SPARE: A qualitative study investigating randomization barriers in a Selective Bladder Preservation trial (SBP) (ISCRCTN: 61126465)

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 5077-5077 ◽  
Author(s):  
C. Moynihan ◽  
E. Hall ◽  
R. Lewis ◽  
A. Birtle ◽  
G. M. Mead ◽  
...  
Keyword(s):  
Information Overload ◽  
Medical Profession ◽  
Randomised Trial ◽  
Treatment Strategies ◽  
Phase Iii ◽  
Chemotherapy Response ◽  
Framework Analysis ◽  
Bladder Preservation ◽  
Phase Iii Trial ◽  
Subsequent Phase

5077 Background: Recruitment to randomised trials is challenging, especially when treatment strategies are complex. While studies have demonstrated barriers to recruitment, few have investigated why patients accept or refuse randomisation. The SPARE feasibility study included such an investigation to highlight difficulties that might be dealt with as the study progressed and to inform procedures for a subsequent phase III trial. Methods: Patients had newly diagnosed invasive bladder cancer and were receiving neoadjuvant chemotherapy prior to invitation to a randomised trial (SPARE) that offered the possibility of radical cystectomy or SBP. Patients allocated SBP received cystectomy or radiotherapy dependent on chemotherapy response. 15 acceptors and 10 decliners to randomisation participated in the qualitative substudy. Methods included recorded transcribed interviews and a ‘framework analysis.’ Results: Both groups experienced initial confusion, ‘muddled’ communication, information overload, and lack of time leading to misunderstandings about the trial. Perceived side effects did not necessarily inform decisions. Decision to participate was seldom made unilaterally. Individual specialist input by, and continuity between, professionals and their patients, appeared to make decision making easier. Acceptors were inclined to altruism and trust in the medical profession, often agreeing to participate with minimum understanding regarding randomisation and/or equipoise and a ‘nothing to lose’ attitude as long as withdrawal from the trial was possible. Decliners tended to opt for radiotherapy, perceived as a rational choice in the context of this trial; many abhorred the thought of surgery that was perceived as undermining and a ‘treatment in reserve.’ Conclusions: Health professionals need to consider streamlining procedures and recognise patient preferences in terms of treatments offered and information received. Recognition of the complexity of communicating equipoise and randomisation may increase ‘informed consent’ levels and recruitment rates. A further qualitative substudy investigating doctor/patient consultations in real time with confidential feedback is proposed to optimise recruitment rates. No significant financial relationships to disclose.

Randomized phase II trial of gemcitabine plus S-1 combination therapy versus S-1 in advanced biliary tract cancer: Results of the Japan Clinical Oncology Group study (JCOG0805).

2012 ◽  
Vol 30 (4_suppl) ◽  
pp. 255-255 ◽  
Author(s):  
Chigusa Morizane ◽  
Takuji Okusaka ◽  
Junki Mizusawa ◽  
Atsuo Takashima ◽  
Makoto Ueno ◽  
...  
Keyword(s):  
Survival Time ◽  
Biliary Tract ◽  
Biliary Tract Cancer ◽  
Phase Iii ◽  
Correct Selection ◽  
Biliary Duct ◽  
Phase Iii Trial ◽  
Tract Cancer ◽  
Subsequent Phase ◽  
Grade 3

255 Background: Gemcitabine plus cisplatin combination (GC) therapy is the standard therapy for advanced biliary tract cancer (BTC). In previous trials, gemcitabine plus S-1 combination (GS) therapy and S-1 mono-therapy had shown considerable efficacy in patients with BTC. The aim of this trial is to evaluate the efficacy and safety of the two regimens and to determine which is more promising as a test arm regimen for a subsequent phase III trial. Methods: Chemotherapy-naive patients with recurrent or unresectable BTC (gallbladder [GB], intrahepatic biliary duct [IHBD], extrahepatic biliary duct [EHBD], ampulla of Vater [AV]), an ECOG PS of 0 - 1, and adequate organ function were randomly assigned to receive GS (gemcitabine: 1000 mg/m2, iv, days 1 and 8; S-1: 60 mg/m2, p.o., days 1 - 14, every 3 weeks) or S-1 (80 mg/m2, p.o., days 1 - 28, every 6 weeks). We assumed that %1-year survival of one regimen is 30% and that of the other regimen is more than 40%. To ensure at least 85% probability of correct selection, 98 eligible pts are required. The decision rule was that the regimen with higher %1-year survival will be considered as more promising regimen. Results: From February 2009 to April 2010, 101 pts (GB, n=38; IHBD, n=35; EHBD, n=20; AV, n=8) were randomized (GS, n=51; S-1, n=50). For the GS arm and S-1 arm, %1-year survivals were 52.9% and 40.0%, the median survival time were 12.5 and 9.0 months (hazard ratio 0.86 [95%CI 0.54-1.36]; p=0.52), and the median progression-free survival time were 7.1 and 4.2 months (0.44 [0.29-0.67]; p<0.0001). Grade 3/4 hematological toxicities were more frequent in the GS arm than in the S-1 arm, (percentage in GS/S-1 arms): neutropenia, 60.8/4.0; leukocytopenia, 29.4/2.0; hemoglobin, 11.8/4.0; and thrombocytopenia, 11.8/4.0; respectively. Although two treatment-related deaths occurred in the GS arm (pneumonitis, acute myocardial infarction), other grade 3/4 non-hematological toxicities were infrequent and reversible in both arms. Conclusions: The GS arm was superior in %1-year survival to S-1. Here we consider GS to be more promising as the test arm for a subsequent phase III trial comparing with GC.

Randomized phase II trial of gemcitabine plus S−1 combination therapy versus S−1 in advanced biliary tract cancer: Results of the Japan Clinical Oncology Group study (JCOG0805).

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. 4031-4031
Author(s):  
Makoto Ueno ◽  
Takuji Okusaka ◽  
Junki Mizusawa ◽  
Atsuo Takashima ◽  
Chigusa Morizane ◽  
...  
Keyword(s):  
Survival Time ◽  
Biliary Tract ◽  
Biliary Tract Cancer ◽  
Phase Iii ◽  
Correct Selection ◽  
Biliary Duct ◽  
Phase Iii Trial ◽  
Tract Cancer ◽  
Subsequent Phase ◽  
Grade 3

4031 Background: Gemcitabine plus cisplatin combination (GC) therapy is the standard therapy for advanced biliary tract cancer (BTC). In previous trials, gemcitabine plus S-1 combination (GS) therapy and S-1 mono-therapy had shown considerable efficacy in patients with BTC. The aim of this trial is to evaluate the efficacy and safety of the two regimens and to determine which is more promising as a test arm regimen for a subsequent phase III trial. Methods: Chemotherapy-naive patients with recurrent or unresectable BTC (gallbladder [GB], intrahepatic biliary duct [IHBD], extrahepatic biliary duct [EHBD], ampulla of Vater [AV]), an ECOG PS of 0 - 1, and adequate organ function were randomly assigned to receive GS (gemcitabine: 1,000 mg/m2, iv, days 1 and 8; S-1: 60 mg/m2, p.o., days 1 - 14, every 3 weeks) or S-1 (80 mg/m2, p.o., days 1 - 28, every 6 weeks). We assumed that %1-year survival of one regimen is 30% and that of the other regimen is more than 40%. To ensure at least 85% probability of correct selection, 98 eligible pts are required. The decision rule was that the regimen with higher %1-year survival will be considered as more promising regimen. Results: From February 2009 to April 2010, 101 pts (GB, n=38; IHBD, n=35; EHBD, n=20; AV, n=8) were randomized (GS, n=51; S-1, n=50). For the GS arm and S-1 arm, %1-year survivals were 52.9% and 40.0%, the median survival time were 12.5 and 9.0 months (hazard ratio 0.86 [95%CI 0.54-1.36]; p=0.52), and the median progression-free survival time were 7.1 and 4.2 months (0.44 [0.29-0.67]; p<0.0001). Grade 3/4 hematological toxicities were more frequent in the GS arm than in the S-1 arm, (percentage in GS/S-1 arms): neutropenia, 60.8/4.0; leukocytopenia, 29.4/2.0; hemoglobin, 11.8/4.0; and thrombocytopenia, 11.8/4.0; respectively. Although two treatment-related deaths occurred in the GS arm (pneumonitis, acute myocardial infarction), other grade 3/4 non-hematological toxicities were infrequent and reversible in both arms. Conclusions: The GS arm was superior in %1-year survival to S-1. Here we consider GS to be more promising as the test arm for a subsequent phase III trial comparing with GC.

scholarly journals Locally advanced breast cancer: report of phase II study and subsequent phase III trial

1992 ◽  
Vol 65 (5) ◽  
pp. 761-765 ◽  
Author(s):  
A Rodger ◽  
WJL Jack ◽  
PDJ Hardman ◽  
GR Kerr ◽  
U Chetty ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Advanced Breast Cancer ◽  
Phase Ii ◽  
Phase Ii Study ◽  
Locally Advanced Breast Cancer ◽  
Locally Advanced ◽  
Advanced Breast ◽  
Phase Iii ◽  
Phase Iii Trial ◽  
Subsequent Phase

scholarly journals The Trials (and Tribulations) of Complementary and Alternative Medicine in Oncology

2019 ◽  
Vol 111 (12) ◽  
pp. 1358-1360 ◽  
Author(s):  
Ethan B Ludmir ◽  
Amit Jethanandani ◽  
Walker Mainwaring ◽  
Austin B Miller ◽  
Timothy A Lin ◽  
...  
Keyword(s):  
Alternative Medicine ◽  
Complementary And Alternative Medicine ◽  
Early Phase ◽  
Phase Iii ◽  
Phase Iii Trial ◽  
Randomized Controlled ◽  
The Status ◽  
Subsequent Phase ◽  
Phase Data ◽  
Complementary And Alternative

Abstract Two decades following the creation of the Office of Cancer Complementary and Alternative Medicine at the National Cancer Institute, the status of complementary and alternative medicine (CAM) research within oncology remains opaque. To better understand the landscape of CAM studies in oncology, we identified CAM-related phase III randomized controlled trials (RCTs) through ClinicalTrials.gov and compared these CAM trials to all non-CAM oncologic RCTs. Pearson χ2 testing was used to compare proportions across groups; all tests were two-sided. Comparing the 25 identified CAM RCTs with 739 non-CAM RCTs, CAM studies were more likely to be sponsored by a cooperative group (64.0% vs 28.6%, P &lt; .001) and less likely to be industry funded (8.0% vs 76.5%, P &lt; .001). CAM trials disproportionately excluded disease-related outcomes as endpoints (8.0% vs 84.6%, P &lt; .001), were unsupported by prior early-phase data (55.0% vs 96.1%, P &lt; .001), and did not meet the primary endpoint (8.7% vs 53.0%, P &lt; .001). Given the observed relationship between encouraging pilot data and subsequent phase III trial success, we contend that future CAM RCTs may yield more promising findings if better supported by appropriately designed and well-characterized early-phase signals.

P3BEP (ANZUP 1302): An international randomized phase III trial of accelerated versus standard BEP chemotherapy for male and female adults and children with intermediate and poor-risk metastatic germ cell tumors (GCTs).

2021 ◽  
Vol 39 (6_suppl) ◽  
pp. TPS390-TPS390
Author(s):  
Shalini Subramaniam ◽  
Guy C. Toner ◽  
Martin R. Stockler ◽  
Andrew James Martin ◽  
Farzana D. Pashankar ◽  
...  
Keyword(s):  
Randomised Trial ◽  
Germ Cell Tumors ◽  
Stage I ◽  
Stage Ii ◽  
Phase Iii ◽  
Intermediate Risk ◽  
Open Label ◽  
Phase Iii Trial ◽  
Poor Risk ◽  
Adults And Children

TPS390 Background: Bleomycin, etoposide, and cisplatin (BEP) given 3-weekly x 4 is standard first-line chemotherapy for metastatic GCT categorised as intermediate risk or poor risk. Acceleration of standard regimens by shortening the cycle length improved cure rates in other cancers. We aim to determine the superiority of accelerated BEP versus standard BEP in this setting. Methods: This open label, randomised, phase III trial is conducted seamlessly in 2-stages. The primary endpoint for stage I (n=150) is complete response (CR); and for stage II (n=500) is progression free survival at 2 years (PFS2y). These sample sizes provide >80% power with a two-sided type error rate of 5% to detect an absolute improvement of 25% in the CR rate (stage II) and of 7% in the PFS2y (stage II). The target population is males and females aged 11 to 45 with intermediate-risk or poor-risk metastatic GCT of the testis, ovary, retroperitoneum, or mediastinum. Participants are randomised (1:1) to 4 cycles of standard BEP (q3w) or accelerated-BEP (q2w) with cisplatin 20mg/m2 D1-5, etoposide 100mg/m2 D1-5, bleomycin 30 KIU weekly x 12, and pegylated G-CSF D6 or filgrastim daily. Study assessments are 30 days after completing chemotherapy, 6 months from randomisation, and after completion of all post-chemotherapy treatments (e.g. surgery). Tumour tissue and baseline blood samples are collected for translational substudies including assessment of favourable versus unfavourable rates of decline in the novel biomarker miR-371. As of 13 October 2020, 140 participants have been recruited from 25 ANZ sites, 14 UK sites (led by Cambridge Clinical Trials Unit), and 140 USA sites (led by Children’s Oncology Group). The first planned interim analysis for safety (n=76) identified no safety concerns. The stage I analysis is anticipated mid-2021. This international randomised trial of chemotherapy for intermediate and poor-risk metastatic GCT is the first to include adults and children of both sexes. ClinicalTrials.gov: NCT02582697 Clinical trial information: NCT02582697.

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