The purpose of this study is to examine the feasibility of both the CPET and the 6MWT as outcome measures for exercise capacity in children with DMD and BMD. This study will focus on ambulatory children with DMD and BMD. These patients frequently…
ID
Source
Brief title
Condition
- Heart failures
- Musculoskeletal and connective tissue disorders congenital
- Muscle disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Feasibility:
Feasibility outcome parameters include measurement completion, acceptability
and adverse events:
Measurement completion:
Measurement completion rate is defined as the number of participants able to
complete the exercise test. A premature determined exercise test can be caused
by insufficient motivation and/or understanding of the test protocol. In this
study, measurement completion for the 6MWT is defined as a test performance
according ATS guidelines (27). Measurement completion of the CPET is defined
as a test according Rowland*s criteria on physiological responses (table 1) or
a minimal test durance of six minutes. Rowland*s criteria are subdivided in
subjective and objective criteria, where every child has to meet the first and
at least one of the latter.
A minimal accepted measurement completion rate for both tests is set at 90%.
Acceptability:
The willingness to perform the test again in the future based on experienced
burden will be assessed with a *Visual Analoge Scale*after each exercise test.
A higher score expresses less willingness to perform the test again in the
future. During the 6MWT the fall frequency, duration from fall to fall recovery
and number and duration of resting periods will be measured.
Adverse events:
Adverse events following exercise testing will be monitored:
Physical complaints will both be evaluated with a *Visual Analoge Scale* before
and after the exercise test and every morning and evening in the seven
consecutive days following the exercise test. Two day days after each exercise
test a semi-structured interview will be conducted by telephone with each
participant and his parents. Clinical features of muscle damage will be checked
and reported (appendix 1) (28,29).
(Research protocol page 15-16)
Secondary outcome
Not applicable
Background summary
Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) are
caused by deficiency or reduced expression of the muscular protein dystrophin
which leads to progressive deterioration of muscle strength and functional
abilities during childhood and cardiomyopathy. There is no curative treatment,
although the use of corticosteroids prolongs the period in which children can
walk. Several new therapeutic strategies, such as exon-skipping therapy and
physical training programmes, aim at attenuating the disease course and
optimising physical functioning and exercise capacity. To evaluate the effect
of these interventions outcome measures are needed that are feasible and have
good clinimetric properties in this specific patient group. In current
practice, the six minute walk test (6MWT) is used to evaluate exercise capacity
and has been used as an outcome measure in clinical trials. However, the
feasibility of the 6MWT in dystrophy patients can be questioned because of
reports of high fall frequency during the test and cognitive deficits in boys
with DMD that interfere with the test performance (1). The cardio pulmonary
exercise test (CPET) is considered the gold-standard to assess exercise
capacity and is used extensively in other pediatric chronic diseases (2). The
CPET has not been recently investigated in children with Duchenne and Becker
Dystrophy, partially due to fears for exercise induced muscle damage to
skeletal muscles or the heart. Importantly, several studies in adolescents and
adults with DMD and BMD have shown no adverse effects of exercise (3,4). In
order to develop reliable and valid outcome measures reflecting exercise
capacity that can be used to test efficacy of experimental treatment, more
insight in the feasibility of the CPET and the 6MWT in patients with Duchenne
Muscular Dystrophy and Becker Dystrophy is required.
(Research protocol Summary; page 7)
Study objective
The purpose of this study is to examine the feasibility of both the CPET and
the 6MWT as outcome measures for exercise capacity in children with DMD and
BMD. This study will focus on ambulatory children with DMD and BMD. These
patients frequently experience walking difficulties and fatigue performing
their activities of mobility and self-care and are involved in large
pharmaceutical and therapeutic intervention studies focused on the improvement
of physical functioning. In order to develop reliable and valid outcome
measures reflecting exercise capacity that can be used to test efficacy of
experimental treatment, more insight in the feasibility of the CPET and the
6MWT in patients with Duchenne Muscular Dystrophy and Becker Dystrophy is
required.
Questions to be answered are the following:
Feasibility outcome parameters include measurement completion, acceptability
and adverse events and are further explained in the main outcome section (25).
1. Is the 6MWT a feasible outcome measure for exercise capacity in ambulatory
children with DMD and BMD?
2. Is the CPET a feasible measure for exercise capacity in ambulatory children
with DMD and BMD?
(Research protocol page 11-12)
Study design
pilot study
Study burden and risks
One study on exercise testing in children have reported a relatively high fall
frequency in the 6MWT (1). Therefore a *safety chaser* (a person that walks
behind the child) will be used to prevent fall incidents. There is no report of
serious adverse events during or after exercise testing in this population
(4,22,36) and extended precautions are taken in this study (cardiac evaluation,
monitoring physiological responses and perceived burden) to reduce possible
risks to a minimum.
In the short run, the benefits of this study for these children and their
parents are that they will receive a comprehensive report on their level of
exercise capacity and their cardiovascular and muscular responses to exercise.
This will contribute to an optimal management of daily life functioning by both
the patients and their parents and their medical caregivers. In the long run,
it will contribute to the development of feasible and valid outcome measures
for disease progression and therapeutic interventions.
(Research protocol page 23)
Lundlaan 6
3508 AB Utrecht
NL
Lundlaan 6
3508 AB Utrecht
NL
Listed location countries
Age
Inclusion criteria
Genetically confirmed diagnosis of DMD or BMD
Ability to follow instructions regarding testing
Parental and child informed consent
6-20 years of age
Ability to walk >= 20 meter without assistive devices
Exclusion criteria
Concomitant medical problems that might intervene with the outcomes of the testing
Previous episodes of rhabdomyolysis
Reduced cardiac function that does not allow cardio pulmonary maximal exercise testing (according to pediatric cardiologist)
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL38598.041.11 |