Condition or disease |
---|
CMT |
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 |
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 |
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 |
Inclusion Criteria:
Exclusion Criteria:
Germany | |
University Medical Centre | |
Goettigen, Germany, 37075 |
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 |
|
||||
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 |
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:
Exclusion Criteria:
|
||||
Sex/Gender |
|
||||
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 |
|
||||
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 |