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Derazantinib and Atezolizumab in Patients With Urothelial Cancer

Registration Number
NCT04045613
Lead Sponsor
Basilea Pharmaceutica
Brief Summary

The purpose of this study was to evaluate efficacy of derazantinib monotherapy or derazantinib-atezolizumab in combination in patients with advanced urothelial cancer harboring fibroblast growth factor receptor (FGFR) genetic aberrations (GA) of various clinical stages of disease progression and prior treatments.

Detailed Description

The study comprised five open-label substudies (1-5) in patients with advanced urothelial cancer harboring FGFR GA (with the exception of substudy 2 which did not require a FGFR GA) who were treated by derazantinib monotherapy or derazantinib in combination with atezolizumab. The study enrolled patients with cisplatin-ineligible status, or patients whose disease progressed after either first-line treatment or prior treatment with FGFR inhibitors.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
95
Inclusion Criteria
  • Histologically-confirmed transitional cell carcinoma of the urothelium of the upper or lower urinary tract
  • Recurrent or progressing stage IV disease, or surgically unresectable, recurrent or progressing disease
  • Documented central FGFR genetic aberration (FGFR1, FGFR2, or FGFR3 mutations / short variants and rearrangements / fusions) (Note; Substudy 2 started with patients requiring an FGFR GA, but this requirement was removed from the protocol later on)
  • Measurable disease, as defined by the Investigator using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of 0, 1 or 2
  • Adequate organ functions as indicated by Screening visit local laboratory values
Exclusion Criteria
  • Receipt of prior cancer treatment within specific interval periods
  • Concurrent evidence of any clinically significant corneal or retinal disorder
  • History of clinically significant cardiac disorders
  • Known CNS metastases
  • Concurrent uncontrolled or active infection with human immunodeficiency virus
  • Active hepatitis B or chronic hepatitis B without current antiviral therapy
  • Active hepatitis C
  • Active tuberculosis
  • Severe bacterial, fungal, viral and/or parasitic infections on therapeutic oral or IV medication at the time of first dose of study drug administration

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Substudy 3: Derazantinib 200 mg twice daily + atezolizumab 1200 mgDerazantinib 200 mg twice daily + atezolizumab 1200 mgPatients with urothelial cancer were treated with derazantinib 200 mg twice daily in combination with atezolizumab 1200 mg given every 3 weeks as IV infusion
Substudy 1: Derazantinib 300 mg once dailyDerazantinib 300 mg once daily monotherapyPatients with urothelial cancer were treated with derazantinib 300 mg once daily
Substudy 5: Derazantinib 200 mg twice dailyDerazantinib 200 mg twice daily monotherapyPatients with urothelial cancer were treated with derazantinib 200 mg twice daily
Substudy 4 (Cohort 4b):Derazantinib 300 mg once daily + atezolizumab 1200 mgDerazantinib 300 mg once daily + atezolizumab 1200 mgPatients with urothelial cancer were treated with derazantinib 300 mg once daily in combination with atezolizumab 1200 mg given every 3 weeks as IV infusion
Substudy 2 (Dose-Level 1): Derazantinib 200 mg once daily + atezolizumab 1200 mgDerazantinib 200 mg once daily + atezolizumab 1200 mgPatients with any solid tumors were treated with derazantinib 200 mg once daily in combination with atezolizumab 1200 mg given every 3 weeks as intravenous (IV) infusion
Substudy 2 (Dose-Level 2): Derazantinib 300 mg once daily + atezolizumab 1200 mgDerazantinib 300 mg once daily+ atezolizumab 1200 mgPatients with any solid tumors were treated with derazantinib 300 mg once daily in combination with atezolizumab 1200 mg given every 3 weeks as IV infusion
Substudy 4 (Cohort 4a):Derazantinib 300 mg once dailyDerazantinib 300 mg once daily monotherapy (QD)Patients with FGFR inhibitor resistant urothelial cancer were treated with derazantinib 300 mg once daily
Primary Outcome Measures
NameTimeMethod
Recommended Phase 2 Dose (RP2D) of Derazantinib-atezolizumab in Combination Based on DLT Criteria, Safety and Efficacy Data (Substudy 2)From first dose up to 2 years

The RP2D was determined by a joint decision taken by the Independent Data Monitoring Committee (IDMC), Investigators, and the Sponsor in reviewing the aggregate of DLT and AE data, and considering efficacy data

Objective Response Rate (ORR) Based on RECIST 1.1 (Substudies 1,3,4 and 5)From first dose up to 2 years

ORR was defined as the proportion of patients who achieved a confirmed clinical response (CR) or partial response (PR) by blinded investigator central review (BICR) using the internationally recognized criteria for the radiological assessment in tumor response of solid tumors (RECIST) Version 1.1

Number of Patients With Dose-limiting Toxicities (DLTs) in Substudy 2From first dose up to 2 years

In Substudy 2, the primary endpoint was the number of patients with DLTs. A DLT was defined as a clinically-significant adverse event (AE) or abnormal laboratory value assessed as unrelated to disease progression, intercurrent illness, or concomitant medications. Any DLT had to be a toxicity considered at least possibly related to derazantinib or the combination of derazantinib and atezolizumab

Secondary Outcome Measures
NameTimeMethod
ORR Based on RECIST 1.1 (Substudy 2)From first dose up to 2 years

ORR was defined as the proportion of patients who achieved a confirmed clinical response (CR) or partial response (PR) by blinded investigator central review (BICR) using the internationally recognized criteria for the radiological assessment in tumor response of solid tumors (RECIST) Version 1.1

Disease Control Rate (DCR) Per RECIST 1.1 in All SubstudiesFrom first dose up to 2 years

DCR was defined as the proportion of patients who achieved a confirmed clinical response (CR), partial response (PR) or stable disease (SD) by BICR using the internationally recognized criteria in accordance with RECIST Version 1.1

Number of Patients With at Least Grade 3 Adverse Events (AEs)From first dose and until 90 days following the last dose

Common Terminology Criteria for Adverse Events (CTCAE) displayed by increasing severity grades 3 to 5 (CTCAE grade 3/4/5 )

Duration of Response (DOR) Per RECIST 1.1From first dose up to 2 years

DOR was calculated from the first date of documented tumor response (confirmed CR or PR) to the date of disease progression as assessed by BICR or death per RECIST 1.1

Progression-free Survival (PFS) by RECIST in All SubstudiesFrom first dose up to 2 years

PFS was calculated as the time from cohort assignment until disease progression as assessed by BICR, or death from any cause, whichever came first

Overall Survival (OS) in All SubstudiesFrom first dose up to 2 years

OS was calculated from the date of cohort assignment until death from any cause

Trial Locations

Locations (66)

CTCA Clinical Research Inc., Atlanta

🇺🇸

Newnan, Georgia, United States

Englander Institute Weill Cornell Medicine

🇺🇸

New York, New York, United States

New York Cancer and Blood Specialists

🇺🇸

Port Jefferson Station, New York, United States

University of Texas Southwestern Medical Center (UTSWMC)

🇺🇸

Dallas, Texas, United States

MD Anderson

🇺🇸

Houston, Texas, United States

NEXT Oncology

🇺🇸

San Antonio, Texas, United States

Medical Oncology Associates PS (dba Summit Cancer Centers)

🇺🇸

Spokane, Washington, United States

Coastal Cancer Care

🇦🇺

Birtinya, Australia

Canberra Hospital and Health Services

🇦🇺

Canberra, Australia

John Flynn Private Hospital

🇦🇺

Tugun, Australia

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CTCA Clinical Research Inc., Atlanta
🇺🇸Newnan, Georgia, United States

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