Solid TumorPhase 1/2NCT01752920
Phase 1/2 Study of Derazantinib (ARQ 087) in Adult Subjects With Advanced Solid Tumors With FGFR Genetic Alterations
A Phase 1/2 study testing Derazantinib low dose range, Derazantinib middle dose range and Derazantinib high dose range in people with Solid Tumor, run by Basilea Pharmaceutica at 12 sites in 2 countries.
Trial summary
This was an open-label, Phase 1/2, dose escalation and signal finding study of derazantinib administered to patients with advanced solid tumors (Part 1; Dose Escalation/Food-effect Cohorts) or with advanced solid tumors with FGFR genetic aberrations, including iCCA with FGFR2 gene fusion (Part 2; Expanded Cohort… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Solid Tumor
- What is being tested?
- Derazantinib low dose range, Derazantinib middle dose range and Derazantinib high dose range
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 119 participants
- Where
- 12 sitesin 2 countries
- Study dates
- 10 Dec 2012to 28 Aug 2018
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 18Show fewer
- 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.
Who cannot join
- 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
Show all 16Show fewer
- 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
- 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
Trial locations and contacts
12 sites in 2 countries.
12 sites in 2 countries
United States8 sites
Emory University, Winship Cancer Institute
Atlanta, Georgia, 30322
Karmanos Cancer Institute, Detroit
Detroit, Michigan, 48201
Comprehensive Cancer Centers of Nevada
Las Vegas, Nevada, 89169
University of Pennsylvania Hospital
Philadelphia, Pennsylvania, 19104
START - South Texas Accelerated Research Therapeutics, LLC
San Antonio, Texas, 78229
Scottsdale Healthcare Research Institute
Scottsdale, Arizona, 85258
University of Washington
Seattle, Washington, 98109
Montefiore-Einstein Center for Cancer Care
The Bronx, New York, 10467
Italy4 sites
Istituto Clinico Humanitas
Milan, 20089
Istituto Nazionale Tumori (National Cancer Institute)
Milan, 20133
Instituto Oncologico Veneto, IRCCS
Padova, 35128
Azienda Ospedaliero-Universitaria Pisana - U.O. Oncologia Medica 2 Univ.
Pisa, 56126
About this study
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
This was an open-label, Phase 1/2, dose escalation and signal finding study of derazantinib administered to patients with advanced solid tumors (Part 1; Dose Escalation/Food-effect Cohorts) or with advanced solid tumors with FGFR genetic aberrations, including iCCA with FGFR2 gene fusion (Part 2; Expanded Cohort, signal finding).
- Conditions
- Solid Tumor
- Being tested
- Derazantinib low dose range, Derazantinib middle dose range, Derazantinib high dose range, Derazantinib at recommended phase 2 dose (RP2D)
- Keywords
- 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
- Medical subject headings
- Liver Neoplasms, Biliary Tract Neoplasms, Cholangiocarcinoma, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Derazantinib low dose rangeDrug
Derazantinib was administered orally at dose levels from 25 mg QOD - 200 mg QD on a 28-day schedule.
Derazantinib middle dose rangeDrug
Derazantinib was administered orally at dose levels from 250 mg QD - 325 mg QD on a 28-day schedule.
Derazantinib high dose rangeDrug
Derazantinib was administered orally at dose levels from 400 mg QD - 425 mg QD on a 28-day schedule.
Derazantinib at recommended phase 2 dose (RP2D)Drug
Derazantinib was administered orally at the recommended phase 2 dose of 300 mg QD on a 28-day schedule.
| Study group | Role | What they receive |
|---|---|---|
| Low Dose Group | Experimental | 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. Receives: Drug: Derazantinib low dose range |
| Middle Dose Group | Experimental | 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. Receives: Drug: Derazantinib middle dose range |
| High Dose Group | Experimental | 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. Receives: Drug: Derazantinib high dose range |
| Expanded Cohort Group | Experimental | 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. Receives: Drug: Derazantinib at recommended phase 2 dose (RP2D) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With Drug-related Treatment-emergent Adverse Events (TEAEs)
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)
Measured: 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)
Secondary outcomes (3)
Proportion of Patients With an Objective Tumor Response Per RECIST 1.1
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.
Measured: 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.
Proportion of Patients With Disease Control Per RECIST 1.1
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.
Measured: 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.
Progression-free Survival (PFS)
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.
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2012 (estimated)
- Study start10 Dec 2012
- Primary completion28 Aug 2018
- Study completion28 Aug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Solid TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 119 participants (actual)
Sponsor and investigators
- Lead sponsor
- Basilea Pharmaceutica
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
- Responsible party
- Sponsor
- Investigators
- Marc Engelhardt, MD, Study director, Basilea Pharmaceutica
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01752920
- Sponsor's protocol ID
- ARQ 087-101
- Status last verified
- May 2023
- First submitted
- 14 Dec 2012
- Last updated
- 5 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
Publications
Papadopoulos KP, El-Rayes BF, Tolcher AW, Patnaik A, Rasco DW, Harvey RD, LoRusso PM, Sachdev JC, Abbadessa G, Savage RE, Hall T, Schwartz B, Wang Y, Kazakin J, Shaib WL. A Phase 1 study of ARQ 087, an oral pan-FGFR inhibitor in patients with advanced solid tumours. Br J Cancer. 2017 Nov 21;117(11):1592-1599. doi: 10.1038/bjc.2017.330. Epub 2017 Oct 3.
PubMed 28972963Mazzaferro V, El-Rayes BF, Droz Dit Busset M, Cotsoglou C, Harris WP, Damjanov N, Masi G, Rimassa L, Personeni N, Braiteh F, Zagonel V, Papadopoulos KP, Hall T, Wang Y, Schwartz B, Kazakin J, Bhoori S, de Braud F, Shaib WL. Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. Br J Cancer. 2019 Jan;120(2):165-171. doi: 10.1038/s41416-018-0334-0. Epub 2018 Nov 13.
PubMed 30420614Hall TG, Yu Y, Eathiraj S, Wang Y, Savage RE, Lapierre JM, Schwartz B, Abbadessa G. Preclinical Activity of ARQ 087, a Novel Inhibitor Targeting FGFR Dysregulation. PLoS One. 2016 Sep 14;11(9):e0162594. doi: 10.1371/journal.pone.0162594. eCollection 2016.
PubMed 27627808
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01752920
- Acronym / sponsor ID
- ARQ 087-101
- Conditions
- Solid Tumor
- Keywords
- FGFR, ARQ 087, Targeted therapy, Molecular therapy, Tyrosine kinase inhibitor, TKI, Receptor tyrosine kinase, RTK
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 119 participants (actual)
- Lead sponsor
- Basilea PharmaceuticaIndustry (pharma, biotech, devices)
- Collaborators
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
- Study start
- 10 Dec 2012 (actual)
- Primary completion
- 28 Aug 2018 (actual)
- Study completion
- 28 Aug 2018 (actual)
- First posted
- 19 Dec 2012 (estimated)
- Results
- Posted 28 Oct 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was an open-label, Phase 1/2, dose escalation and signal finding study of derazantinib administered to patients with advanced solid tumors (Part 1; Dose Escalation/Food-effect Cohorts) or with advanced solid tumors with FGFR genetic aberrations, including iCCA with FGFR2 gene fusion (Part 2; Expanded Cohort, signal finding).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Low Dose Group | Experimental | Drug: Derazantinib low dose rangePatients 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. |
| Middle Dose Group | Experimental | Drug: Derazantinib middle dose rangePatients 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. |
| High Dose Group | Experimental | Drug: Derazantinib high dose rangePatients 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. |
| Expanded Cohort Group | Experimental | Drug: Derazantinib at recommended phase 2 dose (RP2D)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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Derazantinib low dose rangeDerazantinib was administered orally at dose levels from 25 mg QOD - 200 mg QD on a 28-day schedule. | Drug | Low Dose Group |
| Derazantinib middle dose rangeDerazantinib was administered orally at dose levels from 250 mg QD - 325 mg QD on a 28-day schedule. | Drug | Middle Dose Group |
| Derazantinib high dose rangeDerazantinib was administered orally at dose levels from 400 mg QD - 425 mg QD on a 28-day schedule. | Drug | High Dose Group |
| 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. | Drug | Expanded Cohort Group |
Primary outcome
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)
3 secondary outcomes
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.
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.
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 28972963Papadopoulos KP, El-Rayes BF, Tolcher AW, Patnaik A, Rasco DW, Harvey RD, LoRusso PM, Sachdev JC, Abbadessa G, Savage RE, Hall T, Schwartz B, Wang Y, Kazakin J, Shaib WL. A Phase 1 study of ARQ 087, an oral pan-FGFR inhibitor in patients with advanced solid tumours. Br J Cancer. 2017 Nov 21;117(11):1592-1599. doi: 10.1038/bjc.2017.330. Epub 2017 Oct 3.
- Results publication: PubMed 30420614Mazzaferro V, El-Rayes BF, Droz Dit Busset M, Cotsoglou C, Harris WP, Damjanov N, Masi G, Rimassa L, Personeni N, Braiteh F, Zagonel V, Papadopoulos KP, Hall T, Wang Y, Schwartz B, Kazakin J, Bhoori S, de Braud F, Shaib WL. Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. Br J Cancer. 2019 Jan;120(2):165-171. doi: 10.1038/s41416-018-0334-0. Epub 2018 Nov 13.
- Derived from this study: PubMed 27627808Hall TG, Yu Y, Eathiraj S, Wang Y, Savage RE, Lapierre JM, Schwartz B, Abbadessa G. Preclinical Activity of ARQ 087, a Novel Inhibitor Targeting FGFR Dysregulation. PLoS One. 2016 Sep 14;11(9):e0162594. doi: 10.1371/journal.pone.0162594. eCollection 2016.
Related trials for Solid Tumor
RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
Active, not recruitingPhase 1
A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK043 in Patients With Advanced Solid Tumor
Neoplasms
Active, not recruitingPhase 3
A Safety and Efficacy Extension Study of Pertuzumab in Patients With Solid Tumors Previously Enrolled in a Hoffmann-La Roche-Sponsored Pertuzumab Clinical Trial
Solid Tumors
RecruitingPhase 1
IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
Relapsed Solid Tumor Malignancies, Refractory Solid Tumor Malignancies
RecruitingObservational study
Immune Checkpoint Inhibitor Toxicity Risk Prediction in Solid Tumors
Malignant Solid Neoplasm
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Marc Engelhardt, MD study director
Basilea Pharmaceutica
12 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Derazantinib low dose range, Derazantinib middle dose range and Derazantinib high dose range in people with Solid Tumor, run by Basilea Pharmaceutica at 12 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Oct 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01752920, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2023.