Physical Activity Level at Home in CMT1A Patients: Wearable Sensor Assessment

NCT07591779 | Not Yet Recruiting

• 1 other identifier: 87RI26_0017_CMT1A-HOME
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Peripheral Neuropathy Hereditary; Neuromuscular Diseases; Charcot-Marie-Tooth Disease, Type IA; Motor Activity; Walking, Difficulty

Keywords

CMT1A; wearable sensor; actigraphy; physical activity; CMT-FOM; functional assessment; peripheral neuropathy

Outcome Measures

Primary Outcomes (8)

• Pearson/Spearman r: daily step count (ActiGraph) vs. CMT-FOM total score

  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

  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

  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

  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

  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

  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

  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

  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)

Secondary Outcomes (10)

• Intraclass Correlation Coefficient (ICC) of daily step count across 7 days

  Day 7 (home monitoring)

• Coefficient of Variation (CV, %) of daily activity counts across 7 days

  Day 7 (home monitoring)

• Minimal Detectable Change (MDC) of daily step count

  Day 7 (home monitoring)

• Pearson r: daily step count vs. CMT Neuropathy Score (CMT-NS)

  Day 1

• Pearson r: daily step count vs. 6-Minute Walk Test distance (meters)

  Day 1

Study Arms (1)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• Age ≥ 18 years
• Genetically confirmed diagnosis of CMT1A (PMP22 duplication on chromosomal analysis)
• 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
• Ability to walk independently (with or without walking aids)
• Informed consent obtained
• Affiliated to French social security system

You may not qualify if:

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

Sponsors & Collaborators

• University Hospital, Limoges: lead
• Limoges University: collaborator

Study Sites (1)

Chu de Limoges

Limoges, 87042, France

Location

Related Publications (7)

• Mandarakas MR, Eichinger KJ, Bray P, Cornett KMD, Shy ME, Reilly MM, Ramdharry GM, Scherer SS, Pareyson D, Estilow T, McKay MJ; for ACT-CMT Study Group; Herrmann DN, Burns J. Multicenter Validation of the Charcot-Marie-Tooth Functional Outcome Measure. Neurology. 2024 Feb 13;102(3):e207963. doi: 10.1212/WNL.0000000000207963. Epub 2024 Jan 18.

  PMID: 38237108 BACKGROUND

• Shy ME, et al. CMT Neuropathy Score: a reliable scale of disability for Charcot-Marie-Tooth disease. Neurology. 2005;64(10):1738-1744.

  BACKGROUND

• Vinci P, Perelli SL. Footdrop, foot rotation, and plantarflexor failure in Charcot-Marie-Tooth disease. Arch Phys Med Rehabil. 2002 Apr;83(4):513-6. doi: 10.1053/apmr.2002.31174.

  PMID: 11932853 BACKGROUND

• Tofthagen C, et al. Wearable technology for monitoring physical activity in patients with Charcot-Marie-Tooth disease. J Neurol Sci. 2019;396:102-107.

  BACKGROUND

• Tudor-Locke C, Bassett DR Jr. How many steps/day are enough? Preliminary pedometer indices for public health. Sports Med. 2004;34(1):1-8. doi: 10.2165/00007256-200434010-00001.

  PMID: 14715035 BACKGROUND

• Koo TK, Li MY. A Guideline of Selecting and Reporting Intraclass Correlation Coefficients for Reliability Research. J Chiropr Med. 2016 Jun;15(2):155-63. doi: 10.1016/j.jcm.2016.02.012. Epub 2016 Mar 31.

  PMID: 27330520 BACKGROUND

• ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002 Jul 1;166(1):111-7. doi: 10.1164/ajrccm.166.1.at1102. No abstract available.

  PMID: 12091180 BACKGROUND

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease; Motor Activity; Mobility Limitation; Neuromuscular Diseases; Peripheral Nervous System Diseases

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Behavior; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Maxence COMPAGNAT, Pr

CONTACT

05 55 05 65 18; maxence.compagnat@chu-limoges.fr

Simon FRACHET, Dr

CONTACT

simon.frachet@chu-limoges.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 26, 2026
• First Posted: May 18, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): April 15, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: May 22, 2026

Record last verified: 2026-03

Locations

FR (1)