A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma

NCT02150967 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: CBGJ398X22042013-005085-19
• Study Type: interventional
• Target: 143
• Locations: 11 countries
• Sites: 37

Brief Summary

This is a multi-center, open label, single arm phase II study evaluating BGJ398 (infigratinib) anti-tumor activity in advanced or metastatic cholangiocarcinoma patients with fibroblast growth factor receptor (FGFR) genetic alterations.

Conditions

Advanced Cholangiocarcinoma; FGFR2 Gene Mutation

Keywords

cholangiocarcinoma,; FGFR2 gene fusion,; FGFR genetic alteration

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR) as Assessed by Blinded Independent Central Imaging Review (BICR)

  ORR is defined as the percentage (%) of subjects with a best overall response of Complete Response (CR) or Partial Response (PR), as per Response Evaluation Criteria in Solid Tumors (RECIST), Version 1.1, evaluated by computed tomography (CT) or magnetic resonance imaging (MRI) scans every 28 days. RECIST (v1.1) response criteria were as follows: CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to \<10 mm. PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions. Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions). Results were previously disclosed (22 June 2022). There are no additional efficacy endpoints to assess for Cohort 1, thus efficacy data were not re-analyzed for the final analysis. Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.

  Analysis was conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.

Secondary Outcomes (7)

• Overall Response Rate (ORR) as Assessed by the Investigator

  Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff for the primary analysis was 01 March 2021.

• Best Overall Response (BOR)

  Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.

• Disease Control Rate (DCR)

  Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.

• Progression-Free Survival (PFS)

  Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.

• Overall Survival (OS)

  Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.

Other Outcomes (1)

• Growth Modulation Index (GMI)

  Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.

Study Arms (1)

BGJ398 (infigratinib)

EXPERIMENTAL

To estimate the anti-tumor activity of BGJ398 (infigratinib)

Drug: BGJ398 (infigratinib)

Interventions

BGJ398 (infigratinib) DRUG

Capsule for oral use

BGJ398 (infigratinib)

Eligibility Criteria

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

You may qualify if:

• \- Adult patients with histologically or cytologically confirmed cholangiocarcinoma at the time of diagnosis.
• Patients with cancers of the gallbladder or ampulla of Vater are not eligible.
• \- Patients must have received at least one prior regimen containing gemcitabine with or without cisplatin for advanced/ metastatic disease. Patient should have evidence of progressive disease following prior regimen, or if prior treatment discontinued due to toxicity must have continued evidence of measurable or evaluable disease.

You may not qualify if:

• Prior or current treatment with a MEK inhibitor (all Cohorts), BGJ398 (infigratinib) (all Cohorts), or selective FGFR inhibitor (Cohorts 1 and 2 only).
• insufficient organ function
• Absolute Neutrophil Count (ANC) \< 1,000/mm3 \[1.0 x 10\^9/L\]
• Platelets \< 75,000/mm3 \[75 x 10\^9/L\]
• Hemoglobin \< 109.0 g/dL
• Total bilirubin \> 1.5x upper limit of normal (ULN)
• Aspartate aminotransferase/glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/glutamic pyruvic transaminase (ALT/SGPT) \> 2.5x ULN (AST and ALT \> 5x ULN in the presence of liver metastases)
• Serum creatinine \> 1.5x ULN and a calculated or measured creatinine clearance \< 45 mL/min
• Inorganic phosphorus outside of normal limits
• Total and ionized serum calcium outside of normal limits

Sponsors & Collaborators

• QED Therapeutics, a BridgeBio company: lead
• Helsinn Healthcare SA: collaborator

Study Sites (37)

QED Investigative Site

Phoenix, Arizona, 85054, United States

Location

QED Investigative Site

Los Angeles, California, 90033, United States

Location

QED Investigative Site

Los Angeles, California, 90095, United States

Location

QED Investigative Site

San Francisco, California, 94158, United States

Location

QED Investigative Site

Boston, Massachusetts, 02114, United States

Location

QED Investigative Site

Detroit, Michigan, 48201, United States

Location

QED Investigative Site

New York, New York, 10016, United States

Location

QED Investigative Site

New York, New York, 10029, United States

Location

QED Investigative Site

New York, New York, 10065, United States

Location

QED Investigative Site

Columbus, Ohio, 43221, United States

Location

QED Investigative Site

Houston, Texas, 77030-4009, United States

Location

QED Investigative Site

Brussels, 1200, Belgium

Location

QED Investigative Site

Leuven, 3000, Belgium

Location

QED Investigative Site

Cologne, North Rhine-Westphalia, 50937, Germany

Location

QED Investigative Site

Heidelberg, 69120, Germany

Location

QED Investigative Site

Tübingen, Germany

Location

QED Investigative Site

Ancona, AN, 60126, Italy

Location

QED Investigative Site

Milan, MI, 20132, Italy

Location

QED Investigative Site

Roma, RM, 00168, Italy

Location

QED Investigative Site

Moscow, 125367, Russia

Location

QED Investigative Site

Volzhskiy, 404133, Russia

Location

QED Investigative Site

Singapore, 119228, Singapore

Location

QED Investigative Site

Singapore, 169610, Singapore

Location

QED Investigative Site

Seoul, Korea, 03080, South Korea

Location

QED Investigative Site

Seoul, Korea, 06351, South Korea

Location

QED Investigative Site

Barcelona, 8035, Spain

Location

QED Investigative Site

Barcelona, 8908, Spain

Location

QED Investigative Site

Madrid, 28050, Spain

Location

QED Investigative Site

Taipei, Taiwan ROC, 10041, Taiwan

Location

QED Investigative Site

Zhunan, 35053, Taiwan

Location

QED Investigative Site

Khon Kaen, THA, 40002, Thailand

Location

QED Investigative Site

Bangkok, 10330, Thailand

Location

QED Investigative Site

Bangkok, 10400, Thailand

Location

QED Investigative Site

Bebington, CH63 4JY, United Kingdom

Location

QED Investigative Site

Birmingham, B15 2TH, United Kingdom

Location

QED Investigative Site

Manchester, M20 4BX, United Kingdom

Location

QED Investigative Site

Nottingham, NG5 1PB, United Kingdom

Location

Related Publications (3)

• Javle M, Roychowdhury S, Kelley RK, Sadeghi S, Macarulla T, Weiss KH, Waldschmidt DT, Goyal L, Borbath I, El-Khoueiry A, Borad MJ, Yong WP, Philip PA, Bitzer M, Tanasanvimon S, Li A, Pande A, Soifer HS, Shepherd SP, Moran S, Zhu AX, Bekaii-Saab TS, Abou-Alfa GK. Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study. Lancet Gastroenterol Hepatol. 2021 Oct;6(10):803-815. doi: 10.1016/S2468-1253(21)00196-5. Epub 2021 Aug 3.

  PMID: 34358484 DERIVED

• Javle M, Lowery M, Shroff RT, Weiss KH, Springfeld C, Borad MJ, Ramanathan RK, Goyal L, Sadeghi S, Macarulla T, El-Khoueiry A, Kelley RK, Borbath I, Choo SP, Oh DY, Philip PA, Chen LT, Reungwetwattana T, Van Cutsem E, Yeh KH, Ciombor K, Finn RS, Patel A, Sen S, Porter D, Isaacs R, Zhu AX, Abou-Alfa GK, Bekaii-Saab T. Phase II Study of BGJ398 in Patients With FGFR-Altered Advanced Cholangiocarcinoma. J Clin Oncol. 2018 Jan 20;36(3):276-282. doi: 10.1200/JCO.2017.75.5009. Epub 2017 Nov 28.

  PMID: 29182496 DERIVED

• Borad MJ, Gores GJ, Roberts LR. Fibroblast growth factor receptor 2 fusions as a target for treating cholangiocarcinoma. Curr Opin Gastroenterol. 2015 May;31(3):264-8. doi: 10.1097/MOG.0000000000000171.

  PMID: 25763789 DERIVED

MeSH Terms

Conditions

Cholangiocarcinoma

Interventions

infigratinib

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms

Results Point of Contact

• Title: David van Veenhuyzen, Vice President, Clinical Development
• Organization: QED Therapeutics, Inc.

david.vanveenhuyzen@bridgebio.com

1 650 391-9740

Study Officials

• QED Therapeutics

  QED Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2014
• First Posted: May 30, 2014
• Study Start: July 23, 2014
• Primary Completion: March 1, 2021
• Study Completion: February 7, 2022
• Last Updated: July 3, 2023
• Results First Posted: July 15, 2022

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

KR (2); ES (3); US (11); GB (4); SG (2); TW (2); DE (3); RU (2); IT (3); TH (3); BE (2)