Phase 1/2 Study of Derazantinib (ARQ 087) in Adult Subjects With Advanced Solid Tumors With FGFR Genetic Alterations
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ClinicalTrials.gov Identifier: NCT01752920 |
Recruitment Status :
Completed
First Posted : December 19, 2012
Results First Posted : October 28, 2021
Last Update Posted : March 9, 2022
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Condition or disease | Intervention/treatment | Phase |
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Solid Tumor | Drug: Derazantinib low dose range Drug: Derazantinib middle dose range Drug: Derazantinib high dose range Drug: Derazantinib at recommended phase 2 dose (RP2D) | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 119 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1/2 Study of ARQ 087 in Adult Subjects With Advanced Solid Tumors With FGFR Genetic Alterations, Including Intrahepatic Cholangiocarcinoma With FGFR2 Gene Fusion |
Actual Study Start Date : | December 10, 2012 |
Actual Primary Completion Date : | August 28, 2018 |
Actual Study Completion Date : | August 28, 2018 |

Arm | Intervention/treatment |
---|---|
Experimental: Low Dose Group
Patients who received derazantinib orally at dose levels from 25 mg every other day (QOD) - 200 mg daily (QD) on a 28-day schedule until documented progression of disease (clinical or radiological), unacceptable toxicity, or another of the discontinuation criteria.
|
Drug: Derazantinib low dose range
Derazantinib was administered orally at dose levels from 25 mg QOD - 200 mg QD on a 28-day schedule. |
Experimental: Middle Dose Group
Patients who received derazantinib orally at dose levels from 250 mg QD - 325 mg QD on a 28-day schedule until documented progression of disease (clinical or radiological), unacceptable toxicity, or another of the discontinuation criteria.
|
Drug: Derazantinib middle dose range
Derazantinib was administered orally at dose levels from 250 mg QD - 325 mg QD on a 28-day schedule. |
Experimental: High Dose Group
Patients who received derazantinib orally at dose levels from 400 mg QD - 425 mg QD on a 28-day schedule until documented progression of disease (clinical or radiological), unacceptable toxicity, or another of the discontinuation criteria.
|
Drug: Derazantinib high dose range
Derazantinib was administered orally at dose levels from 400 mg QD - 425 mg QD on a 28-day schedule. |
Experimental: Expanded Cohort Group
Patients who received derazantinib orally at the recommended phase 2 dose of 300 mg QD on a 28-day schedule until documented progression of disease (clinical or radiological), unacceptable toxicity, or another of the discontinuation criteria.
|
Drug: Derazantinib at recommended phase 2 dose (RP2D)
Derazantinib was administered orally at the recommended phase 2 dose of 300 mg QD on a 28-day schedule. |
- Number of Patients With Drug-related Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Adverse events were collected and reported from the time of receiving first dose of derazantinib to the end of study assessment and follow-up period (30-day post-treatment) ]Adverse events were graded for severity according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03. CTCAE is classifying AEs and their associated severity from Grade 1 (Mild AE), Grade 2 (Moderate AE), Grade 3 (Severe or medically significant but not immediately life-threatening), Grade 4 (Life-threatening consequences) to Grade 5 (Death related to AE)
- Proportion of Patients With an Objective Tumor Response Per RECIST 1.1 [ Time Frame: Assessments were performed at Baseline, and every 8 weeks during continuous drug administration until the End of Treatment visit (7 [+3] days after the last dose of derazantinib) or as otherwise clinically indicated. ]
The number of patients with an objective tumor response, which included those with either a complete response (CR) or a partial response (PR) based on RECIST v1.1 guidelines which defines criteria for the radiological assessment in tumor response.
The objective response rate (ORR) was defined as the proportion of patients with a CR or PR.
- Proportion of Patients With Disease Control Per RECIST 1.1 [ Time Frame: Assessments were performed at Baseline, and every 8 weeks during continuous drug administration until the End of Treatment visit (7 [+3] days after the last dose of derazantinib) or as otherwise clinically indicated. ]The number of patients with tumor disease control, which included those with either a complete or partial tumor response, or a stable disease (SD) based on RECIST v1.1 guidelines which defines criteria for the radiological assessment in tumor response. The disease control rate (DCR) was defined as the proportion of patients with CR, PR or SD.
- Progression-free Survival (PFS) [ Time Frame: Assessments were performed at Baseline, and every 8 weeks during continuous drug administration until the End of Treatment visit (7 [+3] days after the last dose of derazantinib) or as otherwise clinically indicated. ]PFS was calculated as the time from the date of first dose until documented radiographic disease progression or death from any cause, whichever occurred first. Disease progression is measured according to a specified radiologic increase in tumor size.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Signed written informed consent granted
- Men or women ≥18 years of age
- Histologically or cytologically confirmed, locally advanced, inoperable, or metastatic solid tumors. Patients eligible for enrollment in the Expanded Cohort must have documented and/or confirmed FGFR genetic aberrations, including iCCA with FGFR2 gene fusion.
- Failure to respond to standard therapy, or for whom standard therapy does not exist.
- Evaluable or measurable disease
- Archival and/or fresh biopsy tissue samples must be available prior to the first dose of the study drug
- Life expectancy ≥ 12 weeks
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Hemoglobin (Hgb) ≥ 9.0 g/dL
- Absolute neutrophil count (ANC) ≥ 1.5 x 10^9/L
- Platelet count ≥ 100 x 10^9/L
- Total bilirubin ≤ 1.5 × upper limit of normal (ULN) (≤ 2 x ULN for patients with cholangiocarcinoma)
- Aspartate transaminase (AST) and alanine transaminase (ALT) ≤ 3 × ULN (≤ 5 x ULN for patients with liver metastases)
- Serum creatinine ≤ 1.5 x ULN or creatinine clearance > 60 mL/min/1.73 m^2 for patients with creatinine levels above institutional normal
- Albumin ≥ 2.8 g/dL
- INR 0.8 to ULN or ≤ 3 for patients receiving anticoagulant therapy
- Men or women of child-producing potential must agree to use double-barrier contraceptive measures, oral contraception, or avoid intercourse during the study and for 90 days after the last dose of study drug
- Women of childbearing potential must have a negative serum pregnancy test during Screening Period and within 48 hours of the first dose of derazantinib.
Exclusion Criteria:
- Anti-cancer therapy, such as chemotherapy, immunotherapy, hormonal, targeted therapy, or investigational agents within four weeks or five times of the drug half life, whichever is longer, of the first dose of derazantinib
- Major surgery or radiation therapy within four weeks of the first dose of derazantinib
- Previous treatment with FGFR inhibitors
- History of allergic reactions attributed to compounds of similar chemical or biological composition as derazantinib
- Unable or unwilling to swallow the complete daily dose of derazantinib
- Clinically unstable central nervous system (CNS) metastasis
- History of myocardial infarction (MI) or congestive heart failure defined as Class II to IV per the New York Heart Association classification within 6 months of the first dose of derazantinib (MI occurring >6 months of the first dose of derazantinib will be permitted)
- Significant GI disorder(s) that could interfere with the absorption, metabolism, or excretion of derazantinib (e.g. Crohn's disease, ulcerative colitis, extensive gastric resection)
- History and/or current evidence of clinically relevant ectopic mineralization/calcification
- Previous malignancy within 2 years prior to the first dose of derazantinib, except curatively treated non-melanoma skin cancer, carcinoma in-situ of the breast or cervix, or superficial bladder tumors
- Known human immunodeficiency virus (HIV) infection
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Concurrent uncontrolled illness not related to cancer, including but not limited to:
- Psychiatric illness/substance abuse/social situation that would limit compliance with study requirements.
- Uncontrolled diabetes mellitus
- Blood transfusion within 5 days of the blood draw being used to confirm eligibility
- Pregnant or breastfeeding

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01752920
United States, Arizona | |
Scottsdale Healthcare Research Institute | |
Scottsdale, Arizona, United States, 85258 | |
United States, Georgia | |
Emory University, Winship Cancer Institute | |
Atlanta, Georgia, United States, 30322 | |
United States, Michigan | |
Karmanos Cancer Institute, Detroit | |
Detroit, Michigan, United States, 48201 | |
United States, Nevada | |
Comprehensive Cancer Centers of Nevada | |
Las Vegas, Nevada, United States, 89169 | |
United States, New York | |
Montefiore-Einstein Center for Cancer Care | |
Bronx, New York, United States, 10467 | |
United States, Pennsylvania | |
University of Pennsylvania Hospital | |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, Texas | |
START - South Texas Accelerated Research Therapeutics, LLC | |
San Antonio, Texas, United States, 78229 | |
United States, Washington | |
University of Washington | |
Seattle, Washington, United States, 98109 | |
Italy | |
Istituto Clinico Humanitas | |
Milan, Italy, 20089 | |
Istituto Nazionale Tumori (National Cancer Institute) | |
Milan, Italy, 20133 | |
Instituto Oncologico Veneto, IRCCS | |
Padova, Italy, 35128 | |
Azienda Ospedaliero-Universitaria Pisana - U.O. Oncologia Medica 2 Univ. | |
Pisa, Italy, 56126 |
Study Director: | Marc Engelhardt, MD | Basilea Pharmaceutica |
Documents provided by Basilea Pharmaceutica:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Basilea Pharmaceutica |
ClinicalTrials.gov Identifier: | NCT01752920 |
Other Study ID Numbers: |
ARQ 087-101 |
First Posted: | December 19, 2012 Key Record Dates |
Results First Posted: | October 28, 2021 |
Last Update Posted: | March 9, 2022 |
Last Verified: | March 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
FGFR ARQ 087 Targeted therapy Molecular therapy Tyrosine kinase inhibitor TKI Receptor tyrosine kinase RTK Biomarker Phase 1 Phase I Solid tumor Liver Cancer Hepatobiliary carcinoma Biliary tract cancer |
Cholangiocarcinoma Intrahepatic cholangiocarcinoma FGFR inhibitor Targeted FGFR kinase inhibitor Pan-FGFR inhibitor Selective FGFR inhibitor FGFR pathway FGFR signaling Fibroblast growth factor FGFR1 FGFR2 FGFR3 FGFR4 FGF FGF19 |
Cholangiocarcinoma Neoplasms Adenocarcinoma |
Carcinoma Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type |