Hereditary and Inflammatory Peripheral NeuropathiesNCT07340749
Quality of Life in Hereditary and Inflammatory Peripheral Neuropathies
An observational study of Hereditary and Inflammatory Peripheral Neuropathies, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
Trial summary
Neuromuscular diseases affect the nerves and muscles and can cause weakness, pain, fatigue, and difficulties in daily life. These problems may reduce independence, social participation, and overall quality of life.This observational study aims to evaluate the impact of peripheral neuropathies on quality of life. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary and Inflammatory Peripheral Neuropathies
- What is being tested?
- WHODAS 2.0 questionnaire
- How many people
- 60 participants
- Where
- 1 siteFrance
- Study dates
- 1 Feb 2026to 1 Jul 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study will include two groups of patients. Group A will consist of 30 patients with hereditary peripheral neuropathy (e.g., Charcot-Marie-Tooth disease and other HPNs). Group B will include 30 patients with inflammatory peripheral neuropathy (e.g., acute or chronic inflammatory demyelinating polyneuropathies, autoimmune neuropathies).
Who may be able to join
- Clinical and paraclinical diagnosis compatible with hereditary or inflammatory peripheral neuropathy, confirmed by the investigating physicians (genetic results for HPN if available; clinical/electrophysiological criteria for IPN).
- Written informed consent.
- Ability to complete the questionnaires (or with the assistance of a caregiver if necessary, specifying the mode of assistance).
Who cannot join
- Legal incapacity (under guardianship or conservatorship).
- Moderate to severe cognitive impairment (e.g., MMSE < 27) preventing valid comprehension and completion of the questionnaires.
- Active cancer undergoing chemotherapy or radiotherapy (treatment likely to significantly alter quality of life in a non-specific way).
- Other acute serious medical conditions that could interfere with assessment, at the investigator's discretion.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site
CHU de Nice
Nice, Alpes Maritimes, 06000
Sabrina Sacconi, 0492038953 ext. + 33, sacconi.s@chu-nice.fr
Slioui :Abderhmane, 0492038953 ext. +33, :slioui.a@chu-nice.fr
About this study
Official title: Cross-sectional Observational Study on the Impact of Neuromuscular Diseases on Quality of Life: Focus on Peripheral Neuropathies (Hereditary vs Inflammatory)
Neuromuscular diseases affect the nerves and muscles and can cause weakness, pain, fatigue, and difficulties in daily life. These problems may reduce independence, social participation, and overall quality of life.This observational study aims to evaluate the impact of peripheral neuropathies on quality of life. It focuses on two types of peripheral neuropathies: hereditary neuropathies, caused by genetic factors, and inflammatory neuropathies, caused by immune-related nerve damage. Adult patients with these conditions will be assessed at a single time point, without any change to their usual medical care. Participants will complete questionnaires about daily functioning, well-being, and quality of life. Some questions will also explore the use of assistive devices and supportive tools in everyday life.The study hypothesizes that peripheral neuropathies significantly affect quality of life and that this impact differs between hereditary and inflammatory forms. The results aim to improve understanding of patients' needs and support better, more personalized care."
- Being tested
- WHODAS 2.0 questionnaire
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WHODAS 2.0 questionnaireOther
Also called WHOQOL BREF questionnaire, MPT Questionnaire (SOTU e ATD PA)
WHODAS 2.0: Assesses daily functioning and disability across six domains: understanding, mobility, self-care, interpersonal relationships, domestic activities, and social participation.
WHOQOL-BREF: Measures overall quality of life in four domains: physical, psychological, social relationships, and environment.
MPT Questionnaire (SOTU and ATD PA): Evaluates participants' attitudes, perceptions, and professional practices in a specific organizational or educational context.
| Study group | Role | What they receive |
|---|---|---|
| Patients with Hereditary peripheral neuropathies | "WHODAS 2.0: Assesses daily functioning and disability across six domains: understanding, mobility, self-care, interpersonal relationships, domestic activities, and social participation.
WHOQOL-BREF: Measures overall quality of life in four domains: physical, psychological, social relationships, and environment.
MPT Questionnaire (SOTU and ATD PA): Evaluates participants' attitudes, perceptions, and professional practices in a specific organizational or educational context." Receives: Other: WHODAS 2.0 questionnaire | |
| Patients with inflammatory peripheral neuropathies | "WHODAS 2.0: Assesses daily functioning and disability across six domains: understanding, mobility, self-care, interpersonal relationships, domestic activities, and social participation.
WHOQOL-BREF: Measures overall quality of life in four domains: physical, psychological, social relationships, and environment.
MPT Questionnaire (SOTU and ATD PA): Evaluates participants' attitudes, perceptions, and professional practices in a specific organizational or educational context." Receives: Other: WHODAS 2.0 questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life (WHOQOL-BREF score)
Quality of life (WHOQOL-BREF score) at baseline (M0) in patients with hereditary peripheral neuropathy (HPN) and inflammatory peripheral neuropathy (IPN)
Measured: At the inclusion
Secondary outcomes (7)
Comparison of perceived quality of life
Comparison of perceived quality of life between HPN and IPN groups using WHOQOL-BREF scores
Measured: At the inclusion
Association between functioning and quality of life
correlation between WHODAS 2.0 scores and WHOQOL-BREF scores.
Measured: At the inclusion
Factors associated with impaired functioning and quality of life
identification of clinical or demographic variables linked to lower WHODAS 2.0 or WHOQOL-BREF scores.
Measured: At the inclusion
Role of assistive technologies (MPT)
Evaluation of the impact or use of assistive technologies on functioning and quality of life.
Measured: At the inclusion
Profile of limitations
Assessment using WHODAS 2.0 scores to describe functional limitations
Measured: At the inclusion
Activity restrictions
Assessment using WHODAS 2.0 scores
Measured: At the inclusion
Environmental factors
Assessment using WHODAS 2.0 scores
Measured: At the inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2026
- Study start1 Feb 2026 (estimated)
- Primary completion1 Feb 2026 (estimated)
- Study completion1 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary and Inflammatory Peripheral NeuropathiesStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe sponsor's other registered studies.
- 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
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07340749
- Sponsor's protocol ID
- 26Neuro01
- Status last verified
- Jan 2026
- First submitted
- 5 Jan 2026
- Last updated
- 14 Jan 2026
- 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 14 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cross-sectional Observational Study on the Impact of Neuromuscular Diseases on Quality of Life: Focus on Peripheral Neuropathies (Hereditary vs Inflammatory)
- ClinicalTrials.gov ID
- NCT07340749
- Acronym / sponsor ID
- 26Neuro01
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- 1 Feb 2026 (estimated)
- Primary completion
- 1 Feb 2026 (estimated)
- Study completion
- 1 Jul 2026 (estimated)
- First posted
- 14 Jan 2026 (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.
Neuromuscular diseases affect the nerves and muscles and can cause weakness, pain, fatigue, and difficulties in daily life. These problems may reduce independence, social participation, and overall quality of life.This observational study aims to evaluate the impact of peripheral neuropathies on quality of life. It focuses on two types of peripheral neuropathies: hereditary neuropathies, caused by genetic factors, and inflammatory neuropathies, caused by immune-related nerve damage. Adult patients with these conditions will be assessed at a single time point, without any change to their usual medical care. Participants will complete questionnaires about daily functioning, well-being, and quality of life. Some questions will also explore the use of assistive devices and supportive tools in everyday life.The study hypothesizes that peripheral neuropathies significantly affect quality of life and that this impact differs between hereditary and inflammatory forms. The results aim to improve understanding of patients' needs and support better, more personalized care."
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with Hereditary peripheral neuropathies | – | Other: WHODAS 2.0 questionnaire"WHODAS 2.0: Assesses daily functioning and disability across six domains: understanding, mobility, self-care, interpersonal relationships, domestic activities, and social participation. WHOQOL-BREF: Measures overall quality of life in four domains: physical, psychological, social relationships, and environment. MPT Questionnaire (SOTU and ATD PA): Evaluates participants' attitudes, perceptions, and professional practices in a specific organizational or educational context." |
| Patients with inflammatory peripheral neuropathies | – | Other: WHODAS 2.0 questionnaire"WHODAS 2.0: Assesses daily functioning and disability across six domains: understanding, mobility, self-care, interpersonal relationships, domestic activities, and social participation. WHOQOL-BREF: Measures overall quality of life in four domains: physical, psychological, social relationships, and environment. MPT Questionnaire (SOTU and ATD PA): Evaluates participants' attitudes, perceptions, and professional practices in a specific organizational or educational context." |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study will include two groups of patients. Group A will consist of 30 patients with hereditary peripheral neuropathy (e.g., Charcot-Marie-Tooth disease and other HPNs). Group B will include 30 patients with inflammatory peripheral neuropathy (e.g., acute or chronic inflammatory demyelinating polyneuropathies, autoimmune neuropathies).
Key inclusion criteria (3 listed)
- Clinical and paraclinical diagnosis compatible with hereditary or inflammatory peripheral neuropathy, confirmed by the investigating physicians (genetic results for HPN if available; clinical/electrophysiological criteria for IPN).
- Written informed consent.
- Ability to complete the questionnaires (or with the assistance of a caregiver if necessary, specifying the mode of assistance).
Key exclusion criteria (4 listed)
- Legal incapacity (under guardianship or conservatorship).
- Moderate to severe cognitive impairment (e.g., MMSE < 27) preventing valid comprehension and completion of the questionnaires.
- Active cancer undergoing chemotherapy or radiotherapy (treatment likely to significantly alter quality of life in a non-specific way).
- Other acute serious medical conditions that could interfere with assessment, at the investigator's discretion.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WHODAS 2.0 questionnaireAlso: WHOQOL BREF questionnaire, MPT Questionnaire (SOTU e ATD PA)WHODAS 2.0: Assesses daily functioning and disability across six domains: understanding, mobility, self-care, interpersonal relationships, domestic activities, and social participation. WHOQOL-BREF: Measures overall quality of life in four domains: physical, psychological, social relationships, and environment. MPT Questionnaire (SOTU and ATD PA): Evaluates participants' attitudes, perceptions, and professional practices in a specific organizational or educational context. | Other | Patients with Hereditary peripheral neuropathies, Patients with inflammatory peripheral neuropathies |
Primary outcome
Quality of life (WHOQOL-BREF score)
Time frame: At the inclusion
Quality of life (WHOQOL-BREF score) at baseline (M0) in patients with hereditary peripheral neuropathy (HPN) and inflammatory peripheral neuropathy (IPN)
7 secondary outcomes
Comparison of perceived quality of life
Time frame: At the inclusion
Association between functioning and quality of life
Time frame: At the inclusion
Factors associated with impaired functioning and quality of life
Time frame: At the inclusion
Role of assistive technologies (MPT)
Time frame: At the inclusion
Profile of limitations
Time frame: At the inclusion
Activity restrictions
Time frame: At the inclusion
Environmental factors
Time frame: At the inclusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2026.
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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.
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 Hereditary and Inflammatory Peripheral Neuropathies, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
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 3 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: opening soon, as of the sponsor's last update on 14 Jan 2026. 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 France. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sabrina Sacconi (0492038953 ext. + 33, sacconi.s@chu-nice.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quality of life (WHOQOL-BREF score) (time frame: At the inclusion). 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 14 Jan 2026.