A Phase 0 Multicenter Study of the Pharmacodynamic Effects of Intratumoral Microdose Administration of PBA-0111 in Patients With Solid Tumors

NCT06700070 | Recruiting Early Phase 1 FDA Drug

• 1 other identifier: PBL-0111-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This is a multi-center, single arm, open-label, localized pharmacodynamic biomarker Phase 0 trial designed to study the biological effects within the tumor microenvironment of PBA-0111 when administered intratumorally in microdose quantities via the CIVO device.

Conditions

Head and Neck Squamous Cell Carcinoma; Soft Tissue Sarcoma Adult; Triple Negative Breast Cancer

Keywords

Pure Biologics; PBA-0111; TNBC; STS; HNSCC; tumor microenvironment; in vivo drug sensitivity; in vivo oncology; microdosing; microinjection; intratumoral injection; precision oncology

Outcome Measures

Primary Outcomes (1)

• Quantification of Cell Death and Immune Cell Biomarkers by immuno-histochemistry (IHC) and In-Situ Hybridization (ISH)

  Quantification of biomarker-positive and biomarker-negative cells will be performed within the tumor microenvironment around each of the injection sites in each resected patient sample by IHC and/or ISH. An aggregate analysis of this quantification may be done across patient samples to evaluate trends in tumor response. The biomarkers evaluated may include, but are not limited to, drug targets (e.g., GARP, Cluster of Differentiation 16), biomarkers for cell death (e.g., cleaved caspase 3), natural killer cells (e.g., Cluster of Differentiation 56/Cluster of Differentiation 45/Granzyme B), macrophages (Cluster of Differentiation 86, Cluster of Differentiation 68, Cluster of Differentiation 163), and proinflammatory cytokines (e.g., interferon gamma, tumor necrosis factor alpha, interferon-stimulated gene 15, chemokine interferon gamma-inducible protein 10).

  1-2 days after microdose

Secondary Outcomes (1)

• Number of Patients with Adverse Events

  Up to 28 days after microdose injection.

Study Arms (1)

PBA-0111

EXPERIMENTAL

Patients who are scheduled for surgical biopsy or tumor resection surgery will be injected at 1-2 days prior to surgery using the CIVO device. Each needle of the CIVO device will deliver up to 8.3 microliters of solution, including a vehicle control (sterile saline) or subtherapeutic microdoses of PBA-0111, as single agents. Each microdose is simultaneously injected in a columnar fashion through each of 8, or 5, (in a device configuration determined by tumor dimensions) into a single solid tumor or effaced metastatic lymph node

Drug: PBA-0111

Interventions

PBA-0111 DRUG

Intratumoral microdose injection by the CIVO device.

PBA-0111

Eligibility Criteria

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

You may qualify if:

• \. Ability and willingness to comply with the study's visit and assessment schedule. 2. Male or female ≥ 18 years of age at Visit 1 (Screening). 3. Pathologic diagnosis of HNSCC, STS (see restrictions in Note below), or TNBC (see restrictions in Note below; TNBC defined as estrogen receptor negative \[\<1% positive tumor cells\], progesterone receptor negative \[\<1% positive tumor cells\], and human epidermal growth factor receptor 2 negative \[0 to 1+\]) with a tumor planned for surgical resection. Note: For STS, only the following subtypes are eligible: undifferentiated pleomorphic sarcoma, alveolar soft part sarcoma, synovial sarcoma, cutaneous angiosarcoma, or myxofibrosarcoma. Note: For TNBC, if prior neoadjuvant therapy, evidence of progressive disease, at the discretion of the investigator. 4. Ability and willingness to provide written informed consent. Voluntary written consent must be given before performance of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care. 5. As assessed or confirmed by the surgeon, at least one lesion (primary tumor, recurrent tumor, metastatic tumor, or metastatic lymph node) that is surface accessible for CIVO injection that contains viable minimum tumor tissue volume and characteristics (e.g., based on clinical evaluation, available pre-operative imaging, pre-injection ultrasound imaging, or pathology reports indicating lesion with appropriate viable tumor volume without excessive cysts or necrosis) and for which there is a planned surgical intervention. The patient's presentation, surgical and pathology plan may determine whether a lesion is eligible with respect to a given CIVO MID needle configuration. 6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2. 7. Female patients who: • Are postmenopausal for at least one year before the screening visit, OR • Are surgically sterile, OR • Are of childbearing potential who agree to practice a highly effective method of contraception from the time of signing the ICF up to 3 months following the end of study participation OR agree to completely abstain from heterosexual intercourse. • Agree to refrain from donating ova during study participation. Male patients, even if surgically sterile (i.e., status post-vasectomy), who: • Agree to practice effective barrier contraception from the time of signing the ICF up to 3 months following the end of study participation OR agree to completely abstain from heterosexual intercourse. • Agree to refrain from donating sperm during study participation.

You may not qualify if:

• \. Tumors near or involving critical structures for which, in the opinion of the treating clinician, injection would pose undue risk to the patient. 2. Female patients who are: • Both lactating and breastfeeding, OR • Have a positive β-subunit human chorionic gonadotropin (β-hCG) pregnancy test at screening verified by the Investigator. 3. Any uncontrolled intercurrent illness, condition, serious medical or psychiatric illness, or circumstance that, in the opinion of the Investigator, could interfere with adherence to the study's procedures or requirements, or otherwise compromise the study's objectives. 4. HNSCC known to be of cutaneous origin. 5. Patients with uncontrolled autoimmune diseases (see Appendix 1 for examples) requiring systemic treatment 6. Patients with known HIV/AIDS. 7. Patients with known uncontrolled active hepatitis B (defined as hepatitis B surface antigen \[HBsAg\] positive or detectable hepatitis B virus \[HBV\] DNA) or hepatitis C (defined as anti-hepatitis C virus antibody \[anti-HCV Ab\] positive and detectable hepatitis C virus \[HCV\] RNA) infection. Note: Hepatitis B and C screening tests are not required unless: • Patient has a known history of hepatitis B/C infection • Mandated by local health authority 8. Use of any of the following ≤ 3 weeks prior to CIVO injection: a. Systemic anti-cancer therapy (e.g., cytotoxic chemotherapy, targeted agents, or checkpoint inhibitor immunotherapy, etc.), b. Immunosuppressive drugs (e.g., calcineurin inhibitors) c. Biological response modifiers for autoimmune disease d. Systemic glucocorticoids: oral or parenteral corticosteroids at a dose ≥ 20 mg/day prednisone, or equivalent Note: physiologic replacement dosing of steroids (≤ 3 mg/m2/d prednisone or equivalent), low-dose corticosteroids for dye allergies prior to staging scans or use in anti-emetic prophylaxis for patients undergoing chemotherapy, or topical steroids, are allowed e. Hematopoietic growth factors f. Chemotherapy g. Local radiotherapy of the target lesion planned for CIVO injection and surgical resection 9. Patients who have received a live or live attenuated vaccine within 4 weeks of the baseline/screening visit. 10. Patients who have had allogenic tissue/solid organ transplant 11. Patients with an active infection requiring systemic therapy. 12. Patients for whom participation on this study results in a delay of planned surgical intervention.

Sponsors & Collaborators

• Pure Biologics S.A.: lead

Study Sites (1)

LSU Health Sciences Center

Shreveport, Louisiana, 71115, United States

RECRUITING

MeSH Terms

Conditions

Squamous Cell Carcinoma of Head and Neck; Triple Negative Breast Neoplasms

Condition Hierarchy (Ancestors)

Carcinoma, Squamous Cell; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Head and Neck Neoplasms; Neoplasms by Site; Breast Neoplasms; Breast Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• John Weinberg, MBBCH

  Pure Biologics

  STUDY DIRECTOR

Central Study Contacts

Presage Biosciences

CONTACT

800-530-5404; clinops@presagebio.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is an exploratory clinical trial to evaluate intratumoral mechanistic effects of novel and approved agents on intact human tumors. This substudy is not blinded.

Study Record Dates

• First Submitted: November 19, 2024
• First Posted: November 21, 2024
• Study Start: March 27, 2025
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: August 11, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)