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Phase 1b Trial of BGJ398/BYL719 in Solid Tumors

Phase 1
Completed
Conditions
Advanced Solid Tumors
Metastatic Solid Tumors
Interventions
Registration Number
NCT01928459
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

To study the safety and efficacy of the combination of BGJ398 with BYL719 in patients whose tumors express mutations to PIK3CA with or without alterations to FGFR 1-3.

Detailed Description

This dose escalation/dose expansion study will evaluate the combination of orally administered BGJ398 in combination with orally administered BYL719. During the dose escalation part, the MTD of the combination will be determined in patients whose advanced or metastatic tumors express mutations to PIK3CA. Once the MTD has been determined, the expansion part will begin. Patients will be addd to one of three arms based on the disease type and genetic changes. Patients with metastatic colorectal cancer are not eligible for participation in the expansion part.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
62
Inclusion Criteria
  • Histologically/cytologically confirmed advanced or metastatic solid tumors who have failed standard therapy or for whom no effective standard anti-cancer therapy exists
  • Documented PIK3CA mutations in all patients in dose escalation and expansion with or without documented genetic alterations in FGFR depending upon dose expansion cohort (either local or central determination)
  • Measurable disease defined by RECIST v1.1
  • ECOG performance status of ≤2
Exclusion Criteria
  • Prior PI3Ki or selective FGFR inhibitor treatment (for patients enrolled to expansion part)
  • Colorectal cancer (for patients enrolled to expansion part)
  • Patients with diabetes mellitus requiring insulin treatment and/or with clinical signs or with fasting glucose ≥ 140 mg/dL / 7.8 mmol/L, history of clinically significant gestational diabetes mellitus or documented steroid-induced diabetes mellitus
  • Use of medications that increase serum levels of phosphorus and/or calcium
  • Inorganic phosphorus outside of normal limits
  • Total and ionized serum calcium outside of normal limits

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Solid tumor arm 1BGJ398Patients with solid tumors (except for colorectal cancer) whose tumors express mutations to PIK3CA.
Solid tumor arm 2BGJ398Patients with solid tumors (except for colorectal cancer) whose tumomrs express mutations to PIK3CA and alterations to FGFR 1-3
Dose escalationBYL719To determine the MTD or RDE of the combination of BGJ398 with BYL719 in patients with advanced or metastastic solid tumors that express mutations to PIK3CA.
Solid tumor arm 2BYL719Patients with solid tumors (except for colorectal cancer) whose tumomrs express mutations to PIK3CA and alterations to FGFR 1-3
Metastatic breast cancerBGJ398Evaluation of safety and efficacy in patients with metastatic breast cancer whose tumors contain mutations to PIK3CA and alterations FGFR 1-3.
Metastatic breast cancerBYL719Evaluation of safety and efficacy in patients with metastatic breast cancer whose tumors contain mutations to PIK3CA and alterations FGFR 1-3.
Solid tumor arm 1BYL719Patients with solid tumors (except for colorectal cancer) whose tumors express mutations to PIK3CA.
Dose escalationBGJ398To determine the MTD or RDE of the combination of BGJ398 with BYL719 in patients with advanced or metastastic solid tumors that express mutations to PIK3CA.
Primary Outcome Measures
NameTimeMethod
Incidence rate of dose limiting toxicities (DLTs) of the combination of BGJ398 with BYL719Approximately 8 months

The dose escalation part of the study will be guided by a well-established statistical method/model to estimate the maximum tolerated dose(s) and/or the recommended dose for expansion (RDE). Safety(incidence and nature of DLTs), pharmacokinetic and pharmacodynamic data will guide dose escalation decisioins.

Secondary Outcome Measures
NameTimeMethod
Progression free survivalEvery two months from the date of baseline CT scan

Assessment of preliminary antitumor activity of the combination of BGJ398 with BYL719

Overall response rateEvery two months from the date of baseline CT scan

Assessment of preliminary antitumor activity of the combination of BGJ398 with BYL719; Overall response rate = complete response + partial response

Time vs. concentration profile of BGJ398 and BYL719Every 28 days for up to 10 cycles

Plasma concentration versus time profiles. Plasma PK parameters will be used to characterize the PK profiles of the combination of BGJ398 with BYL719

Safety and tolerability of BGJ398/BYL719 combination at the recommended dose for expansion (RDE)Every 28 days from baseline visit until end of study visit

This will be assessed by looking at the number of Adverse Events (AEs), serious AEs (SAEs) changes in hematology and chemistry values, vital signs, electrocardiograms (ECGs), dose interruptions and reductions

Trial Locations

Locations (8)

H. Lee Moffitt Cancer Center & Research Institute Moffitt 4

🇺🇸

Tampa, Florida, United States

University of Michigan Comprehensive Cancer Center SC

🇺🇸

Ann Arbor, Michigan, United States

Karmanos Cancer Institute Dept of Onc

🇺🇸

Detroit, Michigan, United States

Washington University School of Medicine Onc Dept

🇺🇸

Saint Louis, Missouri, United States

Memorial Sloan Kettering Cancer Center Onc Dept

🇺🇸

New York, New York, United States

Cancer Therapy & Research Center / UT Health Science Center SC

🇺🇸

San Antonio, Texas, United States

Vanderbilt University Medical Center Dept of Onc

🇺🇸

Nashville, Tennessee, United States

Novartis Investigative Site

🇨🇭

Bellinzona, Switzerland

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