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

CMT Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03966287
• Other study ID #: 02202
• Status: Completed
• Start date: June 3, 2019
• Est. completion date: March 31, 2020

Study information

• Verified date: November 2020
• Source: University Medical Center Goettingen
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 200
• Est. completion date: March 31, 2020
• Est. primary completion date: January 31, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Charcot-Marie-Tooth Disease
• CMT
• Hereditary Sensory and Motor Neuropathy
• Nerve Compression Syndromes

Locations

Country	Name	City	State
Germany	University Medical Centre	Goettigen

Sponsors (1)

Lead Sponsor	Collaborator
University Medical Center Goettingen

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	SF-36 Questionnaire	Questionnaire to evaluate health status	1 year
Primary	NPSI-G Questionnaire	Questionnaire to discriminate neuropathic and non-neuropathic pain	1 day
Primary	PAIN DETECT Questionnaire	Questionnaire for the diagnosis of neuropathic pain (intensity, pattern, quality)	4 weeks