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

• ClinicalTrials.gov Identifier: NCT03966287
• Recruitment Status: Completed
• First Posted: May 29, 2019
• Last Update Posted: November 5, 2020
• Sponsor: University Medical Center Goettingen
• Information provided by (Responsible Party): Michael W Sereda, MD, Professor of Neurology, University Medical Center Goettingen

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

• 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

Groups and Cohorts

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

• 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

Germany

University Medical Centre

Goettigen, Germany, 37075

Sponsors and Collaborators

University Medical Center Goettingen

More Information

• Responsible Party: Michael W Sereda, MD, Professor of Neurology, Prof. Dr. Michael W. Sereda, University Medical Center Goettingen
• ClinicalTrials.gov Identifier: NCT03966287
• Other Study ID Numbers: 02202
• First Posted: May 29, 2019
• Last Update Posted: November 5, 2020
• Last Verified: November 2020

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Charcot-Marie-Tooth Disease; Nerve Compression Syndromes; Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Genetic Diseases, Inborn