|- candidate number||6851|
|- NTR Number||NTR2165|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd.|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||7-jan-2010|
|- Secondary IDs||WC2009-098 / 09/322 wetenschapscommissie EMGO instituut / METc VUmc|
|- Public Title||Energy cost of walking in boys with Duchenne.|
|- Scientific Title||Energy cost of walking in boys with Duchenne muscular dystrophy; a reproducibility and sensitivity study.|
|- hypothesis||1. To determine the reproducibility and sensitivity of the Energy Cost of Walking test (ECWT) in boys with Duchenne muscular dystrophy, in comparison with the reproducibility and sensitivity of the 6-Minute Walk Test (6MWT);|
2. To evaluate the 1-year course of limitations in ambulation among boys with Duchenne muscular dystrophy, as quantified with the ECWT and 6MWT.
|- Healt Condition(s) or Problem(s) studied||Duchenne muscular dystrophy, preservation of functioning, Energy cost (EC) of walking, Reproducibility|
|- Inclusion criteria||1. Children aged at least 6 years, and not older than 14;|
2. Children diagnosed with duchenne muscular dystrophy;
3. Children capable of walking independently for more than 150m without any supportive devices, and who are likely to be able to continue walking for more than 150m for at least one year.
|- Exclusion criteria||1. Children who underwent surgery (<6 months ago);|
2. Children who are unable to follow simple instructions;
3. Children who have behavioural problems that compromise participation in the study.
|- mec approval received||yes|
|- multicenter trial||yes|
|- Type||Single arm|
|- planned startdate ||1-mrt-2010|
|- planned closingdate||31-mrt-2013|
|- Target number of participants||20|
|- Primary outcome||1. From the ECWT: speed (m/min), non-dimensional speed (N_speed), net energy cost (EC) (J/kg/m), and net N_EC;|
2. From the 6MWT: speed (m/min), N_speed, and the 6-minute walking distance (6MWD) (m).
|- Secondary outcome||1. From the Life-Habits: participation in daily activities and social roles;|
2. From the Borg scale: the Rating a patient’s Perceived Exertion (RPE score);
3. From the intake and physical examination: age (yrs), height (cm), weight (kg), and leg length (cm);
4. From the Brooke scale: functional capacities.
|- Timepoints||1. February 2010 – October 2010: Recruitment of participants and baseline measurements (T0);|
2. March 2010 – October 2011: Follow up measurements (T1-T3);
3. May 2010 – November 2011: Data-analysis;
4. July 2010 – December 2011: Statistical analysis;
5. October 2010 – January 2012: Writing of scientific articles
|- Trial web site||N/A|
|- status||open: patient inclusion|
|- CONTACT FOR PUBLIC QUERIES||PhD, senior researcher M.A. Brehm|
|- CONTACT for SCIENTIFIC QUERIES||PhD, senior researcher M.A. Brehm|
|- Sponsor/Initiator ||VU University Medical Center, Department of Rehabilitation Medicine|
(Source(s) of Monetary or Material Support)
|Prosensa Therapeutics B.V.|
|- Brief summary||BACKGROUND|
Boys with Duchenne muscular dystrophy (DMD) have limitations in ambulation. The course of these limitations can be measured with the 6-minute walking test (6MWT) or with the energy cost of walking test (ECWT). However, the reproducibility and sensitivity of both these tests in boys with DMD has never been described in the literature.
1. To determine the reproducibility and sensitivity to change of the ECWT and the 6MWT in boys with DMD;
2. To evaluate the one-year course of ambulation limitations among boys with DMD, as quantified with the ECWT and 6MWT.
Ambulatory boys (age 6-14 years) with DMD (n = 20), who are capable of walking independently for more than 150m without any supportive devices, and are likely to be able to continue walking for more than 150m for at least one year.
An observational prospective cohort study with a one-year follow-up will be conducted at the outpatient clinic of the department of Rehabilitation medicine in the VU University medical center (VUmc) in Amsterdam.
To determine the reproducibility and sensitivity of the ECWT and 6MWT, measurements will be performed twice within 2 weeks (at T0 = 0 weeks (test), at T1 = 2 weeks (retest)). For evaluating the 1-year course of ambulation limitations, additional measurements will be performed at T2 = 26 weeks (6 month follow-up) and at T3 = 52 weeks (one year follow-up). Primary study parameters are speed (m/min) and EC walking (J/kg/m), as measured with the ECWT. From the 6MWT, speed (m/min) and the 6-minute walking distance (6MWD) will be calculated. Secondary study parameters are participation in daily-life activities (as measured with the Life-Habits questionnaire), and the patient’s perceived exertion score (as measured with the Borg scale).
Statistical analyses will include descriptive and correlation statistics for all measurements; reproducibility statistics based on the T0 and T1 measurements; and paired samples t-tests between the mean of measurements at T0 and T1 and measurements at T3.
|- Main changes (audit trail)|
|- RECORD||7-jan-2010 - 12-dec-2011|