A Study of FAZ053 Single Agent and in Combination With PDR001 in Patients With Advanced Malignancies.

NCT02936102 | Terminated Phase 1 FDA Drug

• 2 other identifiers: CFAZ053X21012016-001470-15
• Study Type: interventional
• Target: 154
• Locations: 9 countries
• Sites: 12

Brief Summary

The purpose of this "first-in-human" study of FAZ053 is to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of FAZ053 administered Intravenously (i.v.)as a single agent or in combination with PDR001 in adult patients with advanced solid tumors. By blocking the interaction between Programmed Death Ligand-1 (PD-L1) and its receptors, Programmed Death-1 (PD-1) and B7.1, FAZ053 inhibits the PD-L1 immune checkpoint, resulting in activation of an antitumor immune response by activating effector T-cells and inhibiting regulatory T-cells. This study has been designed as a Phase I, open-label, multi-center study with a dose escalation part of FAZ053 as single agent and in combination with PDR001, followed by a dose expansion part of FAZ053 as single agent. FAZ053 will initially be dosed every three weeks. A less frequent dosing regimen such as every 6 weeks may be evaluated in parallel. A patient may continue treatment with FAZ053 single agent or in combination with PDR001 until the patient experiences unacceptable toxicity, confirmed disease progression per immune related Response Criteria and/or treatment is discontinued at the discretion of the investigator or the patient.

Conditions

Advanced Solid Tumors; Triple Negative Breast Cancer; Chordoma and Alveolar Soft Part Sarcoma

Keywords

Phase I; FAZ053; PDR001; Checkpoint inhibitor; PD-L1; PD-1

Outcome Measures

Primary Outcomes (4)

• Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Incidence and severity of AEs and SAEs by treatment group, including changes in vital signs, electrocardiograms (ECGs) and laboratory parameters qualifying and reported as AEs.

  throughout the study and up to 150 days after end of treatment (up to approximately 46 months)

• Incidence of Dose Limiting Toxicities (DLTs)

  A DLT is defined as an adverse event or abnormal laboratory value of Common Terminology Criteria for Adverse Events (CTCAE) grade ≥ 3 assessed as unrelated to disease, disease progression, inter-current illness or concomitant medications, which occurs within the first 21 days of treatment with FAZ053 alone or within the first 42 days when FAZ053 is given in combination with PDR001 during the dose escalation part of the study. Other clinically significant toxicities may be considered to be DLTs, even if not CTCAE grade 3 or higher.

  21 days (single agent FAZ053) and 42 days (FAZ053+PDR001)

• Dose interruptions and reductions

  Number of participants with dose interruptions and reductions as a measure of tolerability.

  Up to approximately 41 months

• Dose intensity

  Dose intensity is defined as actual dose received divided by actual duration of exposure.

  Up to approximately 41 months

Secondary Outcomes (22)

• Serum concentration-time profiles of FAZ053 as single agent and FAZ053 in combination with PDR001.

  41 months

• Presence of anti-FAZ053 and anti-PDR001.

  41 months

• Concentration of anti-FAZ053 and anti-PDR001.

  41 months

• Receptor Occupancy (RO) profiles when FAZ053 is given as single agent.

  41 months

• Total soluble/shed PD-L1 concentration-time profiles when FAZ053 is given as single agent and for FAZ053 in combination with PDR001.

  41 months

Study Arms (2)

FAZ053 single agent

EXPERIMENTAL

Drug: FAZ053

FAZ053 + PDR001

EXPERIMENTAL

Drug: FAZ053; Drug: PDR001

Interventions

FAZ053 DRUG

Anti-PD-L1 Antibody

FAZ053 + PDR001; FAZ053 single agent

PDR001 DRUG

Anti-PD-1 Antibody

FAZ053 + PDR001

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent prior to any procedure.
• Dose escalation cohorts of FAZ053 single agent and FAZ053 in combination with PDR001: Patients with advanced/metastatic solid tumors with measurable or non-measurable disease as determined by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 who may or may not have received prior treatment with an immune checkpoint inhibitor, who have progressed despite standard therapy, or for whom no standard therapy is available.
• Dose expansion groups of FAZ053 single agent: Patients with advanced/metastatic solid tumors with at least one measurable lesion as determined by RECIST version 1.1 who may or may not have received prior treatment with an immune checkpoint inhibitor (for FAZ053 single agent no treatment with an anti-PD-L1 inhibitor is permitted), who have progressed despite standard therapy, or for whom no standard therapy is available and fit into one of the following groups:
• FAZ053 single agent: TNBC/ Chordoma/ ASPS
• Performance Status (PS) ≤ 2:
• Patient must have a site of disease amenable to biopsy and be a candidate for tumor biopsy according to the treating institution's guidelines. Patient must be willing to undergo a new tumor biopsy at screening/ baseline and during therapy on this study.

You may not qualify if:

• Presence of symptomatic central nervous system (CNS) metastases or CNS metastases that require local CNS-directed therapy (e.g. radiotherapy or surgery) or increasing doses of corticosteroids within the prior 2 weeks. Patients with treated brain metastases should be neurologically stable (for 4 weeks post-treatment and prior to study enrollment) and off of steroids for at least 2 weeks before administration of any study treatment.
• History of severe hypersensitivity to study treatment excipients and additives or other monoclonal antibodies (mAbs) and/or their excipients.
• Active, known or suspected autoimmune disease. Patients with vitiligo, residual hypothyroidism only requiring hormone replacement, psoriasis not requiring systemic treatment or conditions not expected to recur in the absence of an external trigger should not be excluded. Patients previously exposed to anti-PD-1/PD-L1 treatment who are adequately treated for skin rash or with replacement therapy for endocrinopathies should not be excluded.
• Treatment with cytotoxic or targeted antineoplastics within 3 weeks of initiation of study treatment. For cytotoxic agents that have major delayed toxicity a washout period of one cycle is indicated (examples are nitrosoureas and mitomycin C which typically require a 6 week washout). Prior antibodies or immunotherapies require a 6 week washout.
• Patients receiving systemic chronic steroid therapy or any immunosuppressive therapy (≥ 10mg/day prednisone or equivalent). Topical, inhaled, nasal and ophthalmic steroids are allowed.
• Active infection requiring systemic antibiotic therapy.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (12)

Memorial Sloan Kettering Cancer Ctr

New York, New York, 10065, United States

Location

UT MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Novartis Investigative Site

Toronto, Ontario, M5G 2M9, Canada

Location

Novartis Investigative Site

Toulouse, 31059, France

Location

Novartis Investigative Site

Tel Aviv, 6423906, Israel

Location

Novartis Investigative Site

Milan, MI, 20133, Italy

Location

Novartis Investigative Site

Modena, MO, 41124, Italy

Location

Novartis Investigative Site

Koto Ku, Tokyo, 135 8550, Japan

Location

Novartis Investigative Site

Singapore, 119074, Singapore

Location

Novartis Investigative Site

Seville, Andalusia, 41013, Spain

Location

Novartis Investigative Site

Barcelona, Catalonia, 08035, Spain

Location

Novartis Investigative Site

Taipei, 10002, Taiwan

Location

Related Links

• CFAZ053X2101 Clinical Trial Results

MeSH Terms

Conditions

Triple Negative Breast Neoplasms; Chordoma; Sarcoma, Alveolar Soft Part

Interventions

spartalizumab

Condition Hierarchy (Ancestors)

Breast Neoplasms; Neoplasms by Site; Neoplasms; Breast Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Sarcoma

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2016
• First Posted: October 18, 2016
• Study Start: October 20, 2016
• Primary Completion: November 22, 2024
• Study Completion: November 22, 2024
• Last Updated: November 14, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); ES (2); TW (1); CA (1); IL (1); IT (2); JP (1); FR (1); SG (1)