The Impact of Charcot-Marie-Tooth Disease in the Real World
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|ClinicalTrials.gov Identifier: NCT03782883|
Recruitment Status : Recruiting
First Posted : December 20, 2018
Last Update Posted : December 20, 2018
|Condition or disease|
The registry uses Vitaccess' MyRealWorld™ digital real-world evidence platform and has been developed in collaboration with CMT experts, Patient Advocacy Organizations (PAOs) and the biopharmaceutical company Pharnext.
Eligible participants install a study app on their smartphone. Researchers access aggregated, anonymised data via a cloud-based research portal.
The platform provides benefits to participants, which it is hoped will encourage persistence with data submission; these include options to upload electronic documents such as scans, a clinically-validated Knowledge feature, and access an online CMT community.
The aggregated data are available in close to real time via "dashboards" and can be analysed according to a number of pre-set criteria (e.g. disease stage, age, geographic location).
State-of-the-art technologies and security policies are used in the platform to ensure industry-standard data storage and privacy for all users. Participants' personally identifiable information will remain confidential at all times, and researchers will not be able to identify individuals.
The study is ethics-approved in all scope countries and led by a Scientific Advisory Board comprising representatives from international patient associations, patients and key opinion leaders from each country, and study leads from both Pharnext Société Anonyme (SA) and Vitaccess Ltd.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||2000 participants|
|Observational Model:||Ecologic or Community|
|Target Follow-Up Duration:||30 Months|
|Official Title:||The Impact of Charcot-Marie-Tooth Disease in the Real World|
|Actual Study Start Date :||October 9, 2018|
|Estimated Primary Completion Date :||April 9, 2021|
|Estimated Study Completion Date :||April 9, 2021|
- EuroQoL 5-dimension 5-level (EQ-5D-5L) [ Time Frame: Monthly throughout 2.5 year study duration ]
The EQ-5D-5L comprises two parts - the EQ-5D-5L descriptive system and the EQ Visual Analogue Scale (EQ VAS).
The descriptive system comprises 5 dimensions (mobility, self care, usual activities, pain/discomfort, and anxiety/depression), each with 5 levels (no problems, slight problems, moderate problems, severe problems, and extreme problems - i.e., higher scores represent worse health). The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state.
The EQ VAS records the respondent's self-rated health on vertical, visual analogue scale with endpoints labelled 'the best health you can imagine' and 'the worst health you can imagine'. Higher scores represent better self-perceived health.
- Work Limitations Questionnaire (WLQ) [ Time Frame: Monthly throughout 2.5 year study duration ]The WLQ measures the impact of CMT on participants' work ability and productivity. The measure comprises 25 items that ask respondents to rate their level of difficulty or ability to perform specific job demands, aggregated into four domains: time management, physical demands, mental-interpersonal demands, and output demands. Domain scores range from 0 to 100 and the recall period is the previous 2 weeks. WLQ domain scores can be converted into an estimate of productivity loss using an algorithm.
- Brief Fatigue Inventory (BFI) [ Time Frame: Monthly throughout 2.5 year study duration ]The BFI assesses the fatigue severity in patients with CMT. The measure comprises 10 items, although the first item asks about usual fatigue over the past week (with the respondent answering "yes" or "no") and is not included in the overall score. The remaining nine items assess general fatigue within two subscales: fatigue severity (three items), and the impact of fatigue on daily functioning (six items). The measure uses a 10-point numeric rating scale, and a recall period of 24 hours. A global fatigue score can be calculated by averaging all nine items.
- Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Intensity 3a and Interference 8a [ Time Frame: Monthly throughout 2.5 year study duration ]
The PROMIS pain intensity measure includes two items that assess pain intensity over the last 7 days (average and worst pain), and one for pain intensity "right now"; each scores using a 5-point scale. Scores range from 2-10. Higher scores represent worst pain. This measure is generic rather than disease-specific.
The PROMIS pain interference measure assesses the extent to which pain hinders engagement with social, cognitive, emotional, physical and recreational activities, sleep, and enjoyment in life over the last 7 days using a 5-point scale. Scores range from 8-40. Higher scores represent worse interference. This measure is generic rather than disease-specific.
- Bespoke questionnaire for this study [ Time Frame: Monthly throughout 2.5 year study duration ]Two cramp-specific items were developed for inclusion in the survey, measuring cramp frequency and intensity. The cramp frequency item asks "In the past 7 days, how many days did you experience cramp?" and has five possible responses: had no cramp, 1-2 days, 3-4 days, 5-6 days, every day. The cramp intensity item asks "In the past 7 days, how intense was your cramp at its worst?" and has five possible responses: had no cramp, mild, moderate, severe, very severe. Higher scores on both items represent greater cramp frequency and intensity respectively.
- Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance 8a [ Time Frame: Monthly throughout 2.5 year study duration ]The PROMIS sleep disturbance measure assesses self-reported perceptions of sleep quality, sleep depth, and restoration associated with sleep over the last 7 days using a 5-point scale. Scores range from 8-40. Higher scores represent worse sleep disturbance. This measure is generic rather than disease-specific.
- Falls Efficacy Scale - International (FES-I) [ Time Frame: Every 3 months throughout 2.5 year study duration ]The FES-I measures the level of concern about falling during social and physical activities inside and outside hte home, whether or not the person actually does the activity. The "usual" level of concern is measured on a four-point Likert scale (1 = not at all concerned to 4 = very concerned), with a time frame of "usual". Scores range from 16-64. Higher scores represent greater concern about falling.
- Lower Extremity Function Scale (LEFS) [ Time Frame: Every 3 months throughout 2.5 year study duration ]The LEFS evaluates difficulties because of lower limb problems in 20 activities, including work/school activities, hobbies, moving around the home, dressing, lifting, standing, sitting, walking, and running. The level of difficulty is assessed for "today" using a 5-point Likert scale (0 = extreme difficulty or unable to perform activity; 5 = No difficulty). Lower scores represent greater difficulties experience because of lower limb problems.
- QuickDash (Disabilities of the Arm, Shoulder and Hand) [ Time Frame: Every 3 months throughout 2.5 year study duration ]The QuickDash measure uses 11 items to measure physical function and symptoms in people with any or multiple musculoskeletal disorders of the upper limb. Six questions relate to the ability to perform six activities over the last week, ranging from opening a jar, carrying, washing and recreational activities; two questions relate to impact on social and daily activities; two questions ask the person to rate the severity of pain and tingling in the arms, shoulder or hand, and one question relates to the effects of pain on sleep. All questions are rated 1-5 (no difficulty/none/not at all through to unable/extreme difficulty). Scores range from 11-55. Higher scores represent greater difficulties with physical function and symptoms.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03782883
|Contact: Mark JW Larkin, PhD||+33 645 900 email@example.com|
|Contact: Helen V Williams, DPhil||+44 7732 firstname.lastname@example.org|
|Oxford, United Kingdom, OX1 1BY|
|Contact: Mark JW Larkin, PhD +441865818983 email@example.com|
|Contact: Helen V Williams, DPhil +441865818983 firstname.lastname@example.org|
|Principal Investigator:||Mark JW Larkin, PhD||Vitaccess Ltd|