outcomes

**Description:** Occurrence of adverse events (AEs), serious adverse events (SAEs), and AEs leading to discontinuation or death on participant level.

**Measure:** Number of Participants with Treatment-Related Adverse Events assessed by CTCAE 5.0

**Time Frame:** through study completion, average 7 months

**Description:** Pathological response on participant level assessed by the investigator and central review, respectively, using a 4-point scale

1. Pathologic Complete Response (pCR): No Residual Viable Tumor (RVT)
2. Major Pathologic Response (MPR)/ "near-pCR": \<= 10% RVT
3. Pathologic Partial Response (pPR): 10% \< RVT \<= 50% "
4. Pathologic Non-Response" (pNR): \>50% RVT

**Measure:** Extent of Pathological Response

**Time Frame:** at 24 weeks

**Description:** Visual response \[during the study and at surgery\] on participant level assessed by the investigator and central review, respectively.

**Measure:** Change of Visual Lesion Appearance

**Time Frame:** at baseline and at 24 weeks

**Description:** Recurrence after surgery on participant level assessed by the investigator

**Measure:** Number of Participants with BCC Recurrence

**Time Frame:** at 12 and 24 months after surgery

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

1. Participant must be ≥ 18 years old \[or the legal age of consent in the jurisdiction in which the study is taking place\], at the time of signing the informed consent.

   Type of Participant and Disease Condition
2. Has primary resectable low or high risk basal cell carcinoma according to the protocol definition
3. Has diagnostic punch biopsy of all lesions intended for injection available prior to the first dose of BO-112.
4. Has adequate organ function defined as defined per protocol
5. Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin \[HCG\]) within 24 hours prior to the start of study drug.
6. Women of childbearing potential must be willing to use two effective methods of birth control while treated with BO-112 and for 4 weeks after the last treatment. The two forms of birth control authorized are defined as the use of a barrier method of contraception (condom with spermicide) in association with one of the following methods of birth control: bilateral tubal ligation; combined oral contraceptives (estrogens and progesterone) or implanted or injectable contraceptives from the time of informed consent.
7. Male patients with female partners of childbearing potential must be willing to use two adequate contraception methods while treated with BO-112 and for 4 weeks after treatment completion.

   Informed Consent
8. Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
9. Able and willing to comply with all study requirements, including surgical removal of lesion/lesion site at completion of study.

Exclusion Criteria:

1. Has history of hypersensitivity to BO-112 or its excipients/vehicle. 2. Has any BCC lesion(s) in site of prior radiation and/or any BCC lesion(s) within 2 cm of the open eyelid margins 4. Has Gorlin's syndrome 5. Has clinically active or uncontrolled skin disease or tattoos that would interfere with evaluation of the area surrounding the target lesion 6. Has another malignant disease requiring treatment 7. Has a history of immunological disorder, severe allergic reaction, moderate or severe asthma or known history of anaphylaxis or any other serious adverse reactions to the investigational products.

8. Female participants: lactating or pregnant. 9. Has received a live vaccine or messenger ribonucleic acid (mRNA) Corona virus disease (COVID) vaccine within 7 days prior to the first dose of study drug or has a vaccination planned during treatment with BO-112 and within 7 days after the last study drug administration.

10. Is immunocompromised. Systemic corticosteroids at \>10 mg/day prednisone or equivalent within 1 week prior to the first dose of BO-112.

11. Has any prior systemic anti-lesion therapy or local treatment for study lesions prior to first dose; any chemotherapy or immunotherapy for any other malignancy within 24 months prior to the first dose of BO-112.

12. Has any experimental or investigational agents within one month of first BO-112 injection.

13. Has received or is expected to receive treatment with psoralen plus ultraviolet A (UVA) or ultraviolet B (UVB) therapy within 6 months prior to the first dose of BO-112.

14. Requires / or has used topical products within 5 cm of a treatment-targeted BCC lesion or systemic therapies that might interfere with the evaluation of the study medication during the study.

15. Has any other concurrent anti-cancer therapy (chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy considered investigational \[used for a not approved indication and in the context of a research investigation\]) within 28 days of first study drug administration; or plans to participate in an experimental drug study while enrolled in this study.

16. Has any medical contraindications to surgery

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** mquintero@highlighttherapeutics.com

**Name:** Marisol Quintero, PhD

**Phone:** +34 682 544 814

**Role:** CONTACT

**Contact 2:**

**Email:** zjirakova@highlighttherapeutics.com

**Name:** Zuzana Jirakova Trnkova, MD, PhD

**Phone:** +34 682 544 814

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Be'er Sheva

**Contacts:**

***Contact 1:***

- **Name:** Adi Saadia, MD

- **Role:** CONTACT

**Country:** Israel

**Facility:** Soroka Medical Center

**Status:** RECRUITING

**Location 2:**

**City:** Haifa

**Contacts:**

***Contact 1:***

- **Name:** Michal Ramon, MD

- **Role:** CONTACT

**Country:** Israel

**Facility:** Rambam Medical Center

**Status:** NOT_YET_RECRUITING

**Location 3:**

**City:** Jerusalem

**Contacts:**

***Contact 1:***

- **Name:** Sharon Merims, MD

- **Role:** CONTACT

**Country:** Israel

**Facility:** Hadassah Ein Kerem Medical Center

**Status:** NOT_YET_RECRUITING

**Location 4:**

**City:** Tel Aviv

**Contacts:**

***Contact 1:***

- **Name:** Gila Isman-Nelkenbaum, MD

- **Role:** CONTACT

**Country:** Israel

**Facility:** Sourasky Medical Center

**Status:** NOT_YET_RECRUITING

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000009375

- Term: Neoplasms, Glandular and Epithelial

- ID: D000009370

- Term: Neoplasms by Histologic Type

- ID: D000009369

- Term: Neoplasms

- ID: D000018295

- Term: Neoplasms, Basal Cell

### Condition Browse Module - Browse Branches

- Abbrev: BC04

- Name: Neoplasms

- Abbrev: All

- Name: All Conditions

### Condition Browse Module - Browse Leaves

- ID: M5534

- Name: Carcinoma

- Relevance: HIGH

- As Found: Carcinoma

- ID: M5537

- Name: Carcinoma, Basal Cell

- Relevance: HIGH

- As Found: Basal Cell Carcinoma

- ID: M12320

- Name: Neoplasms, Glandular and Epithelial

- Relevance: LOW

- As Found: Unknown

- ID: M12315

- Name: Neoplasms by Histologic Type

- Relevance: LOW

- As Found: Unknown

- ID: M20439

- Name: Neoplasms, Basal Cell

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000002277

- Term: Carcinoma

- ID: D000002280

- Term: Carcinoma, Basal Cell

### Intervention Browse Module - Browse Branches

- Abbrev: Infe

- Name: Anti-Infective Agents

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M13957

- Name: Poly I-C

- Relevance: LOW

- As Found: Unknown

### Misc Info Module

- Version Holder: 2024-05-31
