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出境医 / 临床实验 / Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)

Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)

Study Description
Brief Summary:
The study aims to qualitatively and quantitively evaluate pain in patients suffering from CMT with 3 standardized questionnaires (SF-36, NPSI-G, PAIN DETECT) as well as its impact on the quality of life.

Condition or disease
CMT

Detailed Description:

Hereditary neuropathies are a group of clinically and genetically heterogeneous diseases of the peripheral nervous system. Charcot-Marie-Tooth disease (CMT), also known as hereditary motor and sensory neuropathy (HMSN), is the most common form of hereditary neuropathy with a prevalence of approximately 1 in 2,500 and with approximately 30,000 affected persons in Germany. There is no therapy known so far. In addition to the characteristic symptoms of a distal to proximal progressive symmetric muscular atrophy, especially of the lower extremities, there is a weakening of the muscles of the lower legs and feet, with emphasis on the "small" foot muscles and the peroneal muscle group, which clinically manifests as a so-called "stepper gait". It is also known that many CMT patients additionally suffer from pain, but more detailed studies on pain quality and quantity and their impact on patients' quality of life have not yet been systematically performed. These parameters will be investigated in this study, supported by the company Grünenthal, in the form of a questionnaire survey with validated questionnaires on pain and quality of life for at least 200 CMT patients. A representative result could lead to further investigations of pain in CMT patients and ultimately to the development of an adequate pain therapy.

Recruitment of patients will occur mainly through a call of the Germany-wide CMT registry, which is part of the national research network CMT-NET (coordinator: Prof. Michael Sereda). Interested patients are invited to contact the study team at the UMG. The patients will be informed about the study and if further interested 4 questionnaires (1 regarding the personal history as well as 3 standardized questionnaires from literature regarding quality of life and pain (SF-36, NPSI-G, PAIN DETECT) will be sent to the patients together with obligatory study documents and a pre-franked return envelope. A personal visit at the UMG is not planned, so that the patients don´t have any expenses. The data are pseudonymized at the UMG and transferred to a database and finally statistically evaluated by Gruenenthal.

Study Design
Layout table for study information
Study Type : Observational
Actual Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)
Actual Study Start Date : June 3, 2019
Actual Primary Completion Date : January 31, 2020
Actual Study Completion Date : March 31, 2020
Arms and Interventions
Outcome Measures
Primary Outcome Measures :
  1. SF-36 Questionnaire [ Time Frame: 1 year ]
    Questionnaire to evaluate health status

  2. NPSI-G Questionnaire [ Time Frame: 1 day ]
    Questionnaire to discriminate neuropathic and non-neuropathic pain

  3. PAIN DETECT Questionnaire [ Time Frame: 4 weeks ]
    Questionnaire for the diagnosis of neuropathic pain (intensity, pattern, quality)


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
German-wide, registered patients in CMT-registry
Criteria

Inclusion Criteria:

  • Clinical diagnosis of CMT
  • Genetic confirmation of CMT diagnosis
  • Age between 18 and 65 years
  • Signed consent form

Exclusion Criteria:

  • Other relevant neurological or psychiatric diseases, acute or past
  • Serious internal medical illness
  • Drug and / or alcohol dependence
  • Participation in another clinical trial within 4 weeks prior to enrollment
Contacts and Locations

Locations
Layout table for location information
Germany
University Medical Centre
Goettigen, Germany, 37075
Sponsors and Collaborators
University Medical Center Goettingen
Tracking Information
First Submitted Date May 21, 2019
First Posted Date May 29, 2019
Last Update Posted Date November 5, 2020
Actual Study Start Date June 3, 2019
Actual Primary Completion Date January 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 27, 2019)
  • SF-36 Questionnaire [ Time Frame: 1 year ]
    Questionnaire to evaluate health status
  • NPSI-G Questionnaire [ Time Frame: 1 day ]
    Questionnaire to discriminate neuropathic and non-neuropathic pain
  • PAIN DETECT Questionnaire [ Time Frame: 4 weeks ]
    Questionnaire for the diagnosis of neuropathic pain (intensity, pattern, quality)
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)
Official Title Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)
Brief Summary The study aims to qualitatively and quantitively evaluate pain in patients suffering from CMT with 3 standardized questionnaires (SF-36, NPSI-G, PAIN DETECT) as well as its impact on the quality of life.
Detailed Description

Hereditary neuropathies are a group of clinically and genetically heterogeneous diseases of the peripheral nervous system. Charcot-Marie-Tooth disease (CMT), also known as hereditary motor and sensory neuropathy (HMSN), is the most common form of hereditary neuropathy with a prevalence of approximately 1 in 2,500 and with approximately 30,000 affected persons in Germany. There is no therapy known so far. In addition to the characteristic symptoms of a distal to proximal progressive symmetric muscular atrophy, especially of the lower extremities, there is a weakening of the muscles of the lower legs and feet, with emphasis on the "small" foot muscles and the peroneal muscle group, which clinically manifests as a so-called "stepper gait". It is also known that many CMT patients additionally suffer from pain, but more detailed studies on pain quality and quantity and their impact on patients' quality of life have not yet been systematically performed. These parameters will be investigated in this study, supported by the company Grünenthal, in the form of a questionnaire survey with validated questionnaires on pain and quality of life for at least 200 CMT patients. A representative result could lead to further investigations of pain in CMT patients and ultimately to the development of an adequate pain therapy.

Recruitment of patients will occur mainly through a call of the Germany-wide CMT registry, which is part of the national research network CMT-NET (coordinator: Prof. Michael Sereda). Interested patients are invited to contact the study team at the UMG. The patients will be informed about the study and if further interested 4 questionnaires (1 regarding the personal history as well as 3 standardized questionnaires from literature regarding quality of life and pain (SF-36, NPSI-G, PAIN DETECT) will be sent to the patients together with obligatory study documents and a pre-franked return envelope. A personal visit at the UMG is not planned, so that the patients don´t have any expenses. The data are pseudonymized at the UMG and transferred to a database and finally statistically evaluated by Gruenenthal.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Retrospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population German-wide, registered patients in CMT-registry
Condition CMT
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: May 27, 2019)
200
Original Estimated Enrollment Same as current
Actual Study Completion Date March 31, 2020
Actual Primary Completion Date January 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Clinical diagnosis of CMT
  • Genetic confirmation of CMT diagnosis
  • Age between 18 and 65 years
  • Signed consent form

Exclusion Criteria:

  • Other relevant neurological or psychiatric diseases, acute or past
  • Serious internal medical illness
  • Drug and / or alcohol dependence
  • Participation in another clinical trial within 4 weeks prior to enrollment
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Germany
Removed Location Countries  
 
Administrative Information
NCT Number NCT03966287
Other Study ID Numbers 02202
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Michael W Sereda, MD, Professor of Neurology, University Medical Center Goettingen
Study Sponsor University Medical Center Goettingen
Collaborators Not Provided
Investigators Not Provided
PRS Account University Medical Center Goettingen
Verification Date November 2020