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Physical Activity Level at Home in CMT1A Patients: Wearable Sensor Assessment (CMT1A-HOME)

20. Mai 2026 aktualisiert von: University Hospital, Limoges

Study of the Relationship Between Clinical and Functional Characteristics of Patients With CMT1A Disease and Their Level of Physical Activity at Home Measured Using Portable Electronic Sensors

Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common hereditary peripheral neuropathy, affecting approximately 26,000 patients in France. It presents as chronic and progressive sensorimotor deficits predominantly affecting the distal lower limbs, with onset typically in childhood. There is currently no specific pharmacological treatment; management remains symptomatic.

This research will:

In the long run, validated wearable sensors could improve patient follow-up, personalize rehabilitation, and support the design of clinical trials for CMT1A - including trials of the novel "Nano-Cur" treatment currently under development.

Studienübersicht

Status

Noch keine Rekrutierung

Bedingungen

Detaillierte Beschreibung

Peripheral neuropathies are frequent conditions affecting peripheral nerves with highly varied etiologies. Among genetic causes, Charcot-Marie-Tooth (CMT) disease is the leading hereditary neuropathy, with approximately 26,000 patients in France. The most frequent form, CMT1A, is caused by a duplication of the PMP22 gene, leading to chronic progressive sensorimotor deficits predominantly affecting the distal lower limbs, with onset in childhood and significant impact on quality of life.

No specific pharmacological treatment exists for CMT1A. The CMT-FOM scale (Mandarakas et al., 2024, Neurology, 102: e207963) constitutes the reference for functional evaluation, but requires lengthy evaluation and a specialized technical platform, making it unsuitable for routine outpatient consultations. Furthermore, punctual evaluations may be biased by external factors (fatigue, mood, motivation) without faithfully reflecting the patient's daily neurological status.

Non-invasive wearable physical activity sensors offer a promising alternative, enabling repeated and ecologically valid measurements directly at home and over prolonged periods. This study aims to assess whether such ambulatory sensors can evaluate functional impairments in CMT1A patients equivalently to a comprehensive CMT-FOM evaluation.

The study will be conducted among CMT1A patients followed at the National Reference Centre for Rare Peripheral Neuropathies (Service de Neurologie, CHU de Limoges), in partnership with the Quantified Movement Analysis Laboratory (Laboratoire d'AQM), Service de Médecine Physique et de Réadaptation (CHU de Limoges). It represents a collaborative effort between research units NeurIT (UR20218) and HAVAE (UR20217).

This project also forms part of the broader development of the "Nano-Cur" therapeutic compound (NeurIT/LABCiS collaboration), having led to a national and international patent filing and the creation of the start-up Curlim (AFM-Téléthon/AVRUL support).

Studientyp

Beobachtungs

Einschreibung (Geschätzt)

60

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienkontakt

Studieren Sie die Kontaktsicherung

Studienorte

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Probenahmeverfahren

Nicht-Wahrscheinlichkeitsprobe

Studienpopulation

Adult patients diagnosed with genetically confirmed CMT1A (PMP22 duplication) who are followed at the National Reference Centre for Rare Peripheral Neuropathies (Service de Neurologie, CHU de Limoges) and/or who have undergone gait analysis at the Quantified Movement Analysis Laboratory (Laboratoire d'AQM), Service de Médecine Physique et de Réadaptation, CHU de Limoges.

Beschreibung

Inclusion Criteria:

  1. Age ≥ 18 years
  2. Genetically confirmed diagnosis of CMT1A (PMP22 duplication on chromosomal analysis)
  3. Followed at the National Reference Centre for Rare Peripheral Neuropathies (Service de Neurologie, CHU de Limoges) and/or having undergone gait analysis at the Quantified Movement Analysis Laboratory (Laboratoire d'AQM), Service de Médecine Physique et de Réadaptation, CHU de Limoges
  4. Ability to walk independently (with or without walking aids)
  5. Informed consent obtained
  6. Affiliated to French social security system

Exclusion Criteria:

  1. Other associated neurological condition that could independently affect walking or motor activity
  2. Inability to wear the sensor device (skin allergy, sensory intolerance)
  3. Inability to comply with study procedures (cognitive impairment, no fixed domicile)
  4. Participation in another interventional study during the same period
  5. Pregnant or breastfeeding women
  6. Patients under legal protection (guardianship or curatorship)

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

Kohorten und Interventionen

Gruppe / Kohorte
CMT1A-HOME
Adult patients diagnosed with CMT1A followed at the National Reference Centre for Rare Peripheral Neuropathies (CHU de Limoges) and/or the Laboratoire d'AQM (Service de MPR, CHU de Limoges). Participants will undergo: (1) comprehensive in-hospital functional evaluation using the CMT-FOM scale and standard clinical assessments, and (2) home-based physical activity monitoring with ActiGraph LEAP wearable sensors over 7 days.

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Pearson/Spearman r: daily step count (ActiGraph) vs. CMT-FOM total score
Zeitfenster: Day 1 (CMT-FOM)
Correlation coefficient (r : -1 to +1) between mean daily step count (steps/day) over 7 days and CMT-FOM total score obtained at Day 1 hospital visit.
Day 1 (CMT-FOM)
Pearson/Spearman r: daily step count (ActiGraph) vs. CMT-FOM total score
Zeitfenster: Day 7 (home monitoring)
Correlation coefficient (r : -1 to +1) between mean daily step count (steps/day) over 7 days and CMT-FOM total score obtained at Day 1 hospital visit.
Day 7 (home monitoring)
Pearson/Spearman r: daily activity counts (ActiGraph) vs. CMT-FOM total score
Zeitfenster: Day 1
Correlation coefficient (r : -1 to +1) between mean daily activity counts (raw accelerometry counts/day) over 7 days and CMT-FOM total score.
Day 1
Pearson/Spearman r: daily activity counts (ActiGraph) vs. CMT-FOM total score
Zeitfenster: Day 7 (home monitoring)
Correlation coefficient (r : -1 to +1) between mean daily activity counts (raw accelerometry counts/day) over 7 days and CMT-FOM total score.
Day 7 (home monitoring)
Pearson/Spearman r: daily sedentary time (ActiGraph) vs. CMT-FOM total score
Zeitfenster: Day 1
Correlation coefficient (r : -1 to +1) between mean daily sedentary time (minutes/day) over 7 days and CMT-FOM total score.
Day 1
Pearson/Spearman r: daily sedentary time (ActiGraph) vs. CMT-FOM total score
Zeitfenster: Day 7 (home monitoring)
Correlation coefficient (r : -1 to +1) between mean daily sedentary time (minutes/day) over 7 days and CMT-FOM total score.
Day 7 (home monitoring)
Pearson/Spearman r: composite sensor score vs. CMT-FOM sub-scores
Zeitfenster: Day 1
Correlation coefficient (r : -1 to +1) between ActiGraph-derived metrics and CMT-FOM sub-domain scores (upper limb, lower limb, balance, endurance). Reported separately per sub-score.
Day 1
Pearson/Spearman r: composite sensor score vs. CMT-FOM sub-scores
Zeitfenster: Day 7 (home monitoring)
Correlation coefficients (r : -1 to +1) between ActiGraph-derived metrics and CMT-FOM sub-domain scores (upper limb, lower limb, balance, endurance). Reported separately per sub-score.
Day 7 (home monitoring)

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Intraclass Correlation Coefficient (ICC) of daily step count across 7 days
Zeitfenster: Day 7 (home monitoring)
Test-retest reliability (ICC (0 to 1), two-way mixed, absolute agreement) of daily step count measured by ActiGraph LEAP across 7 home monitoring days.
Day 7 (home monitoring)
Coefficient of Variation (CV, %) of daily activity counts across 7 days
Zeitfenster: Day 7 (home monitoring)
Day-to-day variability expressed as CV (%) for raw accelerometry activity counts across 7 days.
Day 7 (home monitoring)
Minimal Detectable Change (MDC) of daily step count
Zeitfenster: Day 7 (home monitoring)
MDC95 of daily step count (steps/day), calculated from SEM and ICC, representing the smallest change exceeding measurement error.
Day 7 (home monitoring)
Pearson r: daily step count vs. CMT Neuropathy Score (CMT-NS)
Zeitfenster: Day 1
Correlation (r : -1 to +1) between mean daily steps/day and CMT-NS total score at Day 1.
Day 1
Pearson r: daily step count vs. 6-Minute Walk Test distance (meters)
Zeitfenster: Day 1
Correlation (r : -1 to +1) between mean daily steps/day and 6MWT distance (meters) measured at Day 1.
Day 1
Pearson r: daily step count vs. 10-Meter Walk Test speed (m/s)
Zeitfenster: Day 1
Correlation (r : -1 to +1) between mean daily steps/day and 10MWT comfortable-pace speed (m/s) at Day 1.
Day 1
Mean daily step count stratified by CMT-FOM severity quartile
Zeitfenster: Day 1
Mean (±SD) daily step count compared across CMT-FOM severity quartiles (Q1-Q4) to characterise activity profiles by disease severity.
Day 1
Mean daily step count stratified by CMT-FOM severity quartile
Zeitfenster: Day 7 (home monitoring)
Mean (±SD) daily step count compared across CMT-FOM severity quartiles (Q1-Q4) to characterise activity profiles by disease severity.
Day 7 (home monitoring)
Mean daily sedentary time stratified by CMT-FOM severity quartile
Zeitfenster: Day 1
Mean (±SD) daily sedentary time (min/day) compared across CMT-FOM severity quartiles.
Day 1
Mean daily sedentary time stratified by CMT-FOM severity quartile
Zeitfenster: Day 7 (home monitoring)
Mean (±SD) daily sedentary time (min/day) compared across CMT-FOM severity quartiles.
Day 7 (home monitoring)

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

University Hospital, Limoges

Mitarbeiter

Limoges University

Publikationen und hilfreiche Links

Die Bereitstellung dieser Publikationen erfolgt freiwillig durch die für die Eingabe von Informationen über die Studie verantwortliche Person. Diese können sich auf alles beziehen, was mit dem Studium zu tun hat.

Allgemeine Veröffentlichungen

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Geschätzt)

1. Juni 2026

Primärer Abschluss (Geschätzt)

15. April 2027

Studienabschluss (Geschätzt)

30. Juni 2027

Studienanmeldedaten

Zuerst eingereicht

26. März 2026

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

15. Mai 2026

Zuerst gepostet (Tatsächlich)

18. Mai 2026

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

22. Mai 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

20. Mai 2026

Zuletzt verifiziert

1. März 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Schlüsselwörter

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • 87RI26_0017_CMT1A-HOME

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Nein

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

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