Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 337 for:    Charcot Marie Tooth

The Impact of Charcot-Marie-Tooth Disease in the Real World

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03782883
Recruitment Status : Recruiting
First Posted : December 20, 2018
Last Update Posted : December 20, 2018
Sponsor:
Collaborators:
Pharnext SA
ACMT Network for Charcot-Marie-Tooth disease
Asociación Madrileña de Enfermedades Neuromusculares (ASEM Madrid)
Charcot-Marie-Tooth Association
Charcot-Marie-Tooth UK
CMT France
Hereditary Neuropathy Foundation
Information provided by (Responsible Party):
Vitaccess Ltd

Brief Summary:
An observational, non-interventional registry study to collect real-world data from people living with Charcot-Marie-Tooth disease (CMT) and its treatment, which will be available to researchers to further the knowledge of Charcot-Marie-Tooth disease and improve patient care.

Condition or disease
Charcot-Marie-Tooth Disease

Detailed Description:

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.


Layout table for study information
Study Type : Observational [Patient Registry]
Estimated Enrollment : 2000 participants
Observational Model: Ecologic or Community
Time Perspective: Prospective
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





Primary Outcome Measures :
  1. 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.



Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.


  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adults (age >18 years) with any stage and any subtype of Charcot-Marie-Tooth disease, who are resident in France, Germany, Italy, Spain, the United Kingdom (UK) or the USA, so long as they have a National Health Service (NHS) or Community Health Index (CHI) number (or equivalent) and are willing to use their own smartphone / tablet.
Criteria

Inclusion Criteria:

(*) Aged 18 years and over (*) Diagnosed with any stage and any subtype of Charcot-Marie-Tooth disease (*) Resident in France, Germany, Italy, Spain, the UK, or the USA (*) Have a National Health Service (NHS) or Community Health Index (CHI) number, or equivalent (*) Willing to use their own smartphone

Exclusion Criteria:

(*) No specific exclusion criteria.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03782883


Contacts
Layout table for location contacts
Contact: Mark JW Larkin, PhD +33 645 900 348 mark.larkin@vitaccess.com
Contact: Helen V Williams, DPhil +44 7732 066832 helen.williams@vitaccess.com

Locations
Layout table for location information
United Kingdom
Vitaccess Ltd Recruiting
Oxford, United Kingdom, OX1 1BY
Contact: Mark JW Larkin, PhD    +441865818983    mark.larkin@vitaccess.com   
Contact: Helen V Williams, DPhil    +441865818983    helen.williams@vitaccess.com   
Sponsors and Collaborators
Vitaccess Ltd
Pharnext SA
ACMT Network for Charcot-Marie-Tooth disease
Asociación Madrileña de Enfermedades Neuromusculares (ASEM Madrid)
Charcot-Marie-Tooth Association
Charcot-Marie-Tooth UK
CMT France
Hereditary Neuropathy Foundation
Investigators
Layout table for investigator information
Principal Investigator: Mark JW Larkin, PhD Vitaccess Ltd

Additional Information:

Layout table for additonal information
Responsible Party: Vitaccess Ltd
ClinicalTrials.gov Identifier: NCT03782883     History of Changes
Other Study ID Numbers: 5101-01-2018
First Posted: December 20, 2018    Key Record Dates
Last Update Posted: December 20, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Aggregated data research dashboards will be available via industry subscriptions or free academic access.

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Tooth Diseases
Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Stomatognathic Diseases
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn