Neoplasms

Digestive System Diseases

All Conditions

Rare Diseases

Anus Neoplasms

Rectal Neoplasms

Colorectal Neoplasms

Intestinal Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Intestinal Diseases

Anus Diseases

Rectal Diseases

Anal Cancer

Stefania Manfrida, MD

Stefania Manfrida

Stefania Mandrida

Radiochemotherapy is the standard treatment for neoplasms of the anal canal with excellent rates of local control and preservation of the anal sphincter. However, patients may experience a deterioration of quality of life related to sequelae of the treatment particularly at intestinal, anal sphincter and sexual level. Few studies to date have documented patient-reported outcomes (PROs) in this area. The aim of this observational study is to verify the quality of life (QOL) of the patients by means of self-completed questionnaires.

In recent times, the focus on collecting patient-reported outcomes (PROs) has increased in healthcare. The Food and Drug Administration defines PROs as a -measurement of any aspect of a health status of the patient that comes directly from the patient-. These includes disease symptoms, functional aspects and quality of life (QOL). The collection of PROs, combined with physician-assessed toxicity, is constantly being incorporated into clinical practice and studies to provide a more holistic picture of the impact of treatment on patients and prove useful for planning future interventions. Although the use of PROs has now become the standard for measuring quality of life of patients there are still few studies on the self-reported long-term QOL of anal cancer patients and on disease or therapy related symptoms that affect QOL. Therefore, the EORTC (European Organisation for Research and Treatment of Cancer) has recently developed the specific Quality of Life Questionnaire for anal cancer (QLQ-AN27) module to explore typical symptoms of anal cancer and its therapy. In this study, the QLQ-AN27 questionnaire will be administered together with the EORTC Quality of Life 30 (QLQ 30) in patients with anal cancer who have undergone radio-chemotherapy. The questionnaire may be administered before treatment, after treatment and during follow-ups until completion of the fifth year. This is an observational study both retrospective and prospective.

Patient Reported Outcomes (PROs) in Anal Cancer Patient Treated by Intensity Modulated Radiotherapy (IMRT).

Analysis of quality of life (QOL) by administration of PRO questionnaires to patients with cancer of the anus treated with IMRT radiochemotherapy.

Being alive without a temporary or permanent ileostomy or colostomy

Presence of disease progression inside the target volume of radiation therapy

The time from last treatment to cancer recurrence or death from any cause

Acute toxicity caused by chemoradiation according to CTCAE v. 5.0

Late toxicity caused by chemoradiation according to CTCAE v. 5.0

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Colonic Diseases

Patients' data (baseline characteristics, clinical and PROMs outcomes) will be collected according to patient-centered standardized set regarding ICHOM recommendations. Data collection and the follow-up schedule are carried out at : baseline (before any treatment or surgery), 1- and 6-month follow-up and then once a year.

Use of a standardized set to collect clinical and patient-centered data, to assess the trend of the Global Health Status overtime in colorectal cancer patients

Didier Mutter, MD, PhD

Colorectal cancer (CRC) affects men and women of all racial and ethnic groups and accounts for more than 600,000 deaths per year, globally. Current treatment options may involve surgery, chemotherapy (both adjuvant and neoadjuvant), radiation therapy, and palliative care, each with trade-offs between disease management and patients' quality of life. Unfortunately, significant disparity exists in the quality of care and there is a need for standardization to ensure high-value health care for all patients.

This study evaluates the introduction of a Value-Based Health Care (VBHC) patient-centered framework in CRC treatments. VBHC is an innovative approach that aims to improve health care by identifying and systematically measuring both medical and patient-reported health care outcomes and costs. By applying sets of disease-specific outcomes measurements, health care providers (HCP) can compare care strategies and make informed choices with regard to optimization of care, necessary investments and possible cost reductions.

The adoption of a VBHC patient-centered approach may have a significant impact on therapeutic areas constituting a major disease and cost burden for the global health care, such as CRC. It has the potential to improve cancer care planning, monitoring, and management of patients, by promoting better communication and shared decision making by patients and HCP.

A patient-reported outcome measurement (PROM) is defined as any report about a health condition and its treatment that comes directly from the patient. The use of a tailored pathway including PROMs improve both quality of life (QoL) and survival in cancer patients. Another essential requirement of VBHC approach is the outcome monitoring, to allow HCP accessing to evidence-based, simplified information on the hospital clinical practice and potentially increase health value for both patients and HCP. For patients with CRC, the International Consortium for Health Outcomes Measurement (ICHOM) developed a comprehensive patient-centered outcomes measurement set that could be used in the clinical practice to monitor patients' status.

The purpose of this study is to evaluate the introduction of a VBHC approach in CRC treatments, using a validated VBHC set of clinical outcomes and PROMs, to understand which practice would be most effective in achieving patient-centered care. The underlying hypothesis is that a periodic analysis of these outcomes could increase health value for both patients and HCPs.

Colorectal cancer (CRC) affects men and women of all racial and ethnic groups and accounts for more than 600,000 deaths per year, globally. Current treatment options may involve surgery, chemotherapy (both adjuvant and neoadjuvant), radiation therapy, and palliative care, each with trade-offs between disease management and patients' quality of life. Unfortunately, significant disparity exists in the quality of care, and so outcomes delivered across institutions for each treatment modality, suggesting that unwarranted variation in the provision of care occurs for patients with CRC. Hence, there is a need for standardization to ensure high-value health care for all patients, regardless the hospital where they present.

This study evaluates the introduction of a Value-Based Health Care (VBHC) patient-centered framework in CRC treatments. VBHC is an innovative approach that aims to improve health care by identifying and systematically measuring both medical and patient-reported health care outcomes. By applying sets of disease-specific outcomes measurements, health care providers (HCP) can compare care strategies and make informed choices with regard to optimization of care, necessary investments and possible cost reductions. The need to move toward a more patient-centered approach has been highlighted to be crucial in the context of quality care. Patient-centered care is a key component of a health system that ensures that all patients have access to the outcomes that matter for them.

The adoption of a VBHC patient-centered approach may have a significant impact on therapeutic areas constituting a major disease and cost burden for the global health care, such as CRC. CRC causes significant morbidity and mortality, being the third most common cancer and the fourth most common cause of cancer deaths worldwide and the second most common cause of cancer deaths in Europe. The 5-years relative survival rate is 47% in Europe and 60% in the US. Moreover, the economic burden of CRC is expected to increase in the future, partly due to changing demographics and the introduction of new and resource-demanding treatments and screening methods. In this scenario, the introduction of VBHC-based clinical pathways has the potential to improve cancer care planning, monitoring, and management of patients, by promoting better communication and shared decision making by patients and healthcare providers.

Research Hypothesis In general, an essential requirement to be able to apply a VBHC approach is the clear definition of the disease-specific outcomes, both clinical and patient-reported. Clinical outcomes can be measured by activity data such as complication rates, type of surgery, re-hospitalization rates, or by agreed scales and other forms of measurement. A patient-reported outcome measurement (PROM) is defined as any report about a health condition and well-being (quality of life) that comes directly from the patient using a self-reported measure, without interpretation of the patient's response by a physician or anyone else.

PROMs are being advocated for use in routine clinical cancer practice and for the early detection of patient distress. The use of a tailored pathway including PROMs has been shown to improve both quality of life (QoL) and survival in cancer patients. The QoL, by assessing physical function and symptoms such as pain, and social functioning are known to be independent prognostic factors for overall survival of metastatic CRC patients.

Another essential requirement of VBHC approach is the outcome monitoring, to allow HCP accessing to evidence-based, simplified information on the hospital clinical practice and potentially increase health value for both patients and HCPs. It has been demonstrated that facilitation of data interpretation can improve medical care quality. In particular, a Dutch nationwide study showed that, by providing continuous feedback of benchmarked performance information to colorectal surgeons, clinical outcomes of patients undergoing colorectal cancer surgery improves and cost decreases.

For patients with CRC, the International Consortium for Health Outcomes Measurement (ICHOM) developed a comprehensive patient-centered outcomes measurement set that could be used in the clinical practice to monitor patients' status. Therefore, an exploratory study collecting ICHOM set of data could provide useful information to evaluate the feasibility, benefits and barriers of a VBHC approach for CRC.

The purpose of this study is then to evaluate the introduction of a VBHC approach in CRC treatments, using a validated VBHC set of clinical outcomes and PROMs, to understand which practice would be most effective in achieving patient-centered care. The underlying hypothesis is that a periodic analysis of these outcomes could increase health value for both patients and HCPs. The study will first explore the most accurate feasible trend of the Global Health Status (Quality of Life evolution) of patients but also clinical outcomes and other PROMs trends over time, the cause(s) of these trends, and whether any outcomes' predictors can be found. Furthermore, a general assessment of the impact of the VBHC approach on CRC treatments will be performed, in terms of general satisfaction and knowledge in medical care. Finally, cost associated with performed procedures and complications will be estimated ambispectively using the Time-driven Activity-based Costing (TDABC) methodology, where applicable. Additional participating sites shall be free to apply the same methodology or to measure costs derived from traditional hospital cost accounting systems.

FOcUs on Colorectal CAncer oUtcomes: Long-Term Study

The EORTC-QLQ-C30 (European Organisation for Research and Treatment of Cancer - Quality of Life Questionnaire - C30) is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / Quality of Life (QoL) scale, and six single items.

The global health status / Quality of Life scale runs from 1 (very poor) to 7 (excellent) and the others from 1 (not at all) to 4 (very much). All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus:

* a high score for a functional scale represents a high / healthy level of functioning,
* a high score for the global health status / QoL represents a high QoL,
* but a high score for a symptom scale / item represents a high level of symptomatology / problems.

The EORTC-QLQ-C30 questionnaire is composed of both multi-item scales and single-item measures:

* the global health status score (Quality of Life), runs from 1 (very poor) to 7 (excellent);
* the functional scores (Physical function, Emotional function, Anxiety...), runs from 1 (not at all) to 4 (very much).

The trend of each category of PROMS given by the EORTC-QLQ-C30 will be evaluated in four groups of patients: tumor on the right and transverse part of the colon; tumor on the left part of the colon; rectal tumor; metastatic colorectal cancer.

The symptom scores (Fatigue, Nausea and vomiting, Pain ...), running from 1 (not at all) to 4 (very much), and given by EORTC (European Organisation for Research and Treatment of Cancer) will be evaluated by the use of the EORTC-QLQ-CR29 questionnaire.

The trend of each category of PROMS given by the EORTC-QLQ-CR29 in four groups of patients: tumor on the right and transverse part of the colon; tumor on the left part of the colon; rectal tumor; metastatic colorectal cancer.

Trend over time will be analysed by means of Generalized Estimating Equation models as appropriate to account for repeated measures using patient as the subject. Baseline characteristics and outcomes will be assessed regarding ICHOM recommendations:

* descriptive analysis of baseline characteristics (patients' demographic-, clinical-, tumor- and treatment factors, and treatment variables)
* descriptive analysis of care status disutility (short-term treatment complications)
* evaluation of the degree of health (Quality of Life, functioning, and long-term adverse effects) : from 1 (very poor) to 7 (excellent)
* evaluation of the survival and disease control (number of overall survival, disease-specific survival, recurrence, and progression-free survival)
* descriptive analysis of the quality of death (quality of end of life care (last 30 days of life))
* evaluation of the symptoms score using EORTC-QLQ-C30 and CR29: from 1 (not at all) to 4 (very much)

The average number of missing clinical outcomes and PROMs data collected using the standardized set developed by the ICHOM for CRC at each follow-up visit will be calculated and used to determine the level of relevance and confidence for each variable

The proportion of patients involved in this study compared to the whole cohort of patients who have been treated for a colorectal cancer at Strasbourg's University Hospital and the proportion of patient who refused to participate to the study.

The number of follow-up forms and their timelines, by patient

The outcomes and PROMs will be classified into the three main classes of missing pattern, i.e. univariate, monotone, and arbitrary, to select actions for future data collection

Predictors of clinical outcomes and PROMs will be investigated by performing univariate and multivariate statistical analysis, when appropriate

Control charts will be used to monitor clinical outcomes and PROMs in both Variable data charts as individual, average and range charts and Combination Charts (x-bar standard deviation, process capability, etc.).

Control charts will have the following features:

* Data points could be averages of subgroup or individual measurements plotted on the x and y axis and joined by a line. Time is always on the x-axis.
* The Average or Center Line is the average or mean of the data points and is drawn across the middle section of the graph.
* The Upper Control Limit (UCL) is drawn above the centerline. This is equal to 3 or 2 times of the standard deviation line.
* The Lower Control Limit (LCL) is drawn below the centerline. This is equal to 3 or 2 times of the standard deviation line.

The impact of a VBHC approach on patient will be qualitatively evaluated by collecting and analysing the annual survey responses from patients on their perception and satisfaction of a VBHC approach: scale from "very satisfied" to "not satisfied".

The impact of a VBHC approach on HCPs will be qualitatively evaluated by collecting and analysing the annual interview responses from HCPs concerning the satisfaction (scale from "very satisfied" to "not satisfied"), the introduction of new recommendations during the follow-up period (e.g., addition or deletion of a specific follow-up visit) and knowledge in clinical practice (e.g., inclusion of pain specialists or psychologists). Survey data will be reported using summary statistics and supporting graphical representations.

At the hepato-digestive pole, Strasbourg's University Hospital, healthcare costs will be measured by applying the Time-driven Activity-based Costing (TDABC) methodology. Other eventual additional participating sites will be free to apply the same methodology or to measure costs derived from traditional hospital cost accounting systems. An assessment of the outcomes of the two different approaches could be made between different methodologies

IHU Strasbourg

Respiratory Tract (Lung and Bronchial) Diseases

Infections

Symptoms and General Pathology

Lung Neoplasms

Carcinoma, Non-Small-Cell Lung

Pneumonia

Communicable Diseases

Respiratory Tract Infections

Lung Diseases

Respiratory Tract Diseases

Antineoplastic Agents

All Drugs and Chemicals

Durvalumab

Patients will be enrolled after their treating physician has prescribed durvalumab and before they start durvalumab treatment. Patients will receive mobile and wearable devices alongside their durvalumab treatment without any additional interventions.

Using a spirometer, an armband, and a tablet to collect data.

Multiparametric Mobile Technology

A study of whether mobile devices can improve the detection of pulmonary AEs (including pneumonitis) in stage III NSCLC patients post-CRT, while on durvalumab.

Patients undergoing post-CRT treatment for lung cancer with consolidation durvalumab can experience pulmonary AEs that could become severe if not recognized and treated in time.

Data collected will be used to evaluate the likelihood of early detection of pulmonary AEs in unresectable Stage III NSCLC patients on durvalumab. This project seeks to understand if multiparametric mobile technology collecting patient reported outcomes, vital signs, and respiratory function, integrate well into a patients daily life and aid physicians in early detection of pulmonary AEs.

Mobile Devices to Detect Early Pneumonitis in Stage III NSCLC Patients on Durvalumab.

Prospective, Interventional Pilot Study of Mobile Devices and Digital Applications to Detect Early Pneumonitis and Other Pulmonary Adverse Events in Unresectable Stage III Non-Small Cell Lung Cancer Patients on Durvalumab

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

An AE was occurrence of an undesirable medical condition or the deterioration of a pre-existing medical condition following or during exposure to a pharmaceutical product or device, whether or not considered causally related to the product or device medical occurrence in a participant. The TEAEs of pneumonitis were defined as any pneumonitis event that occurred or worsened at any time after the start of administration of the first dose of durvalumab and through 30 days after the last dose of durvalumab. Severity (intensity of any event) was assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse events (CTCAE) v5. The AEs were assigned to a Grade from 1 through 5 as follows: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe or medically significant but not immediately life-threatening requiring hospitalization; Grade 4: Life-threatening consequences; Grade 5: Death due to any AE.

Pulmonary TEAEs were defined as any pulmonary AEs that occurred or worsened at any time after the start of administration of the first dose of durvalumab and through 30 days after the last dose of durvalumab. Number of participants with permanent discontinuation of durvalumab due to pulmonary TEAEs including pneumonitis are reported.

The overall duration of durvalumab treatment, while participants were a part of this wearable study, was calculated as end date of durvalumab treatment minus first dose of durvalumab (Day 1) plus 1.

Number of participants who discontinued durvalumab early due to any reason are reported.

Treatment interruptions were defined as at least 1 temporary withholding of durvalumab treatment. Treatment withheld was defined as temporarily withheld of durvalumab recorded in case report form. Short interruptions defined as the durvalumab infusion interruption during the administration recorded in CRF in a single visit were excluded from the analysis. Due to data issue, the reason for treatment withheld was not captured in the database.

The overall duration of durvalumab treatment interruption was calculated as the sum of the duration of each treatment withheld/resumed. The duration of interruption included only treatment withheld. Short interruption which resumed during the same visit was not included in the calculation for duration of interruption.

Pulmonary TEAEs were defined as any pulmonary AEs that occurred or worsened at any time after the start of administration of the first dose of durvalumab and through 30 days after the last dose of durvalumab.

Pulmonary TEAEs were defined as any pulmonary AEs that occurred or worsened at any time after the start of administration of the first dose of durvalumab and through 30 days after the last dose of durvalumab. Duration of pulmonary TEAEs was calculated as end date of pulmonary TEAE minus onset date of pulmonary TEAE plus 1. For AEs that were missing an end date, the data cut-off date was used as the AE end date for calculation of AE duration.

Pneumonitis TEAEs were defined as any pneumonitis AEs that occurred or worsened at any time after the start of administration of the first dose of durvalumab and through 30 days after the last dose of durvalumab.

Duration of development of Grade 3 to 5 pneumonitis AEs is defined as the period from Day 1 to earliest of each grade of pneumonitis AE (grade 3, grade 4, and grade 5). Severity (intensity of an event) was assessed using the NCI-CTCAE v5. AEs were assigned to a Grade from 1 through 5 as follows: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe or medically significant but not immediately life-threatening requiring hospitalization; Grade 4: Life-threatening consequences; Grade 5: Death due to any AE.

The EORTC QLQ-C30 consisted of 30 questions and included functional scales (FS) (items 1-7 and items 20-27), symptom scales (items 8-19 and item 28) and global measure of health status (GHS) (items 29-30). The scale ranged from 1-4 for most outcome measures of systems, with 1 rated as "not at all" and 4 rated as "very much". Scores were averaged and transformed to 0 to 100, a high score for functional scales/GHS represented better functioning ability/QoL, whereas a high score for symptom scales represented stronger symptoms/worse QoL. Participants with pneumonitis TEAEs with causal relationship with durvalumab are presented. Time point 1: prior to occurrence of initial pneumonitis AE; Time point 2: at same time point as initial pneumonitis AE; Time point 3: when highest CTCAE grade of pneumonitis AE occurred. Baseline was defined as the date of informed consent for this parameter.

The EORTC QLQ-LC13 was a disease-specific 13-item questionnaire for lung cancer used in conjunction with the EORTC QLQ-C30. It comprised both multi-item and single-item measures of lung cancer associated symptoms (LCAS) (items 31-35 and items 40-42), treatment related side effects (TREF) (items 36-39) and pain medication (item 43). The scale ranged from 1-4 for most outcome measures of systems, 1 rated as "not at all" and 4 rated as "very much". Scores were averaged and transformed to 0 to 100, higher scores for LCAS and TREF: greater level of symptoms/worse QoL and higher scores for pain medication: better pain relief from medication. Participants with pneumonitis TEAEs with causal relationship with durvalumab are presented. Time point 1: prior to occurrence of initial pneumonitis AE; Time point 2: at same time point as initial pneumonitis AE; Time point 3: when highest CTCAE grade of pneumonitis AE occurred. Baseline was defined as the date of informed consent for this parameter.

AstraZeneca

The SAF included all enrolled participants who took at least 1 dose of durvalumab.

Participants were trained on the mobile applications and received mobile and wearable devices alongside their durvalumab treatment without any additional interventions. Participants received durvalumab for up to 12 months or until confirmed disease progression, permanent discontinuation of durvalumab, the initiation of alternative cancer therapy, unacceptable toxic events, death, or withdrawal of consent, whichever was sooner.

All Participants

Age, Continuous

Sex: Female, Male

Race and Ethnicity were not collected from any participant.

Race and Ethnicity Not Collected

The Safety Analysis set (SAF) included all enrolled participants who took at least 1 dose of durvalumab.

This pilot trial was terminated early due to the Coronavirus Disease-2019 (COVID-19) pandemic, shortage of site staff, and limited access to sites resulting in participant enrollment and retention challenges. The trial was not terminated early due to any safety concerns.

Global Clinical Lead

Grade 2

Grade 3

Number of Participants With Treatment-Emergent Adverse Event (TEAE) of Pneumonitis by Grade

Number of Participants With Permanent Discontinuation of Durvalumab Due to Pulmonary TEAEs

Duration of Durvalumab Treatment

Number of Participants With Early Discontinuation of Durvalumab

Number of Participants With Treatment Interruptions

Duration of Durvalumab Treatment Interruption

Number of Participants With Pulmonary TEAEs Excluding Pneumonitis

The SAF included all enrolled participants who took at least 1 dose of durvalumab. Only those participants with pulmonary TEAEs excluding pneumonitis were included in this analysis.

Duration of Pulmonary TEAEs Excluding Pneumonitis

The SAF included all enrolled participants who took at least 1 dose of durvalumab. Only those participants with pneumonitis TEAEs were analyzed.

Number of Participants Who Received Prescription Medication to Manage Pneumonitis TEAEs

Duration of Prescription Medication Received by Participants to Manage Pneumonitis TEAEs

Data was not collected due to early termination of the trial.

Duration of Development of Grade 3 to Grade 5 TEAEs, Including Pneumonitis

FS; Time point 1

FS; Time point 2

FS; Time point 3

Symptom scale; Time point 1

Symptom scale; Time point 2

Symptom scale; Time point 3

GHS; Time point 1

GHS; Time point 2

GHS; Time point 3

The SAF included all enrolled participants who took at least 1 dose of durvalumab. For each symptom scale, only those participants with data available were included in the analysis.

Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 Items (EORTC QLQ-C30) in Participants With Pneumonitis TEAEs

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Patient Reported Outcomes (PROs) in Anal Cancer Patient Treated by Intensity Modulated Radiotherapy (IMRT).	NCT06364579	Anal Cancer Pat...		18 Years -	Fondazione Policlinico Universitario Agostino Gemelli IRCCS
FOcUs on Colorectal CAncer oUtcomes: Long-Term Study	NCT03965325	Colorectal Canc...	Use of a standa...	18 Years -	IHU Strasbourg
Patient Reported Outcomes (PROs) in Anal Cancer Patient Treated by Intensity Modulated Radiotherapy (IMRT).	NCT06364579	Anal Cancer Pat...		18 Years -	Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Mobile Devices to Detect Early Pneumonitis in Stage III NSCLC Patients on Durvalumab.	NCT04381494	Unresectable St...	Multiparametric...	18 Years - 130 Years	AstraZeneca
FOcUs on Colorectal CAncer oUtcomes: Long-Term Study	NCT03965325	Colorectal Canc...	Use of a standa...	18 Years -	IHU Strasbourg

Spots Global Cancer Trial Database for pros

Every month we try and update this database with for pros cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for pros

Every month we try and update this database with for pros cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for pros cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.