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

Phase 1
Active, not recruiting
Conditions
Triple Negative Breast Cancer
Chordoma and Alveolar Soft Part Sarcoma
Advanced Solid Tumors
Interventions
Drug: FAZ053
Drug: PDR001
Registration Number
NCT02936102
Lead Sponsor
Novartis Pharmaceuticals
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
154
Inclusion Criteria
  • 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.
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Exclusion Criteria
  • 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.

Other protocol-defined inclusion/exclusion criteria may apply.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
FAZ053 single agentFAZ053-
FAZ053 + PDR001FAZ053-
FAZ053 + PDR001PDR001-
Primary Outcome Measures
NameTimeMethod
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)throughout the study and up to 150 days after end of treatment (up to approximately 46 months)

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.

Incidence of Dose Limiting Toxicities (DLTs)21 days (single agent FAZ053) and 42 days (FAZ053+PDR001)

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.

Dose interruptions and reductionsUp to approximately 41 months

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

Dose intensityUp to approximately 41 months

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

Secondary Outcome Measures
NameTimeMethod
Duration of response per RECIST 1.141 months
Presence 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
Serum concentration-time profiles of FAZ053 as single agent and FAZ053 in combination with PDR001.41 months
Concentration of anti-FAZ053 and anti-PDR001.41 months
Histopathology of tumor infiltrating lymphocytes (TILs) by eosin (H&E) stain.41 months
Overall response rate (ORR) per RECIST v1.141 months
Histopathology of tumor infiltrating lymphocytes (TILs) by hematoxylin.41 months
Best overall response per RECIST v1.141 months
Disease control rate per RECIST 1.141 months
Progression free survival (PFS) per RECIST 1.141 months
Overall response rate (ORR) per immune related Response Criteria (irRC).41 months
Progression free survival (PFS) per immune related Response Criteria (irRC).41 months
Characterization of Tumor Infiltrating Lymphocytes (TILs) by Immunohistochemistry (IHC)41 months
Characterization of myeloid cell infiltrate by IHC.41 months
Area under the curve (AUC) for FAZ053 as single agent and FAZ053 in combination with PDR001.41 months
Cmax for FAZ053 as single agent and FAZ053 in combination with PDR001.41 months
Tmax for FAZ053 as single agent and FAZ053 in combination with PDR001.41 months
Half-life for FAZ053 as single agent and FAZ053 in combination with PDR001.41 months
Clast for FAZ053 as single agent and FAZ053 in combination with PDR001.41 months
Tlast for FAZ053 as single agent and FAZ053 in combination with PDR001.41 months

Trial Locations

Locations (3)

Memorial Sloan Kettering Cancer Ctr

🇺🇸

New York, New York, United States

UT MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Novartis Investigative Site

🇨🇳

Taipei, Taiwan

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