CMTNCT03966287
Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)
An observational study of CMT, run by University Medical Center Goettingen at 1 site in Germany.
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- CMT
- How many people
- 200 participants
- Where
- 1 siteGermany
- Study dates
- 3 Jun 2019to 31 Mar 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: German-wide, registered patients in CMT-registry
Who may be able to join
- Clinical diagnosis of CMT
- Genetic confirmation of CMT diagnosis
- Age between 18 and 65 years
- Signed consent form
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- CMT
What is being measured
The primary outcome is the main question the study is designed to answer.
SF-36 Questionnaire
Questionnaire to evaluate health status
Measured: 1 year
NPSI-G Questionnaire
Questionnaire to discriminate neuropathic and non-neuropathic pain
Measured: 1 day
PAIN DETECT Questionnaire
Questionnaire for the diagnosis of neuropathic pain (intensity, pattern, quality)
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2019
- Study start3 Jun 2019
- Primary completion31 Jan 2020
- Study completion31 Mar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CMTStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Medical Center Goettingen
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Michael W Sereda, MD, Professor of Neurology, University Medical Center Goettingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03966287
- Sponsor's protocol ID
- 02202
- Status last verified
- Nov 2020
- First submitted
- 21 May 2019
- Last updated
- 5 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)
- ClinicalTrials.gov ID
- NCT03966287
- Acronym / sponsor ID
- 02202
- Conditions
- CMT
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- University Medical Center GoettingenUniversity, hospital or other
- Study start
- 3 Jun 2019 (actual)
- Primary completion
- 31 Jan 2020 (actual)
- Study completion
- 31 Mar 2020 (actual)
- First posted
- 29 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- German-wide, registered patients in CMT-registry
Key inclusion criteria (4 listed)
- Clinical diagnosis of CMT
- Genetic confirmation of CMT diagnosis
- Age between 18 and 65 years
- Signed consent form
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of CMT, run by University Medical Center Goettingen at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
SF-36 Questionnaire (time frame: 1 year). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2020.