Prospective, Multicenter, Open-label Phase IV trial of Trifluridine/Tipiracil to Evaluate the Health-related Quality of Life in Patients with Metastatic Colorectal Cancer.

Organizational Data

DRKS-ID:
DRKS00012977
Recruitment Status:
Recruiting complete, study complete
Date of registration in DRKS:
2017-09-06
Last update in DRKS:
2022-09-06
Registration type:
Prospective

Acronym/abbreviation of the study

Tallisur

URL of the study

No Entry

Brief summary in lay language

Treatment of adult patients with metastatic colorectal cancer (mCRC) who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapies, anti-VEGF agents, and anti-EGFR agents. This trial is designed to investigate the HRQoL in patients treated with FTD/TPI and those who are treated with BSC while being suitable for treatment with FTD/TPI according to the SmPC. The planned trial will be performed in the approved patient population to analyze QoL as practice-related care research, taking into account common treatment practice in oncology in Germany. Quality of life will be assessed by means of the EORTC-QLQ-C30. Additionally, the EQ-5D-5L questionnaire will be used as an instrument for evaluation of QoL that may be applied together with other measures.

Brief summary in scientific language

In Germany, 60,580 new cases of colorectal cancer were diagnosed in 2013, thereof 33,370 cases in males. 5-year survival rates in patients with metastatic colorectal cancer (mCRC), representing Stage IV CRC, reached only about 15%. Although the outcome of patients with mCRC has clearly improved during recent years with median survival now reaching more than 30 months in recent clinical trials, more treatment options are needed for patients with disease progression after fluoropyrimidine (5-FU), irinotecan, oxaliplatin, applicable anti-VEGF agents and anti-EGFR agents or those unable to tolerate these agents. FTD/TPI (Lonsurf®) has been authorized in the EEA since April 2016 for treatment of these patients. On the basis of the severity of the tumour disease with rather limited treatment options within the context of a previously treated tumour disease in the end-oflife situation, the health-related quality of life (HRQoL) is very important to describe the impact of treatment on the patient’s functioning regarding physical health (including disease-related morbidity), social, emotional, cognitive and role aspects. Changes of HRQoL during and after treatment with FTD/TPI have not been investigated so far in clinical trials. Thus, this trial is designed to investigate the HRQoL in patients treated with FTD/TPI and those who are treated with BSC while being suitable for treatment with FTD/TPI according to the SmPC. It has to be the explicit and informed choice of the patient, to limit the treatment to BSC. This design of a control trial with BSC as appropriate comparative treatment was chosen according to advice by the German Federal Joint Committee (GB-A). The planned trial will be performed in the approved patient population to analyze QoL as practice-related care research, taking into account common treatment practice in oncology in Germany. Quality of life will be assessed by means of the EORTC-QLQ-C30. Additionally, the EQ-5D-5L questionnaire will be used as an instrument for evaluation of QoL that may be applied together with other measures. The trial design also takes into account G-BA’s request for further patientrelevant data with regard to HRQoL, disease-related morbidity, and progression vs. FTD/TPI-associated adverse reactions as discussed with the G-BA.

Health condition or problem studied

Free text:
metastatic colorectal cancer (mCRC)
ICD10:
C18 - Malignant neoplasm of colon
ICD10:
C19 - Malignant neoplasm of rectosigmoid junction
ICD10:
C20 - Malignant neoplasm of rectum
Healthy volunteers:
No Entry

Interventions, Observational Groups

Arm 1:
FTD/TPI (35 mg/m2/dose) is administered orally twice daily (BID) on Days 1 to 5 and Days 8 to 12 of each 28-day cycle as long as benefit is observed or until unacceptable toxicity occurs.
Arm 2:
Each observation cycle is 28 days. Close observation will be performed until occurrence of radiological or clinical progression. Close observation will also end if the patients are given any anti-tumour therapy (chemotherapy including FTD/TPI, targeted therapy, antibodies, any antihormonal tumour treatment, immunotherapy.

Endpoints

Primary outcome:
Rate of responders with continued unchanged or improved HRQoL (Health-related quality of life). Response is calculated as mean of the score of the EORTC QLQ-C30, global health status/quality of life scale [QL2] at all scheduled time points of QoL analysis in the time interval from two days before start of Cycle 2 until the end of treatment /end of close observation compared to the baseline score of the global health status /quality of life scale. Response will be defined as improvement (≥ 10 scores) or stabilization (> -10 and < 10 scores) compared to the baseline score of the global health status/quality of life scale.
Secondary outcome:
Quality of life • Rate of responders in the QoL analysis (measured by the EORTC QLQ-C30, global health status/quality of life scale) at every scheduled time point for EORTC QLQ-C30 separately in the time interval from two days before start of Cycle 2 until the end of treatment/end of close observation, at every time point compared to the baseline score of the global health status/quality of life scale. Response will be defined as improvement (≥ 10 scores) or stabilization (> -10 and < 10 scores) compared to the baseline score of the global health status/quality of life scale at the specified time point. • HRQoL analysis measured by the EORTC QLQ-C30 questionnaire (all scales/single items; all time points from baseline until end of follow up) • HRQoL analysis measured by the questionnaire EQ-5D-5L descriptive system; all time points from baseline until end of follow up • Scores of the EQ VAS as a measure of overall self-rated health status compared to baseline EQ VAS score; all time points until end of follow up • Time to health-related QoL deterioration measured by EORTC QLQ-C30; global health status/quality of life scale; deterioration defined as a first change of score to < -10 compared to baseline score • Time to health-related QoL deterioration measured by EQ VAS score; deterioration compared to baseline VAS score • Disease-specific symptoms of the mCRC measured by the respective subdomains of the EORTC QLQ-C30 (symptom scales fatigue, nausea and vomiting, appetite loss, pain, dyspnoea, constipation, diarrhoea) • HRQoL analysis measured by the EORTC QLQ C30 questionnaire (all scales/single items; all time points from baseline until follow up, analysis of response) for following comparisons (if applicable): - During treatment/close observation vs. after treatment/close observation - Treatment with FTD/TPI vs. close observation with BSC - During treatment/close observation (patients with >= 2 cycles) vs. during treatment/close observation (patients with < 2 cycles and early progression in Cycle 1) • HRQoL analysis measured by the questionnaire EQ-5D-5L and the EQ VAS (all timepoints from baseline to follow up) for following comparisons (if applicable): - During treatment/close observation vs. after treatment/close observation - Treatment with FTD/TPI vs. close observation with BSC - During treatment/close observation (patients with >= 2 cycles) vs. during treatment/close observation (patients with < 2 cycles and early progression in Cycle 1) Efficacy • Progression-free survival (clinical or radiological progression) [PFS] • Overall survival ([OS], calculated from start of treatment/close observation on study) • Exploratory analysis of objective response rate (ORR) Safety • Type, incidence, and severity of FTD/TPI-related adverse reactions (severity evaluated according to CTCAE version 4) • Tumour-related symptoms and adverse events • Treatment duration/exposure to FTD/TPI (Group A)

Study Design

Purpose:
Other
Allocation:
Non-randomized controlled study
Control:
  • Control group receives no treatment
Phase:
IV
Study type:
Interventional
Mechanism of allocation concealment:
No Entry
Blinding:
No
Assignment:
Parallel
Sequence generation:
No Entry
Who is blinded:
No Entry

Recruitment

Recruitment Status:
Recruiting complete, study complete
Reason if recruiting stopped or withdrawn:
No Entry

Recruitment Locations

Recruitment countries:
  • Germany
Number of study centers:
Multicenter study
Recruitment location(s):
  • University medical center Universitätsklinikum Gießen und Marburg GmbH Marburg
  • Medical center Gesundheitszentrum St. Marien GmbH Amberg
  • University medical center Universitätsklinikum Leipzig AöR Leipzig
  • Doctor's practice Heidelberg
  • University medical center Klinikum der Universität München - Großhadern München
  • Doctor's practice Köln
  • Medical center Klinikum Traunstein Traunstein
  • Medical center Klinikum Kassel Kassel
  • Medical center Städt. Klinikum München Bogenhausen München
  • Medical center Klinikum Mutterhaus der Borromäerinnen gGmbH Trier
  • Medical center Hämatologisch/Onkologische Tagesklinik Landshut Landshut
  • Medical center Hämato-Onkologischer Studienkreis am Klinikum Aschaffenburg Aschaffenburg
  • Medical center Kliniken Nordoberpfalz AG, Klinikum Weiden Weiden
  • Doctor's practice Leipzig
  • Medical center St. Franziskus-Hospital Münster Münster
  • Doctor's practice Hannover
  • Medical center Vivantes Netzwerk für Gesundheit GmbH Klinikum Neukölln Berlin
  • University medical center Uniklinik RWTH Aachen Aachen
  • Medical center Evangelisches Klinikum Bethel gGmbH Bielefeld
  • Doctor's practice Dresden
  • Medical center Med. Klinik I, Hämatologie / Onkologie Rheine
  • Medical center III. Med. Klinik Augsburg
  • University medical center Klinik f. Innere Med. II, Abtl. f. Hämatologie und intern. Onkologie Jena
  • Doctor's practice Hamburg
  • Medical center Klinik f. Onkologie Berlin
  • Medical center Datteln
  • Doctor's practice Kaiserslautern
  • Doctor's practice Friedrichshafen
  • Medical center Merseburg
  • Doctor's practice Goslar
  • Medical center Dortmund
  • University medical center Klinik für Innere Med. I Ulm
  • Doctor's practice Offenburg
  • Doctor's practice Lübeck
  • Medical center Klinik f. Hämatologie und Onkologie München
  • Doctor's practice Worms
  • Doctor's practice Oldenburg in Holstein
  • University medical center Med. Klinik m. Schwerpunkt Hämatologie, Onkologie und Tumorimmunologie Berlin
  • Medical center Klinik f. Innere Med. III Rostock
  • Doctor's practice Frechen
  • Medical center Klinikum am Steinenberg, Medizinische Klinik I Reutlingen
  • University medical center Med. Klinik IV und V Gießen
  • Medical center Klinik f. Allgemeine Innere Med., Onkologie/Hämatologie, Gastrenterologie u. Infektiologie Esslingen
  • Doctor's practice Saarbrücken

Recruitment period and number of participants

Planned study start date:
2017-09-22
Actual study start date:
2017-09-22
Planned study completion date:
No Entry
Actual Study Completion Date:
2020-12-24
Target Sample Size:
195
Final Sample Size:
202

Inclusion Criteria

Sex:
All
Minimum Age:
18 Years
Maximum Age:
no maximum age
Additional Inclusion Criteria:
• Patients had provided written informed consent prior to any procedure • Patients of ≥ 18 years of age at the time of signing the informed consent • Histologically or cytologically confirmed UICC stage IV carcinoma of colon or rectum with metastasis (metastatic colorectal cancer) with need for treatment due to progression • At least one measurable or non-measurable lesion as defined by RECIST version 1.132 • Patients who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-, oxaliplatinand irinotecan-based chemotherapies, anti-VEGF agents, and anti-EGFR agents • Patients able to take medications orally (ie, no feeding tube) • mCRC patients independent from their ECOG performance status at study enrolment • Adequate organ function as defined by the following laboratory values obtained within 7 days prior to first administration of FTD/TPI on Day 1 of Cycle 1 (haematology and laboratory values for patients who are administered only BSC need not be obtained within 7 days prior toobservation Cycle 1) a. Absolute neutrophil count of ≥ 1.5 × 109/L, b. Platelet count ≥ 75 × 109/L, c. Total serum bilirubin of ≤ 1.5 upper limit of normal (ULN), d. Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3.0 × ULN; if liver function abnormalities are due to underlying liver metastasis, AST and ALT ≤ 5 × ULN e. Calculated creatinine clearance (CrCl) ≥ 30 mL/min • Only applicable for females who receive treatment with FTD/TPI (Group A): Females of childbearing potential (FCBPs) must have a negative pregnancy test (urine or serum) within 7 days prior to enrolment. FCBPs must agree to use highly effective contraceptive measures with a failure rate of less than 1% per year when used consistently and correctly as defined in Section 4.1 of the CTFG guidance “Recommendations related to contraception and pregnancy testing in clinical trials” during the study treatment with FTD/TPI and up to 6 months after the discontinuation of study drug FTD/TPI. Complete sexual abstinence is acceptable as a highly effective contraceptive method only if the subject is refraining from heterosexual intercourse during the entire study treatment with FTD/TPI and up to 6 months after the discontinuation of study drug FTD/TPI and the reliability of sexual abstinence is in line with the preferred and usual lifestyle of the subject. Women using hormonal contraceptives should agree to add a barrier contraceptive method. A woman will be considered as being of childbearing potential unless she has gone through menopause for at least 1 year (i.e. minimum of one year without menses) or unless she has a history of tubal ligation, bilateral oophorectomy or hysterectomy that is clearly documented in the patient’s source documents. • Only applicable for males who receive treatment with FTD/TPI (Group A): Males must agree to use effective contraceptive measures or to practice complete abstinence during the study treatment with FTD/TPI and up to 6 months after the discontinuation of study drug FTD/TPI • Patients capable to understand the purposes and risks of the study, who are willing and able to participate in the study, who are able to understand and to fill in the questionnaire and from whom written and dated informed consent to participate in the study has been obtained.

Exclusion Criteria

• Patients requesting not to be treated with FTD/TPI but considering other tumour treatment (e.g. palliative radiotherapy) • Concurrently active malignancies other than mCRC excluding malignancies that are disease free for more than 5 years, adequately treated basal cell or squamous cell skin cancer or carcinoma-in-situ deemed cured by adequate treatment, e.g. in situ cervical, breast or prostate cancer. • Brain or leptomeningeal metastases not controlled through surgery or radiotherapy • Active infection (i.e, body temperature ≥38°C due to infection) • Intestinal obstruction • Uncontrolled diarrhea • Uncontrolled diabetes • Pulmonary fibrosis or interstitial pneumonitis • Renal failure with CrCl <30 ml/min • Hepatic failure ≥ CTCAE version 4 Grade 3 • Cerebrovascular accident within the last 6 months • Myocardial infarction within the last 6 months, severe/unstable angina, symptomatic congestive heart failure New York Heart Association (NYHA) class III or IV • Gastrointestinal hemorrhage within last 3 months • Autoimmune disorders or history of organ transplantation that require immunosuppressive therapy. • Psychiatric disease that may increase the risk associated with study participation or study drug administration, or may interfere with the generation of QoL results. • Any other severe concomitant disease or disorder, including the presence of laboratory abnormalities, which places the subject at unacceptable risk or which could influence patient’s ability to participate in the study and his/her safety during the study or interfere with interpretation of study results • Treatment with any of the following within the specified time frame prior to first administration of FTD/TPI or Day 1 of observation cycle 1 (if no administration of FTD/TPI): a. Major surgery within prior 4 weeks (the surgical incision should be fully healed prior to study drug administration). b. Any anticancer therapy within prior 2 weeks. c. Extended field radiation within prior 4 weeks or limited field radiation within prior 2 weeks. • Participation in any other clinical trial or treatment with any experimental drug or other experimental therapy within 28 days prior to first administration of FTD/TPI or Day 1 of observation cycle 1 (if no administration of FTD/TPI); participation in a non-interventional study is permitted). • Patients who have already received FTD/TPI • Unresolved non-haematological toxicity of ≥ CTCAE version 4 Grade III attributed to prior therapies excluding anemia, alopecia, skin pigmentation and platinum induced neurotoxicity • Hypersensitivity to trifluridine, tipiracil or any of the excipients • Hereditary galactose intolerance, Lapp lactase deficiency or glucosegalactose malabsorption • Pregnant or breast-feeding female • Inappropriate for entry into this study in the judgment of the investigator • Patient has been committed to an institution by virtue of an order issued either by the judicial or the administrative authorities. • Patients possibly dependent from the investigator including the spouse,

Addresses

Primary Sponsor

Address:
Servier Deutschland GmbH
Elsenheimerstraße 53
80687 München
Germany
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail:
Contact per E-Mail
URL:
No Entry
Investigator Sponsored/Initiated Trial (IST/IIT):
No

Contact for Scientific Queries

Address:
SERVIER Deutschland GmbH
Dr. med. Bettina Barthel
Elsenheimerstraße 53
80687 München
Germany
Telephone:
+49 (0)89 570 95-175
Fax:
+49 (0)89 570 9545706
Contact per E-Mail:
Contact per E-Mail
URL:
http://www.servier.de/

Contact for Public Queries

Address:
SERVIER Deutschland GmbH
Dr. med. Bettina Barthel
Elsenheimerstraße 53
80687 München
Germany
Telephone:
+49 (0)89 570 95-175
Fax:
+49 (0)89 570 9545706
Contact per E-Mail:
Contact per E-Mail
URL:
http://www.servier.de/

Principal Investigator

Address:
SERVIER Deutschland GmbH
Dr. med. Bettina Barthel
Elsenheimerstraße 53
80687 München
Germany
Telephone:
+49 (0)89 570 95-175
Fax:
+49 (0)89 570 9545706
Contact per E-Mail:
Contact per E-Mail
URL:
http://www.servier.de/

Sources of Monetary or Material Support

Commercial (pharmaceutical industry, medical engineering industry, etc.)

Address:
Servier Deutschland GmbH
Elsenheimerstraße 53
80687 München
Germany
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail:
Contact per E-Mail
URL:
No Entry

Ethics Committee

Address Ethics Committee

Address:
Ethikkommission der Med. Fakultät der LMU
Pettenkoferstraße 8
80336 München
Germany
Telephone:
+49-89-440055191
Fax:
+49-89-440055192
Contact per E-Mail:
Contact per E-Mail
URL:
No Entry

Vote of leading Ethics Committee

Vote of leading Ethics Committee
Date of ethics committee application:
2017-06-13
Ethics committee number:
17-429
Vote of the Ethics Committee:
Approved
Date of the vote:
2017-08-30

Further identification numbers

Other primary registry ID:
No Entry
EudraCT Number:
2017-000292-83
UTN (Universal Trial Number):
No Entry
EUDAMED Number:
No Entry

IPD - Individual Participant Data

Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
No Entry
IPD Sharing Plan:
No Entry

Study protocol and other study documents

Study protocols:
No Entry
Study abstract:
No Entry
Other study documents:
No Entry
Background literature:
No Entry
Related DRKS studies:
No Entry

Publication of study results

Basic reporting

Basic Reporting / Results tables:
No Entry
Brief summary of results:
No Entry