Additional file 1: of Treatment with L-citrulline in patients with post-polio syndrome: study protocol for a single-center, randomised, placebo-controlled, double-blind trial

Recommended items to address in a clinical trial protocol and related documents. (PDF 57 kb

Feasibility of Using Microsoft Kinect to Assess Upper Limb Movement in Type III Spinal Muscular Atrophy Patients

<div><p>Although functional rating scales are being used increasingly as primary outcome measures in spinal muscular atrophy (SMA), sensitive and objective assessment of early-stage disease progression and drug efficacy remains challenging. We have developed a game based on the Microsoft Kinect sensor, specifically designed to measure active upper limb movement. An explorative study was conducted to determine the feasibility of this new tool in 18 ambulant SMA type III patients and 19 age- and gender-matched healthy controls. Upper limb movement was analysed elaborately through derived features such as elbow flexion and extension angles, arm lifting angle, velocity and acceleration. No significant differences were found in the active range of motion between ambulant SMA type III patients and controls. Hand velocity was found to be different but further validation is necessary. This study presents an important step in the process of designing and handling digital biomarkers as complementary outcome measures for clinical trials.</p></div

Trace plot.

<p>Movement trajectories of all 9 tracked body points in x-y dimension for a patient with a tremor and a healthy control.</p

Repeatability.

<p>The first column shows the scatter plots of three features for the two assessments of controls within the same day (angles in degrees and velocity in m/s). The second column shows the Bland-Altman plots of the same two assessments. Values are colored by individual IDs of the controls. The third column displays between visit assessments for SMA patients and controls. Measurements from the same subject are connected by lines and are colored by groups.</p

Pearson correlations for the three features from test-retest data within the same day for controls and from between visits for controls and patients.

<p>Pearson correlations for the three features from test-retest data within the same day for controls and from between visits for controls and patients.</p

Feature—disease association.

<p>Distributions of three features are displayed by group and by visit. Elbow angle and lifting angle show no group differences as opposed to velocity.</p

Learning effect.

<p>Total time spent in finishing the test for all visits is plotted with lines connecting the records from the same subject. Thick lines display the linear fit per group, with 95% confidence intervals.</p

Game scene.

<p>In the game scene, a visual skeleton figure represents the body of the subject. A flashing indicator and information below (pink) instruct the subject where to reach with which hand. On the upper left corner a counter and a timer are shown.</p

Indirect calorimetry.

<p>Indirect calorimetry results demonstrating consistently decreased rates of resting energy expenditure per kg muscle in 24 hours (A), reduced usage of carbohydrates as oxidative fuel (B), and increased usages of fatty acids (C) in oxidative energy metabolism.</p

Clinical changes.

<p>Clinical changes over treatment period of 16 weeks are shown. Four of the five patients showed improvements in 2 min walking distance (A), the MFM total score (B) and the MFM D1 subscore (C).</p