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A Bayesian-frequentist two-stage single-arm phase II clinical trial design

STATISTICS IN MEDICINE 2012年第19期31卷 2055-2067页

作者： Dong, Gaohong Shih, Weichung Joe Moore, Dirk Quan, Hui Marcella, Stephen Univ Med & Dent New Jersey Dept Biostat Piscataway NJ 08854 USA Novartis Pharmaceut Integrated Informat Sci IIS Integrated Hosp Care IHC E Hanover NJ USA Sanofi Aventi Biostat & Programming Bridgewater MA USA Univ Med & Dent New Jersey Dept Epidemiol Piscataway NJ 08854 USA

It is well-known that both frequentist and Bayesian clinical trial designs have their own advantages and disadvantages. To have better properties inherited from these two types of designs, we developed a Bayesianfrequentist two-stage single-arm phase II clinical trial design. This design allows both early acceptance and rejection of the null hypothesis ( H0). The measures (for example probability of trial early termination, expected sample size, etc.) of the design properties under both frequentist and Bayesian settings are derived. Moreover, under the Bayesian setting, the upper and lower boundaries are determined with predictive probability of trial success outcome. Given a beta prior and a sample size for stage I, based on the marginal distribution of the responses at stage I, we derived Bayesian Type I and Type II error rates. By controlling both frequentist and Bayesian error rates, the Bayesian-frequentist two-stage design has special features compared with other two-stage designs. Copyright (C) 2012 John Wiley & Sons, Ltd.

关键词： Bayesian Type I and Type II error rates frequentist Type I and Type II error rates predictive probability probability of early termination expected sample size

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Japan Diabetes Outcome Intervention Trial-1 (J-DOIT1), a nationwide cluster randomized trial of type 2 diabetes prevention by telephone-delivered lifestyle support for high-risk subjects detected at health checkups: rationale, design, and recruitment

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BMC PUBLIC HEALTH 2013年第1期13卷 81-81页

作者： Sakane, Naoki Kotani, Kazuhiko Takahashi, Kaoru Sano, Yoshiko Tsuzaki, Kokoro Okazaki, Kentaro Sato, Juichi Suzuki, Sadao Morita, Satoshi Izumi, Kazuo Kato, Masayuki Ishizuka, Naoki Noda, Mitsuhiko Kuzuya, Hideshi Natl Hosp Org Kyoto Med Ctr Clin Res Inst Div Prevent Med Kyoto Japan Hyogo Hlth Serv Assoc Nishinomiya Hyogo Japan Nagoya Univ Grad Sch Med Dept Gen Med Family & Community Med Nagoya Aichi 4648601 Japan Nagoya City Univ Dept Publ Hlth Grad Sch Med Sci Nagoya Aichi Japan Yokohama City Univ Dept Biostat & Epidemiol Yokohama Kanagawa 232 Japan Japan Fdn Promot Int Med Res Corp Off Strateg Outcomes Res Program Tokyo Japan Natl Ctr Global Hlth & Med Dept Diabet & Metab Med Tokyo Japan Sanofi KK Biostat Biostat & Programming Clin Sci & Operat R Tokyo Japan Natl Ctr Global Hlth & Med Diabet Res Ctr Tokyo Japan Koseikai Takeda Hosp Kyoto Japan

Background: Lifestyle modifications are considered the most effective means of delaying or preventing the development of type 2 diabetes (T2DM). To contain the growing population of T2DM, it is critical to clarify effective and efficient settings for intervention and modalities for intervention delivery with a wide population reach. The Japan Diabetes Outcome Intervention Trial-1 (J-DOIT1) is a cluster randomized controlled trial to test whether goal-focused lifestyle coaching delivered by telephone can prevent the development of T2DM in high-risk individuals in a real-world setting. This paper describes the study design and recruitment of the study subjects. Methods: For the recruitment of study subjects and their follow-up annually over 3 years, we employed health checkups conducted annually at communities and worksites. Health care divisions recruited from communities and companies across Japan formed groups as a cluster randomization unit. Candidates for the study, aged 20-65 years with fasting plasma glucose (FPG) of 5.6-6.9 mmol/l, were recruited from each group using health checkups results in 2006. Goal-focused lifestyle support is delivered by healthcare providers via telephone over a one-year period. Study subjects will be followed-up for three years by annual health checkups. Primary outcome is the development of diabetes defined as FPG >= 7.0 mmol/l on annual health checkup or based on self-report, which is confirmed by referring to medical cards. Results: Forty-three groups (clusters), formed from 17 health care divisions, were randomly assigned to an intervention arm (22 groups) or control arm (21 clusters) between March 2007 and February 2008. A total of 2840 participants, 1336 from the intervention and 1504 from the control arm, were recruited. Consent rate was about 20%, with no difference between the intervention and control arms. There were no differences in cluster size and characteristics of cluster between the groups. There were no differences

关键词： Fasting Plasma Glucose Impaired Fasting Glucose Diabetes Prevention Program Health Checkup Diabetes Prevention Study

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Impact of cabazitaxel on 2-year survival and palliation of tumour-related pain in men with metastatic castration-resistant prostate cancer treated in the TROPIC trial

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ANNALS OF ONCOLOGY 2013年第9期24卷 2402-2408页

作者： Bahl, A. Oudard, S. Tombal, B. Ozguroglu, M. Hansen, S. Kocak, I. Gravis, G. Devin, J. Shen, L. de Bono, J. S. Sartor, A. O. Univ Hosp Bristol NHS Fdn Trust Bristol Haematol & Oncol Ctr Bristol Avon England Univ Paris 05 Dept Med Oncol Hop Europeen Georges Pompidou Paris France Clin Univ St Luc Div Urol B-1200 Brussels Belgium Istanbul Univ Cerrahpasa Med Fac Dept Med Oncol Istanbul Turkey Odense Univ Hosp Dept Oncol DK-5000 Odense Denmark Masaryk Mem Canc Inst Clin Comprehens Canc Care Brno Czech Republic Hop Jour Inst Paoli Calmette Dept Med Oncol Marseille France Sanofi Dept Biostat & Programming Bridgewater MA USA Inst Canc Res Dept Drug Dev Sutton SM2 5PT Surrey England Royal Marsden NHS Fdn Trust Sutton Surrey England Tulane Univ Dept Med Tulane Canc Ctr New Orleans LA 70118 USA Tulane Univ Dept Urol Tulane Canc Ctr New Orleans LA 70118 USA

Cabazitaxel significantly improves overall survival (OS) versus mitoxantrone in patients with metastatic castration-resistant prostate cancer after docetaxel failure. We examined patient survival at 2 years and tumour-related pain with cabazitaxel versus mitoxantrone. Updated TROPIC data (cut-off 10 March 2010) were used to compare 2-year survival between treatment groups and assess patient demographics and disease characteristics. Factors prognostic for survival >= 2 years were assessed. Pain and Eastern Cooperative Oncology Group performance status were evaluated in the overall patient population. Median follow-up was 25.5 months. After 2 years, more patients remained alive following cabazitaxel than mitoxantrone [odds ratio 2.11;95% confidence interval (CI) 1.33-3.33]. Treatment with cabazitaxel was prognostic for survival >= 2 years. Demographics/baseline characteristics were balanced between treatment arms irrespective of survival. Pain at baseline and pain response were comparable between treatment groups. Average daily pain performance index was lower for cabazitaxel versus mitoxantrone (all cycles;95% CI -0.27 to -0.01;P = 0.035) and analgesic scores were similar. Grade >= 3 peripheral neuropathies were uncommon and comparable between treatment groups. Cabazitaxel prolongs OS at 2 years versus mitoxantrone and has low rates of peripheral neuropathy. Palliation benefits of cabazitaxel were comparable to those of mitoxantrone. The study was registered with (NCT00417079).

关键词： cabazitaxel symptom relief palliative care prostate cancer treatment response quality of life

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Effect of abiraterone acetate on fatigue in patients with metastatic castration-resistant prostate cancer after docetaxel chemotherapy

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ANNALS OF ONCOLOGY 2013年第4期24卷 1017-1025页

作者： Sternberg, C. N. Molina, A. North, S. Mainwaring, P. Fizazi, K. Hao, Y. Rothman, M. Gagnon, D. D. Kheoh, T. Haqq, C. M. Cleeland, C. de Bono, J. S. Scher, H. I. San Camillo Hosp Dept Med Oncol I-00152 Rome Italy Forlanini Hosp Dept Med Oncol I-00152 Rome Italy Janssen Res & Dev Oncol Los Angeles CA USA Cross Canc Inst Dept Oncol Edmonton AB T6G 1Z2 Canada Haematol Clin Australia Brisbane Qld Australia Oncol Clin Australia Brisbane Qld Australia Univ Paris 11 Inst Gustave Roussy Villejuif France Janssen Global Serv Raritan NJ USA Truven Hlth Analyt Santa Barbara CA USA Janssen Res & Dev Biostat & Programming Los Angeles CA USA Univ Texas MD Anderson Canc Ctr Dept Symptom Res Houston TX 77030 USA Inst Canc Res Div Clin Studies Div Canc Therapeut Sutton Surrey England Royal Marsden Hosp Sutton Surrey England Mem Sloan Kettering Canc Ctr Genitourinary Oncol Serv New York NY 10021 USA

Fatigue is a common, debilitating side-effect of prostate cancer and its treatment. Patient-reported fatigue was evaluated as part of COU-AA-301, a randomized, placebo-controlled, phase III trial of abiraterone acetate and prednisone versus placebo and prednisone in metastatic castration-resistant prostate cancer (mCRPC) patients after docetaxel chemotherapy. This is the first phase III study in advanced prostate cancer to evaluate fatigue outcomes using a validated fatigue-specific instrument. The Brief Fatigue Inventory (BFI) questionnaire was used to measure patient-reported fatigue intensity and fatigue interference with activities of daily life. All analyses were conducted using prespecified responder definitions of clinically meaningful changes. A total of 797 patients were randomized to abiraterone acetate and prednisone, and 398 were randomized to placebo and prednisone. Compared with prednisone alone, in patients with clinically significant fatigue at baseline, abiraterone acetate and prednisone significantly increased the proportion of patients reporting improvement in fatigue intensity (58.1% versus 40.3%, P = 0.0001), improved fatigue interference (55.0% versus 38.0%, P = 0.0075), and accelerated improvement in fatigue intensity (median 59 days versus 194 days, P = 0.0155). In patients with mCRPC progressing after docetaxel chemotherapy, abiraterone acetate and prednisone yielded clinically meaningful improvements in patient-reported fatigue compared with prednisone alone.

关键词： advanced prostate cancer palliation patient-reported outcome

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A statistical approach to central monitoring of data quality in clinical trials

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CLINICAL TRIALS 2012年第6期9卷 705-713页

作者： Venet, David Doffagne, Erik Burzykowski, Tomasz Beckers, Francois Tellier, Yves Genevois-Marlin, Eric Becker, Ursula Bee, Valerie Wilson, Veronique Legrand, Catherine Buyse, Marc IDDI Inc Houston TX 77060 USA IDDI Louvain Belgium Free Univ Brussels IRIDIA Brussels Belgium Hasselt Univ Interuniv Inst Biostat & Stat Bioinformat I BioSt Diepenbeek Belgium GlaxoSmithKline Biol Wavre Belgium Sanofi Aventis R&D Biostat & Programming Bridgewater NJ USA F Hoffmann LaRoche Ltd Basel Switzerland Translat Res Oncol TRIO Paris France Catholic Univ Louvain Inst Stat Biostat & Actuarial Sci ISBA B-1348 Louvain Belgium

Background Classical monitoring approaches rely on extensive on-site visits and source data verification. These activities are associated with high cost and a limited contribution to data quality. Central statistical monitoring is of particular interest to address these shortcomings. Purpose This article outlines the principles of central statistical monitoring and the challenges of implementing it in actual trials. Methods A statistical approach to central monitoring is based on a large number of statistical tests performed on all variables collected in the database, in order to identify centers that differ from the others. The tests generate a high-dimensional matrix of p-values, which can be analyzed by statistical methods and bioinformatic tools to identify extreme centers. Results Results from actual trials are provided to illustrate typical findings that can be expected from a central statistical monitoring approach, which detects abnormal patterns that were not (or could not have been) detected by on-site monitoring. Limitations Central statistical monitoring can only address problems present in the data. Important aspects of trial conduct such as a lack of informed consent documentation, for instance, require other approaches. The results provided here are empirical examples from a limited number of studies. Conclusion Central statistical monitoring can both optimize on-site monitoring and improve data quality and as such provides a cost-effective way of meeting regulatory requirements for clinical trials. Clinical Trials 2012;9: 705-713. http://***

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Long-acting injectable risperidone and oral antipsychotics in patients with schizophrenia - a 1-year non-interventional study

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EUROPEAN NEUROPSYCHOPHARMACOLOGY 2013年 23卷 S240-S240页

作者： Schreiner, A. Svensson, A. Wapenaar, R. Cherubin, P. Princet, P. Serazetdinova, L. Starostina, N. Zink, M. Janssen Cilag EMEA Dept Med & Sci Affairs Neuss Germany Janssen Cilag EMEA Dept Med & Sci Affairs Sollentuna Sweden Janssen Cilag BV Biostat & Programming Tilburg Netherlands Janssen Cilag EMEA Dept Med & Sci Affairs Issy Les Moulineaux France Ctr Hosp Specialise Fains Veel Dept Psychiat Fains Veel France Fed Inst Healthcare Dept Psychiat St Petersburg Russia Fed Inst Healthcare Dept Psychiat Novograd Russia Klin Psychiat & Psychotherapie Zent Inst Seel Gesundheit Mannheim Germany

P.1.g.085

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Comparison of the response to treatment between Asian and Caucasian men with benign prostatic hyperplasia: Long-term results from the combination of dutasteride and tamsulosin study

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INTERNATIONAL JOURNAL OF UROLOGY 2012年第11期19卷 1031-1035页

作者： Chung, Byung-Ha Lee, Seung Hwan Roehrborn, Claus G. Siami, Paul F. Major-Walker, Kim Wilson, Timothy H. Montorsi, Francesco Yonsei Univ Hlth Syst Dept Urol Seoul South Korea UT SW Med Ctr Dept Urol Dallas TX USA Deaconess Clin Evansville IN USA GlaxoSmithKline Clin Dev Res Triangle Pk NC USA GlaxoSmithKline Biostat & Programming Res Triangle Pk NC USA Univ Vita Salute San Raffaele Dept Urol Milan Italy

The Combination of Avodart and Tamsulosin study was a 4-year, randomized, double-blind study of the efficacy and safety of dutasteride and tamsulosin, alone or in combination, in men with moderate-to-severe benign prostatic hyperplasia. In this post-hoc investigation, we analyzed primary and secondary end-points from the Combination of Avodart and Tamsulosin study in Asian (n = 325) and Caucasian men (n = 4259). The incidence of acute urinary retention or benign prostatic hyperplasia-related surgery did not differ significantly between treatment groups in the Asian subpopulation. In Caucasian men, the incidence of acute urinary retention/benign prostatic hyperplasia-related surgery was significantly lower in the combination therapy group compared with the tamsulosin monotherapy group (P < 0.001), but not compared with dutasteride monotherapy. Combination therapy significantly increased the time to benign prostatic hyperplasia clinical progression and resulted in improved International Prostate Symptom Score, maximum urinary flow rate, quality of life, and reduced prostate volume in Asian and Caucasian men who received combination therapy compared with tamsulosin monotherapy. Combination therapy also significantly improved (P < 0.05) time to benign prostatic hyperplasia clinical progression, International Prostate Symptom Score, maximum urinary flow rate and quality of life versus dutasteride in the Caucasian subpopulation. The adverse-event profile was comparable between subpopulations. In conclusion, Asian and Caucasian men respond similarly to these treatments, despite apparent racial differences in 5a-reductase activity.

关键词： Asian benign prostatic hyperplasia Caucasian dutasteride tamsulosin

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Randomized 5-Treatment Crossover Study to Assess the Effects of External Heat on Serum Fentanyl Concentrations During Treatment With Transdermal Fentanyl Systems

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JOURNAL OF CLINICAL PHARMACOLOGY 2012年第8期52卷 1174-1185页

作者： Moore, Kenneth T. Sathyan, Gayatri Richarz, Ute Natarajan, Jaya Vandenbossche, Joris Johnson & Johnson Pharmaceut Res & Dev Clin Pharmacol B-2340 Beerse Belgium Johnson & Johnson Pharmaceut Res & Dev Clin Pharmacol Titusville NJ USA Adamas India Pharmaceut Private Ltd Bangalore Karnataka India Johnson & Johnson Pharmaceut Serv Global Med Affairs Baar Switzerland Johnson & Johnson Pharmaceut Res & Dev Biostat & Programming Raritan NJ USA

This randomized, open-label, 5-treatment, 5-sequence crossover study was designed to evaluate the effects of a heating pad on serum fentanyl concentrations with reservoir and matrix transdermal fentanyl systems. Subjects were randomized to 1 of 5 treatment sequences, receiving 5 fentanyl treatments (1 per period) for 36 hours: 25 mu g/h reservoir without heat, 25 mu g/h reservoir with heat, 25 mu g/h matrix without heat, 25 mu g/h matrix with heat, and a 50 mu g/h reservoir without heat. The 25 mu g/h systems with heat had a heating pad applied from 0 to 10 and 26 to 36 hours post application. Washout periods between treatments were 5 to 14 days. Naltrexone was given to block the opioid effects of fentanyl. Study results indicate that external heat had a similar effect on both matrix and reservoir systems, with heat applied during the first 10 hours of treatment increasing fentanyl exposure by approximately 61% to 81% at 10 hours (observed serum concentration at 10 hours) and overall exposure (area under the curve from 0 to 10 hours) by approximately 120% to 184%, but had minimal effect from 26 to 36 hours. The increased exposure observed with heat in both 25 mu g/h systems, between 0 and 10 hours, was higher than that obtained with the 50 mu g/h reservoir system applied without heat.

关键词： Transdermal fentanyl external heat serum concentration

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Optimal Sample Sizes and Go/No-Go Decisions for Phase II/III Development Programs Based on Probability of Success

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STATISTICS IN BIOPHARMACEUTICAL RESEARCH 2011年第3期3卷 463-475页

作者： Jiang, Kaihong Sanofi Aventis Biostat & Programming Bridgewater NJ 08807 USA

In the course of late-stage drug development, a phase II/III clinical development program faces a series of sample size planning and decision problems: the simultaneous sample sizes determination (SSD) across phase II and phase III trials at the program planning stage, the go/no-go decisions after phase II data have been observed, and the SSD of phase III trials incorporating phase II data. Conventionally, SSD are carried out on a trial-by-trial basis and independent of one another even though the trials are within the same program;and the go/no-go decisions are made based on the p-values, point estimates, and confidence intervals which may be, at times, ambiguous for decision making. In a combined empirical Bayesian and frequentist framework, this article proposes a probability of success (POS) function to allow an integrated approach to the SSD and go/no-go decision problems for a phase II/III program in which both the phase II and III trials share a common, normally distributed response variable. For the first time, optimal sample sizes can be determined simultaneously for a phase II/III program using the proposed POS function. As the POS function includes the conventional power function as a special case, it enjoys broader applications including SSD of phase II trials and that of phase III trials incorporating phase II data-neither of which can be handled as readily using the conventional power function.

关键词： Bayesian Due diligence Integrated Portfolio management Program-wise planning Simultaneous sample size estimation

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ABSENCE OF PHOTOSENSITIVITY POTENTIAL OF TMC435 IN HEALTHY VOLUNTEERS

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JOURNAL OF HEPATOLOGY 2012年 56卷 S458-S458页

作者： Simion, A. Janssens, L. Peeters, M. Ouwerkerk-Mahadevan, S. Spittaels, K. De Smedt, G. Akuma, S. H. Beumont-Mauviel, M. Tibotec BVBA Dept Clin Dev Beerse Belgium Janssen Pharmaceut NV Dept Biostat & Programming Janssen Res & Dev Beerse Belgium Tibotec BVBA Dept Stat Beerse Belgium

1159

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