Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2011 Sep;1(9):1217-1235.
doi: 10.4155/cli.11.113.

Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings

Kate Bushby  1 , Edward Connor
Affiliations

Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings

Kate Bushby et al. Clin Investig (Lond). 2011 Sep.

Abstract

In June 2010, 25 representatives from Europe and the US met in Washington, DC, USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the context of clinical trial design and analysis. The workshop was organized in response to a September 2009 European Medicines Agency meeting where a clear directive was given that an international consensus needs to be developed that provides a foundation for age-appropriate clinical outcome measures for use in clinical trials of emerging therapeutics for DMD. Data were presented from eight multicenter longitudinal datasets, representing nearly 1900 patients over a 20-year time period. This experience confirmed the feasibility of repeated evaluations performed at multiple sites and addressed several core issues in drug development for DMD, such as the 'new' natural history in the steroidera, reliability and sensitivity of specific outcome measures, as well as disease staging and patient selection. These data form a valuable asset for academic investigators, pharmaceutical sponsors and regulatory agencies involved in DMD therapeutics. The group remains committed working together on a number of collaborative goals to support the therapeutics development effort in this orphan disease and to make these data available to stakeholders working in the field.

PubMed Disclaimer

Figures

Figure 1
Figure 1. 10-m run/walk times in Duchenne muscular dystrophy patients and controls by age with an arbitrary 120 s maximum for individuals who are unable to perform the task
The slope of the line for DMD participants is not representative of a true rate of change and is influenced by the artificial scores for non-ambulatory participants. DMD: Duchenne muscular dystrophy.
Figure 2
Figure 2
10-m run/walk velocities by age using the same data as Figure 1, showing a slope of functional decline that is representative of the population studied, inclusive of non-ambulatory individuals, and that enables comparisons with healthy controls.
Figure 3
Figure 3
Mean timed rise from supine by age.
Figure 4
Figure 4
Mean timed 10-m walk/run by age.
Figure 5
Figure 5
Mean North Star Ambulatory Assessment total score by age.
Figure 6
Figure 6
North Star Ambulatory Assessment score by age.
Figure 7
Figure 7
6-min walk test score by age.
Figure 8
Figure 8
Total score of Motor Function Measure-32 versus age in 72 Duchenne muscular dystrophy patients.
Figure 9
Figure 9
Progression of total score in 41 Duchenne muscular dystrophy ambulant and non-ambulant patients at 1-year intervals.
Figure 10
Figure 10
Proportion not using a wheelchair fulltime in Duchenne muscular dystrophy patients by age.
Figure 11
Figure 11
Natural history of Duchenne muscular dystrophy.
See this image and copyright information in PMC

References

    1. Hoffman EP, Bronson A, Levin AA, et al. Restoring dystrophin expression in Duchenne muscular dystrophy muscle. Am J Path. 2011;179(1):12–22. - PMC - PubMed
    1. Muntoni F on behalf of the meeting Steering Committee and the TREAT-NMD Network. The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009. Neuromuscul Disord. 2010;20(5):355–362. - PubMed
    1. Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy. I The design of the protocol. Muscle Nerve. 1981;4(3):186–197. - PubMed
    1. Escolar DM, Henricson EK, Mayhew A, et al. Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children. Muscle Nerve. 2001;24(6):787–793. - PubMed
    1. Vignos PJJ, Spencer GEJ, Archibald KC. Management of progressive muscular dystrophy of childhood. JAMA. 1963;184:89–96. - PubMed

LinkOut - more resources