Pressurized Intraperitoneal Aerosol Chemotherapy-Related Clinical Trials in the Treatment of Peritoneal Metastases

Oncology ◽  
2021 ◽  
pp. 1-10
Author(s):  
Kristjan Ukegjini ◽  
Paul Martin Putora ◽  
Marisa Guidi ◽  
Krisztian Süveg ◽  
Nikola Cihoric ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Peritoneal Metastases ◽  
Phase Iii ◽  
Current Status ◽  
Specific Information ◽  
Adjuvant Setting ◽  
Phase Iii Trials ◽  
The Impact ◽  
Palliative Setting

<b><i>Introduction:</i></b> Pressurized intraperitoneal aerosol chemotherapy (PIPAC) is a treatment option for patients with peritoneal metastases. We evaluated the current status of ongoing prospective clinical trials investigating PIPAC to provide an overview and predict trends in this field. <b><i>Methods:</i></b> All 367,494 records of clinical trials registered at ClinicalTrials.gov were searched for trials dealing with PIPAC. Active or unpublished trials were further analyzed. <b><i>Results:</i></b> In total, 22 clinical trials were identified and selected for further analyses. Most trials had a single-arm design and were phase I or II. No phase III trials were registered. Academic centers were recorded as primary sponsors in the majority of trials (63.6%). Every year, between 2 and 5 new trials were initiated. In 17 trials (81.8%), PIPAC was used in a palliative setting only, 2 trials performed PIPAC in a neoadjuvant setting, and 2 trials performed PIPAC in an adjuvant setting. Six different drugs (doxorubicin, cisplatin, oxaliplatin, nab-paclitaxel, 5-fluorouracil, and docetaxel) were used in these clinical trials. Most trials investigated the efficacy (<i>n</i> = 15) or safety (<i>n</i> = 7) of PIPAC therapies. <b><i>Conclusions:</i></b> The results of ongoing clinical trials will bring specific information on indications for PIPAC as well as the impact of PIPAC on quality of life and overall survival.

scholarly journals Comparing COVID-19 vaccines for their characteristics, efficacy and effectiveness against SARS-CoV-2 and variants of concern

2021 ◽  
Author(s):  
Fiolet ◽  
Yousra Kherabi ◽  
Conor MacDonald ◽  
Jade Ghosn ◽  
Nathan Peiffer-Smadja
Keyword(s):  
South Africa ◽  
Herd Immunity ◽  
Real Life ◽  
Cost Effective ◽  
Phase Iii ◽  
Real Life Data ◽  
National Drug ◽  
Phase Iii Trials ◽  
The Impact

Vaccines are critical cost-effective tools to control the COVID-19 pandemic. However, the emergence of more transmissible SARS-CoV-2 variants may threaten the potential herd immunity sought from mass vaccination campaigns.The objective of this study was to provide an up-to-date comparative analysis of the characteristics, adverse events, efficacy, effectiveness and impact of the variants of concern (Alpha, Beta, Gamma and Delta) for fourteen currently authorized COVID-19 vaccines (BNT16b2, mRNA-1273, AZD1222, Ad26.COV2.S, Sputnik V, NVX-CoV2373, Ad5-nCoV, CoronaVac, BBIBP-CorV, COVAXIN, Wuhan Sinopharm vaccine, QazCovid-In, Abdala and ZF200) and two vaccines (CVnCoV and NVX-CoV2373) currently in rolling review in several national drug agencies.Overall, all COVID-19 vaccines had a high efficacy against the traditional strain and the variants of SARS-CoV-2, and were well tolerated. BNT162b2, mRNA-1273 and Sputnik V had the highest efficacy (&gt;90%) after two doses at preventing symptomatic cases in phase III trials. Efficacy was ranging from 10.4% for AZD1222 in South Africa to 50% for NVX-CoV2373 in South Africa and 50 % for CoronaVac in Brazil, where the 501YV.2 and P1 variants were dominant. Seroneutralization studies showed a negligible reduction in neutralization activity against Alpha for most of vaccines, whereas the impact was modest for Delta. Beta and Gamma exhibited a greater reduction in neutralizing activity for mRNA vaccines, Sputnik V and CoronaVac. Regarding observational real-life data, most studies concerned the Pfizer and Moderna vaccines. Full immunization with mRNA vaccines effectively prevents SARS-CoV-2 infection against Alpha and Beta. All vaccines appeared to be safe and effective tools to prevent symptomatic and severe COVID-19, hospitalization and death against all variants of concern, but the quality of evidence greatly varied depending on the vaccines considered. There are remaining questions regarding specific populations excluded from trials, the duration of immunity and heterologous vaccination. Serious adverse event and particularly anaphylaxis (2.5-4.7 cases per million doses among adults) and myocarditis (3.5 cases per million) for mRNA vaccines ; thrombosis with thrombocytopenia syndrome for Janssen vaccine (3 cases per million) and AstraZeneca vaccine (2 cases per million) and Guillain-Barre syndrome (7.8 cases per million) for Janssen vaccine are very rare. COVID-19 vaccine benefits outweigh risks, despite rare serious adverse effect.

Patient-Reported Outcomes in Phase II Cancer Clinical Trials: Lessons Learned and Future Directions

2007 ◽  
Vol 25 (32) ◽  
pp. 5058-5062 ◽  
Author(s):  
Lynne I. Wagner ◽  
Lari Wenzel ◽  
Edward Shaw ◽  
David Cella
Keyword(s):  
Clinical Trials ◽  
Phase Ii ◽  
Symptom Management ◽  
Patient Reported Outcomes ◽  
Phase Iii ◽  
Cancer Clinical Trials ◽  
Phase Ii Trials ◽  
Patient Reported ◽  
Phase Iii Trials ◽  
The Impact

With increasing limits on the resources available to conduct cancer clinical trials, the inclusion of patient-reported outcomes (PROs) in treatment and symptom management trials must be prioritized. Although it has been suggested on occasion that phase III trials should take precedence over phase II trials, we argue that there is a clear and important role for PRO assessment in phase II trials going forward. To illustrate the value realized from including PROs in phase II trials, we provide case examples from cancer treatment and supportive care. The benefits of including PROs in symptom management intervention research are exemplified using phase II trials targeting cognitive impairment. The inclusion of PROs in phase II cancer clinical trials adds important information about the impact of treatment in health-related quality of life, and advances the science of PRO measurement. These contributions significantly enhance the design of phase III trials, ultimately leading to the efficient utilization of clinical trial resources.

Upfront Therapy for Myeloma: Tailoring Therapy across the Disease Spectrum

2012 ◽  
pp. 508-514 ◽  
Author(s):  
S. Vincent Rajkumar
Keyword(s):  
Phase Iii ◽  
Current Status ◽  
Limited Data ◽  
Disease Spectrum ◽  
Phase Ii Studies ◽  
Expected Outcome ◽  
Individualized Approach ◽  
Phase Iii Trials ◽  
Choice Of Therapy

Overview: The treatment of multiple myeloma is evolving rapidly. Despite the number of regimens and combinations available, there is lack of data from phase III trials demonstrating superiority of one regimen over the other in terms of overall survival and/or quality of life. The only clear survival signals have come from studies that compared newer regimens with historic ones such as melphalan-prednisone (MP) or vincristine-doxorubicin hydrochloride-thalidomide (VAD). Thus, the choice of therapy at present is often made based on physician discretion, bias, and limited data from phase II studies. Further, the regimens available have considerably different profiles in terms of safety, convenience, and cost. Given the dramatic variations in expected outcome depending on the various known prognostic factors, a risk-adapted strategy is required to provide the best available therapy to each patient based on host factors as well as prognostic markers of disease aggressiveness. This article reviews the current status of myeloma therapy and risk stratification. Results from major phase III trials are reviewed, and a risk-adapted individualized approach to therapy is presented and discussed.

A review of clinical endpoints and use of quality-of-life outcomes in phase III metastatic breast cancer clinical trials.

2012 ◽  
Vol 30 (27_suppl) ◽  
pp. 129-129
Author(s):  
Raman Tatla ◽  
Denis Landaverde ◽  
Charles Victor ◽  
David Miles ◽  
Sunil Verma
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Clinical Trials ◽  
Metastatic Breast Cancer ◽  
Metastatic Breast ◽  
Phase Iii ◽  
Clinical Endpoints ◽  
Phase Iii Clinical Trials ◽  
The Impact

129 Background: The management of metastatic breast cancer (MBC) is often considered to be palliative, with most interventions intended to relieve disease symptoms, minimize treatment effects and prolong patient survival. The impact of disease and treatment on a patient's funcitonal abilities has led to an emphasis of incorporating quality of life (QoL) measures into clinical trials. The main objective of this study is to evaluate phase III clinical trials in MBC, and assess the inclusion of QOL as an endpoint, in addition to conventional efficacy endpoints. Methods: A structured PubMed search was conducted to identify phase III clinical trials published between Jan. 1990 and Aug. 2011, evaluating systemic treatment in MBC patients. Data pertaining to treatment regimens, study endpoints and clinical findings were collected, with a particular focus on progression-based (PB), overall survival (OS), and QoL endpoints. Results: Of 520 publications identified, 122 phase III MBC clinical trials met the inclusion criteria. Of these studies, 98.4% and 95.9% included PB and OS respectively, as clinical endpoints, while QoL was assessed in only 46 (37.7%) studies. While the inclusion of QoL was not associated with the significance of PB results, there was an association between the inclusion of QoL and OS results, with 59% of significant OS studies and 32% of non-significant OS studies including QoL as a clinical endpoint (p=0.016). When stratified by treatment arm, it was found that studies favouring standard therapy were more likely to include QoL (75%, p=0.045), compared to those favouring the intervention (56%), and those without significant differences (32%). Conclusions: Although the importance of QoL is often emphasized in MBC management and treatment decisions, only one-third of identified phase III clinical trials included an assessment of QoL. About half of these trials showed no statistically significant differences in the QoL endpoint; of not, instruments of varying validity were utilized. There needs to be a greater emphasis on the evaluation of QoL, with the use of standard and validated QoL tools in MBC clinical trials, especially as we increasingly focus on progression-based endpoints.

Quality-of-life (QoL) assessment and reporting in prostate cancer: A systematic review of phase III trials published between 2012 and 2016.

2019 ◽  
Vol 37 (7_suppl) ◽  
pp. 219-219 ◽  
Author(s):  
Laura Marandino ◽  
Anna La Salvia ◽  
Cristina Sonetto ◽  
Emmanuele De Luca ◽  
Daniele Pignataro ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Phase Iii ◽  
Castration Resistant ◽  
Primary Endpoints ◽  
Phase Iii Trials ◽  
Eortc Qlq C30 ◽  
Hormone Sensitive ◽  
Eortc Qlq ◽  
The Impact

219 Background: We previously reported that QoL is not included among endpoints and QoL results are significantly underreported in a high proportion of recently published phase III trials in oncology. In this study our aim was to describe QoL prevalence and heterogeneity in QoL reporting in prostate cancer (PC) phase III trials. Methods: Whole database included all primary publications (P) of phase III trials evaluating anticancer drugs published between 2012 and 2016 by 11 major journals. For this analysis, we extracted the subset of PC trials. We analyzed QoL inclusion among endpoints, presence of QoL results and methodology of QoL analysis. Results: 35 P were identified (21 in castration-resistant [CRPC], 9 in advanced hormone sensitive [aHSPC], incl. both metastatic and biochemical relapsed, and 5 in earlier stages). In 13 (37.1%) QoL was not listed among study endpoints: 7/21 (33.3%) in CRPC, 3/9 (33.3%) in aHSPC, and 3/5 (60%) in earlier stages. Out of 22 primary P of trials including QoL among endpoints, QoL results were not reported in 9 (40.9%). Overall, no QoL data were available in 22/35 (62.9%) primary P (61.9% in CRPC, 44.4% in aHSPC and 100% in earlier disease). QoL data were not available in 15/25 (60%) trials with overall survival (OS) as primary endpoint, and in 7/10 (70%) trials with other primary endpoints. QoL data were not available in 7/16 (43.8%) trials with a positive result (25% in CRPC, 40% in aHSPC, 100% in earlier stages). In 18 trials with available QoL results (incl. secondary publications), most common QoL tools were FACT-P (11, 61.1%) and EORTC QLQ-C30 (6, 33.3%). Common methods of analysis were mean changes (6, 33.3%), mean scores over time (6, 33.3%), time to deterioration (6, 33.3%) and proportion of responders (3, 16.7%). QoL analysis was focused on the impact of toxicity in 10 cases (mostly in earlier stages), and on disease symptoms in 10 cases (mostly in CRPC). Conclusions: QoL is absent in a high proportion of recently published phase III trials in PC, although presence of QoL results is better in positive trials, especially in CRPC. Methodology of QoL analysis is heterogeneous in terms of type of instruments, analysis and presentation of results.

A review of clinical endpoints and use of quality of life outcomes in phase III metastatic breast cancer clinical trials.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e16547-e16547
Author(s):  
Raman Tatla ◽  
Denis Landaverde ◽  
Charles Victor ◽  
David Miles ◽  
Sunil Verma
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Clinical Trials ◽  
Metastatic Breast Cancer ◽  
Metastatic Breast ◽  
Phase Iii ◽  
Clinical Endpoints ◽  
Phase Iii Clinical Trials ◽  
The Impact

e16547 Background: The management of metastatic breast cancer (MBC) is often considered to be palliative, with therapy used to relieve disease symptoms, minimize treatment effects and prolong survival. The impact of disease and treatment on patients has led to an emphasis of quality of life (QoL) measures in clinical trials. This study’s primary objective is to evaluate phase III clinical trials in MBC, and assess the inclusion of QoL as an endpoint. Methods: A PubMed search was conducted to identify phase III clinical trials published from Jan 1990 to Aug 2011, which evaluated systemic therapy in MBC patients. Data pertaining to treatment regimens, study endpoints and clinical findings were collected, with a focus on progression-based (PB), overall survival (OS), and QoL endpoints. The instrument(s) used to evaluate QoL were also noted (if applicable). Results: Of 520 publications identified, 122 phase III MBC clinical trials met the inclusion criteria. Of these studies, 98.4% and 95.9% included PB and OS respectively, as clinical endpoints, while QoL was assessed in only 46 (37.7%) studies. 14 instruments were identified as QoL tools among the studies, with EORTC QLQ-C30 and FACT-B accounting for 54.7% of the instruments used. While the inclusion of QoL was not related to the significance of PB results, there was an association between the inclusion of QoL and OS results, with 59% of significant OS studies and 32% of non-significant OS studies including QoL as an endpoint (p=0.016). Stratification by treatment arm found that studies favouring standard therapy were more likely to include QoL (75%, p=0.045), compared to those favouring the intervention (56%), and those without significant differences (32%). Conclusions: Although the importance of QoL is often emphasized in MBC management, only one-third of identified phase III clinical trials included its assessment. Half of these trials showed no statistically significant differences in QoL endpoint; of note, instruments of varying validity were utilized. There needs to be greater emphasis on the evaluation of QoL, with the use of standard and validated QoL tools in MBC clinical trials, especially as we increasingly focus on progression-based endpoints.

scholarly journals Calling for improved quality in the registration of traditional Chinese medicine during the public health emergency: a survey of trial registries for COVID-19, H1N1, and SARS

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Zhuoran Kuang ◽  
◽  
Xiaoyan Li ◽  
Jianxiong Cai ◽  
Yaolong Chen ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Chinese Medicine ◽  
Traditional Chinese Medicine ◽  
Secondary Outcome ◽  
Specific Information ◽  
Clinical Trial Registry ◽  
Data Set ◽  
Diagnosis Criteria

Abstract Objective To assess the registration quality of traditional Chinese medicine (TCM) clinical trials for COVID-19, H1N1, and SARS. Method We searched for clinical trial registrations of TCM in the WHO International Clinical Trials Registry Platform (ICTRP) and Chinese Clinical Trial Registry (ChiCTR) on April 30, 2020. The registration quality assessment is based on the WHO Trial Registration Data Set (Version 1.3.1) and extra items for TCM information, including TCM background, theoretical origin, specific diagnosis criteria, description of intervention, and outcomes. Results A total of 136 records were examined, including 129 severe acute respiratory syndrome coronavirus 2 (COVID-19) and 7 H1N1 influenza (H1N1) patients. The deficiencies in the registration of TCM clinical trials (CTs) mainly focus on a low percentage reporting detailed information about interventions (46.6%), primary outcome(s) (37.7%), and key secondary outcome(s) (18.4%) and a lack of summary result (0%). For the TCM items, none of the clinical trial registrations reported the TCM background and rationale; only 6.6% provided the TCM diagnosis criteria or a description of the TCM intervention; and 27.9% provided TCM outcome(s). Conclusion Overall, although the number of registrations of TCM CTs increased, the registration quality was low. The registration quality of TCM CTs should be improved by more detailed reporting of interventions and outcomes, TCM-specific information, and sharing of the result data.

scholarly journals Improved gastrointestinal profile with diroximel fumarate is associated with a positive impact on quality of life compared with dimethyl fumarate: results from the randomized, double-blind, phase III EVOLVE-MS-2 study

2021 ◽  
Vol 14 ◽  
pp. 175628642199399 ◽  
Author(s):  
Annette Wundes ◽  
Sibyl Wray ◽  
Ralf Gold ◽  
Barry A. Singer ◽  
Elzbieta Jasinska ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Medication Use ◽  
Dimethyl Fumarate ◽  
Daily Activities ◽  
Phase Iii ◽  
Symptomatic Medication ◽  
Gi Symptoms ◽  
Symptom Intensity ◽  
The Impact

Background: Diroximel fumarate (DRF) is a novel oral fumarate approved for relapsing forms of multiple sclerosis (MS). DRF demonstrated significantly improved gastrointestinal (GI) tolerability versus dimethyl fumarate (DMF) with fewer days of Individual Gastrointestinal Symptom and Impact Scale (IGISIS) scores ⩾2, GI adverse events (AEs), and treatment discontinuations due to GI AEs. Our aim was to evaluate the impact of GI tolerability events on quality of life (QoL) for patients with relapsing–remitting MS who received DRF or DMF in EVOLVE-MS-2. Methods: A post hoc analysis was conducted in patients who were enrolled in the randomized, blinded, 5-week, EVOLVE-MS-2 [ClinicalTrials.gov identifier: NCT03093324] study of DRF versus DMF. Patients completed daily IGISIS and Global GISIS (GGISIS) eDiary questionnaires to assess GI symptom intensity and interference with daily activities and work. Results: In total, 504 patients (DRF, n = 253; DMF, n = 251) received study drug and 502 (DRF, n = 253; DMF, n = 249) completed at least one post-baseline questionnaire. With DRF, GI symptoms were less likely to interfere ‘quite a bit’ or ‘extremely’ with regular daily activities [IGISIS: DRF, 9.5% (24/253) versus DMF, 28.9% (72/249)] or work productivity [GGISIS: DRF, 6.1% (10/165) versus DMF, 11.3% (18/159)]. DRF-treated patients had fewer days with ⩾1 h of missed work (DRF, 43 days, n = 20 versus DMF, 88 days, n = 26). DMF-treated patients reported highest GI symptom severity and missed work at week 2–3 shortly after completing the titration period, which coincided with the majority of GI-related treatment discontinuations [58.3% (7/12)]. GI tolerability AEs [DRF, 34.8% (88/253); DMF, 48.2% (121/251)], concomitant symptomatic medication use [DRF, 19.3% (17/88) versus DMF, 30.6% (37/121)], and GI-related discontinuations (DRF, 0.8% versus DMF, 4.8%) were lower with DRF versus DMF. Conclusions: The improved GI tolerability with DRF translated into clinically meaningful benefits to QoL, as patients experienced less impact on daily life and work and required less concomitant symptomatic medication use. Trial registration: [ClinicalTrials.gov identifier: NCT03093324]

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