Literature DB >> 26991448

A model-based approach to sample size estimation in recent onset type 1 diabetes.

Brian N Bundy1, Jeffrey P Krischer2.   

Abstract

BACKGROUND: The area under the curve C-peptide following a 2-h mixed meal tolerance test from 498 individuals enrolled on five prior TrialNet studies of recent onset type 1 diabetes from baseline to 12 months after enrolment were modelled to produce estimates of its rate of loss and variance.
RESULTS: Age at diagnosis and baseline C-peptide were found to be significant predictors, and adjusting for these in an ANCOVA resulted in estimates with lower variance.
CONCLUSIONS: Using these results as planning parameters for new studies results in a nearly 50% reduction in the target sample size. The modelling also produces an expected C-peptide that can be used in observed versus expected calculations to estimate the presumption of benefit in ongoing trials.
Copyright © 2016 John Wiley & Sons, Ltd. Copyright © 2016 John Wiley & Sons, Ltd.

Entities:  

Keywords:  C-peptide; clinical trial; type 1 diabetes

Mesh:

Substances:

Year:  2016        PMID: 26991448      PMCID: PMC5117187          DOI: 10.1002/dmrr.2800

Source DB:  PubMed          Journal:  Diabetes Metab Res Rev        ISSN: 1520-7552            Impact factor:   4.876


  8 in total

1.  Interleukin-1 antagonism in type 1 diabetes of recent onset: two multicentre, randomised, double-blind, placebo-controlled trials.

Authors:  Antoinette Moran; Brian Bundy; Dorothy J Becker; Linda A DiMeglio; Stephen E Gitelman; Robin Goland; Carla J Greenbaum; Kevan C Herold; Jennifer B Marks; Philip Raskin; Srinath Sanda; Desmond Schatz; Diane K Wherrett; Darrell M Wilson; Jeffrey P Krischer; Jay S Skyler; Linda Pickersgill; Eelco de Koning; Anette-G Ziegler; Bernhard Böehm; Klaus Badenhoop; Nanette Schloot; Jens Friis Bak; Paolo Pozzilli; Didac Mauricio; Marc Y Donath; Luis Castaño; Ana Wägner; Hans Henrik Lervang; Hans Perrild; Thomas Mandrup-Poulsen
Journal:  Lancet       Date:  2013-04-05       Impact factor: 79.321

2.  Antigen-based therapy with glutamic acid decarboxylase (GAD) vaccine in patients with recent-onset type 1 diabetes: a randomised double-blind trial.

Authors:  Diane K Wherrett; Brian Bundy; Dorothy J Becker; Linda A DiMeglio; Stephen E Gitelman; Robin Goland; Peter A Gottlieb; Carla J Greenbaum; Kevan C Herold; Jennifer B Marks; Roshanak Monzavi; Antoinette Moran; Tihamer Orban; Jerry P Palmer; Philip Raskin; Henry Rodriguez; Desmond Schatz; Darrell M Wilson; Jeffrey P Krischer; Jay S Skyler
Journal:  Lancet       Date:  2011-06-27       Impact factor: 79.321

3.  Co-stimulation modulation with abatacept in patients with recent-onset type 1 diabetes: a randomised, double-blind, placebo-controlled trial.

Authors:  Tihamer Orban; Brian Bundy; Dorothy J Becker; Linda A DiMeglio; Stephen E Gitelman; Robin Goland; Peter A Gottlieb; Carla J Greenbaum; Jennifer B Marks; Roshanak Monzavi; Antoinette Moran; Philip Raskin; Henry Rodriguez; William E Russell; Desmond Schatz; Diane Wherrett; Darrell M Wilson; Jeffrey P Krischer; Jay S Skyler
Journal:  Lancet       Date:  2011-06-28       Impact factor: 79.321

4.  Rituximab, B-lymphocyte depletion, and preservation of beta-cell function.

Authors:  Mark D Pescovitz; Carla J Greenbaum; Heidi Krause-Steinrauf; Dorothy J Becker; Stephen E Gitelman; Robin Goland; Peter A Gottlieb; Jennifer B Marks; Paula F McGee; Antoinette M Moran; Philip Raskin; Henry Rodriguez; Desmond A Schatz; Diane Wherrett; Darrell M Wilson; John M Lachin; Jay S Skyler
Journal:  N Engl J Med       Date:  2009-11-26       Impact factor: 91.245

5.  C-peptide is the appropriate outcome measure for type 1 diabetes clinical trials to preserve beta-cell function: report of an ADA workshop, 21-22 October 2001.

Authors:  Jerry P Palmer; G Alexander Fleming; Carla J Greenbaum; Kevan C Herold; Lisa D Jansa; Hubert Kolb; John M Lachin; Kenneth S Polonsky; Paolo Pozzilli; Jay S Skyler; Michael W Steffes
Journal:  Diabetes       Date:  2004-01       Impact factor: 9.461

6.  Fall in C-peptide during first 2 years from diagnosis: evidence of at least two distinct phases from composite Type 1 Diabetes TrialNet data.

Authors:  Carla J Greenbaum; Craig A Beam; David Boulware; Stephen E Gitelman; Peter A Gottlieb; Kevan C Herold; John M Lachin; Paula McGee; Jerry P Palmer; Mark D Pescovitz; Heidi Krause-Steinrauf; Jay S Skyler; Jay M Sosenko
Journal:  Diabetes       Date:  2012-06-11       Impact factor: 9.337

7.  Sample size requirements for studies of treatment effects on beta-cell function in newly diagnosed type 1 diabetes.

Authors:  John M Lachin; Paula L McGee; Carla J Greenbaum; Jerry Palmer; Mark D Pescovitz; Peter Gottlieb; Jay Skyler
Journal:  PLoS One       Date:  2011-11-10       Impact factor: 3.240

8.  Failure to preserve beta-cell function with mycophenolate mofetil and daclizumab combined therapy in patients with new- onset type 1 diabetes.

Authors:  Peter A Gottlieb; Scott Quinlan; Heidi Krause-Steinrauf; Carla J Greenbaum; Darrell M Wilson; Henry Rodriguez; Desmond A Schatz; Antoinette M Moran; John M Lachin; Jay S Skyler
Journal:  Diabetes Care       Date:  2010-01-12       Impact factor: 17.152

  8 in total
  12 in total

1.  A quantitative measure of treatment response in recent-onset type 1 diabetes.

Authors:  Brian N Bundy; Jeffrey P Krischer
Journal:  Endocrinol Diabetes Metab       Date:  2020-05-14

2.  Type 1 Diabetes TrialNet: A Multifaceted Approach to Bringing Disease-Modifying Therapy to Clinical Use in Type 1 Diabetes.

Authors:  Polly J Bingley; Diane K Wherrett; Ann Shultz; Lisa E Rafkin; Mark A Atkinson; Carla J Greenbaum
Journal:  Diabetes Care       Date:  2018-04       Impact factor: 19.112

3.  Strength in Numbers: Opportunities for Enhancing the Development of Effective Treatments for Type 1 Diabetes-The TrialNet Experience.

Authors:  Carla J Greenbaum; Cate Speake; Jeffrey Krischer; Jane Buckner; Peter A Gottlieb; Desmond A Schatz; Kevan C Herold; Mark A Atkinson
Journal:  Diabetes       Date:  2018-05-16       Impact factor: 9.461

4.  Cell type-specific immune phenotypes predict loss of insulin secretion in new-onset type 1 diabetes.

Authors:  Matthew J Dufort; Carla J Greenbaum; Cate Speake; Peter S Linsley
Journal:  JCI Insight       Date:  2019-02-21

5.  Low-Dose Anti-Thymocyte Globulin (ATG) Preserves β-Cell Function and Improves HbA1c in New-Onset Type 1 Diabetes.

Authors:  Michael J Haller; Desmond A Schatz; Jay S Skyler; Jeffrey P Krischer; Brian N Bundy; Jessica L Miller; Mark A Atkinson; Dorothy J Becker; David Baidal; Linda A DiMeglio; Stephen E Gitelman; Robin Goland; Peter A Gottlieb; Kevan C Herold; Jennifer B Marks; Antoinette Moran; Henry Rodriguez; William Russell; Darrell M Wilson; Carla J Greenbaum
Journal:  Diabetes Care       Date:  2018-07-16       Impact factor: 19.112

Review 6.  Beta cell function in type 1 diabetes determined from clinical and fasting biochemical variables.

Authors:  John M Wentworth; Naiara G Bediaga; Lynne C Giles; Mario Ehlers; Stephen E Gitelman; Susan Geyer; Carmella Evans-Molina; Leonard C Harrison
Journal:  Diabetologia       Date:  2018-08-30       Impact factor: 10.122

7.  A composite immune signature parallels disease progression across T1D subjects.

Authors:  Cate Speake; Samuel O Skinner; Dror Berel; Elizabeth Whalen; Matthew J Dufort; William Chad Young; Jared M Odegard; Anne M Pesenacker; Frans K Gorus; Eddie A James; Megan K Levings; Peter S Linsley; Eitan M Akirav; Alberto Pugliese; Martin J Hessner; Gerald T Nepom; Raphael Gottardo; S Alice Long
Journal:  JCI Insight       Date:  2019-12-05

8.  Factors Associated With the Decline of C-Peptide in a Cohort of Young Children Diagnosed With Type 1 Diabetes.

Authors:  Andrea K Steck; Xiang Liu; Jeffrey P Krischer; Michael J Haller; Riitta Veijola; Markus Lundgren; Simi Ahmed; Beena Akolkar; Jorma Toppari; William A Hagopian; Marian J Rewers; Helena Elding Larsson
Journal:  J Clin Endocrinol Metab       Date:  2021-03-08       Impact factor: 6.134

9.  Circulating Unmethylated Insulin DNA As a Biomarker of Human Beta Cell Death: A Multi-laboratory Assay Comparison.

Authors:  Cate Speake; Alyssa Ylescupidez; Daniel Neiman; Ruth Shemer; Benjamin Glaser; Sarah A Tersey; Sahar Usmani-Brown; Pamela Clark; Joshua J Wilhelm; Melena D Bellin; Kevan C Herold; Raghavendra G Mirmira; Yuval Dor; Carmella Evans-Molina
Journal:  J Clin Endocrinol Metab       Date:  2020-03-01       Impact factor: 6.134

10.  Low-Dose Anti-Thymocyte Globulin Preserves C-Peptide, Reduces HbA1c, and Increases Regulatory to Conventional T-Cell Ratios in New-Onset Type 1 Diabetes: Two-Year Clinical Trial Data.

Authors:  Michael J Haller; S Alice Long; J Lori Blanchfield; Desmond A Schatz; Jay S Skyler; Jeffrey P Krischer; Brian N Bundy; Susan M Geyer; Megan V Warnock; Jessica L Miller; Mark A Atkinson; Dorothy J Becker; David A Baidal; Linda A DiMeglio; Stephen E Gitelman; Robin Goland; Peter A Gottlieb; Kevan C Herold; Jennifer B Marks; Antoinette Moran; Henry Rodriguez; William E Russell; Darrell M Wilson; Carla J Greenbaum
Journal:  Diabetes       Date:  2019-04-09       Impact factor: 9.461

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