Promoting meaningful clinical trial outcome measures for Duchenne muscular dystrophy

2016 ◽  
Vol 26 ◽  
pp. S187 ◽  
Author(s):  
H. Peay ◽  
A. Kennedy ◽  
R. Fischer ◽  
A. Bronson ◽  
P. Furlong
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Outcome Measures ◽  
Trial Outcome ◽  
Clinical Trial Outcome

T.P.1.02 The 6min walk test (6MWT) as a clinical trial outcome measure in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

2008 ◽  
Vol 18 (9-10) ◽  
pp. 739 ◽  
Author(s):  
C.M. McDonald ◽  
E.K. Henricson ◽  
J.J. Han ◽  
A.R. Nicorici ◽  
R.T. Abresch ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Muscular Dystrophy ◽  
Outcome Measure ◽  
Becker Muscular Dystrophy ◽  
Walk Test ◽  
Trial Outcome ◽  
Clinical Trial Outcome

scholarly journals Comparison of rheumatoid arthritis clinical trial outcome measures: A simulation study

2003 ◽  
Vol 48 (11) ◽  
pp. 3031-3038 ◽  
Author(s):  
Jennifer J. Anderson ◽  
James A. Bolognese ◽  
David T. Felson
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trial ◽  
Outcome Measures ◽  
Simulation Study ◽  
Trial Outcome ◽  
Clinical Trial Outcome

scholarly journals Guidelines for the conduct of clinical trials for spinal cord injury (SCI) as developed by the ICCP panel: clinical trial outcome measures

Spinal Cord ◽  
2006 ◽  
Vol 45 (3) ◽  
pp. 206-221 ◽  
Author(s):  
J D Steeves ◽  
D Lammertse ◽  
A Curt ◽  
J W Fawcett ◽  
M H Tuszynski ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Spinal Cord ◽  
Spinal Cord Injury ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Trial Outcome ◽  
Clinical Trial Outcome ◽  
Cord Injury

scholarly journals Enhancing Neurofibromatosis Clinical Trial Outcome Measures Through Patient Engagement: Lessons From REiNS

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012430
Author(s):  
Vanessa L. Merker ◽  
Andrés J. Lessing ◽  
Irene Moss ◽  
Maureen Hussey ◽  
Beverly Oberlander ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Outcome Measures ◽  
Patient Engagement ◽  
Online Survey ◽  
Trial Outcome ◽  
Clinical Trial Outcome ◽  
Patient Representatives ◽  
Trial Outcomes ◽  
Facilitators And Barriers ◽  
Patient Representative

Objective:As part of an evaluation of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration patient representative program, we surveyed REiNS members to 1) identify facilitators and barriers to involving patient representatives and 2) understand if and how involving patient representatives affected recommendations for clinical trial outcomes.Methods:We administered an anonymous, online survey to all REiNS members. Facilitators and barriers to patient representative involvement were solicited using a modified free listing technique; responses were inductively grouped into higher-order categories and ranked based on saliency score (Smith’s s). Open-ended questions assessed patient representative expectations for engagement, perceived benefits/costs of patient engagement, and patient representative contributions; responses were analyzed using conventional content analysis.Results:63/172 (37%) members responded, including 18/30 (60%) patient representatives. Providing sufficient opportunities to meaningfully engage in research tasks and cultivating a respectful, inclusive atmosphere were key facilitators to patient representatives’ satisfaction and ability to make an impact. Respondents perceived that patient representatives directly (through their input on research tasks) and indirectly (through effects on other stakeholders’ knowledge and communication style) improved the organization’s research, leading to selection of more meaningful, relevant, and feasible clinical trial outcome measures. Ongoing challenges to patient engagement include difficulty scheduling meetings and concerns about the level of scientific knowledge patient representatives needed to effectively engage.Conclusions:Involving patient representatives in REiNS improved perceived quality of neurofibromatosis clinical trial outcome measures. Negotiating sufficient opportunities to engage, fostering an inclusive atmosphere and navigating time pressures are key to effective patient engagement.

Ataluren: Clinical Trial Results in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

2016 ◽  
Vol 47 (S 01) ◽  
Author(s):  
U. Schara ◽  
C. McDonald ◽  
K. Bushby ◽  
M. Tulinius ◽  
R. Finkel ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Nonsense Mutation ◽  
Clinical Trial Results
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