Fiche traitement
test of walk
Centre Hospitalier Universitaire de Nice • Myopathies
Suivi actif
NA
Résumé
test of walk est développé par Centre Hospitalier Universitaire de Nice. Le traitement est actuellement en NA. Il est associé à Myopathies. Son objectif thérapeutique principal est Immunomodulation + Thérapie cellulaire. Le statut actuel est : suivi actif.
À vérifier
Immunomodulation + Thérapie cellulaire
NA
Centre Hospitalier Universitaire de Nice
France
The facial-glenohumeral muscular dystrophy type 1 (DMFSH1) is characterized by a selective and asymmetrical involvement of the facial muscles, the shoulder girdle and the anterolateral lodge legs. Genetically, the disease is transmitted in an autosomal dominant manner and is caused by a pathogen contraction of repeat units (UR) say D4Z4 localized to the telomeric portion of chromosome 4qA. The loss of UR causes hypomethylation of DNA and chromatin relaxation of the region that lead to inappropriate expression of DUX4 retrogene highly toxic. The inappropriate expression induces a T cell reaction inflammatory response that participate and increase muscle damage. In favor of this hypothesis, several muscle MRI studies have shown that atrophy and fibro-adipose degeneration (hyper signal in T1) were preceded by the appearance of muscle inflammation (hyper signal T2STIR) confirmed on histologically and dysregulation of genes involved in adaptive and innate immunity. scientific hypothesis and potential benefits: the investigateur hypothesize that in patients of DMFSH1, the immune system cells may participate in the pathophysiology of the disease through changes in serum secretion of one or more cytokines and / or a modification of the response of inflammatory cells in some cell damage stimuli. Design: this is a single-center pilot study, interventional. In this study, the investigator will assay the serum cytokines and changes in peripheral blood cells of the expression of cytokines in response to some stimuli in 20 patients with Type 1 DMFSH genetically confirmed at an intermediate stage of clinical disease (kept walking, but at least one muscle of lower limbs reached) and compare with controls from the CYTOKINAGE study. The investigator will also carry patients clinical testing (MMT sum score) and functional (6minute test march MFM) and a MRI not injected whole body (T1 sequences + and T2STIR) to study the relationship between these parameters and secretion cytokines or serum in response to certain stimuli Main objective: to compare serum levels of IL-6 in patients with DMFSH and controls.
2026-03-20
Début phase actuelle
Non disponible dans les sources synchronisées
Timeline clinique
NA
Début phase actuelle
Non disponible dans les sources synchronisées
Préclinique
Phase 1
Phase 2
Phase 3
Approuvé
Objectif scientifique de l’étude
classification automatique vérifiable
Immunomodulation
Thérapie cellulaire
Confiance automatique : 95%
Ce pourcentage n’est pas une probabilité de succès clinique.
Il mesure uniquement la force de la classification automatique de l’objectif scientifique. Le calcul part de 45 points, ajoute 7 points par unité de poids du mot-clé le plus fortement détecté, puis est plafonné à 95 %. Exemple : un score maximal de 2 donne 45 + (2 × 7) = 59 %.
Indices détectés : immune → Immunomodulation, inflamm → Immunomodulation, t cell → Immunomodulation, stem cell → Thérapie cellulaire
Ce score est heuristique et doit être vérifié dans le protocole ou une publication.
Description affichée depuis l’essai compatible NCT04694456, afin d’éviter le mélange entre plusieurs indications d’une même molécule.
Aucune incohérence majeure détectée entre la maladie, l’objectif et la description.
Transparence et qualité de la fiche
données vérifiablesClassificationCohérente automatiquement
Confiance de la fiche100 %
Source principaleSource officielle ou publication indexée
Dernière vérification2026-03-20
Anomalies détectées0
Validation humaineValidation humaine non effectuée
Donnée issue d’une source
Donnée normalisée automatiquement
Interprétation algorithmique
Classification scientifique multi-axes
ne pas confondre statut et résultatÉtat de l’étudeTerminée
Disponibilité des résultatsRésultats à vérifier
InterprétationImpossible à déterminer
Niveau de preuveNon évaluable
Un essai terminé n’est pas nécessairement positif. Un essai arrêté n’est classé comme échec scientifique que lorsqu’un résultat ou une raison explicite le confirme.
Indicateurs factuels
sans score subjectifPhase enregistréeNA
Études associées1
Publications associées19
Statut consolidéSuivi actif
Aucune chance de succès, note d’innovation, potentiel thérapeutique ou probabilité de commercialisation n’est calculé tant qu’un modèle validé et des données suffisantes ne sont pas disponibles.
Historique factuel de la molécule
24 événement(s) daté(s)Cette liste reprend uniquement les dates trouvées dans les registres et publications liés. Elle ne constitue pas une prévision.
| Date | Événement | Source |
|---|---|---|
| 2018-01-30 | Début de l’étudeNCT04694456 | Registre d’essai clinique |
| 2021-05-18 | Fin de l’étude prévue / enregistréeNCT04694456 | Registre d’essai clinique |
| 2026-03-20 | Dernière mise à jour des donnéestest of walk | Biomedical Watch |
| 2026-07-08 | Création de l’enregistrementNCT04694456 | Registre d’essai clinique |
| 2026-07-08 | PublicationFeasibility and behavioral impact of a wearable-supported digital health intervention on attitudes and behaviors toward sleep and physical activity: A proof-of-concept study in Kobe City. | Digital health |
| 2026-07-08 | PublicationFive-Year Outcomes of Paclitaxel-Coated Balloons in Patients with Diabetes from the BIOLUX P-III Single-Arm Study. | Vascular health and risk management |
| 2026-07-08 | PublicationYiqi Fumai Lyophilized Injection for Improving Exercise Tolerance in Chronic Heart Failure: Protocol for a Prospective Cohort Study. | JMIR research protocols |
| 2026-07-08 | PublicationThe reliability and validity of the 30-second chair sit-to-stand test and the five-repetition sit-to-stand test in individuals following lumbar spinal surgery. | European spine journal : official publication of the European Spine Society, the European Spinal Deformity Society, and the European Section of the Cervical Spine Research Society |
| 2026-07-10 | PublicationThe Impact of Exercise Tolerance Assessed by 6-Min Walking Distance on Clinical Outcomes in Patients With Interstitial Lung Disease Associated With Connective Tissue Diseases. | International journal of rheumatic diseases |
| 2026-07-10 | PublicationPulmonary function and physical fitness in obese children and adolescents: a comparison of impulse oscillometry, spirometry, and 6-min walk test. | European journal of pediatrics |
| 2026-07-12 | PublicationMultimodal Integration of Gait, Balance, and Infrared Thermography Enhances Machine-Learning Classification of Knee Osteoarthritis: A Cross-Sectional Study. | Archives of physical medicine and rehabilitation |
| 2026-07-12 | PublicationA comparison of conservative versus surgical treatment in patients with foot drop due to peroneal nerve entrapment: results of a prematurely terminated randomized controlled trial. | Brain & spine |
| 2026-07-13 | Dernière mise à jour du registreNCT04694456 | Registre d’essai clinique |
| 2026-07-14 | Publication[Real-world testing of digital health: the INSPIRE Living Lab : An interdisciplinary clinical innovation platform]. | Orthopadie (Heidelberg, Germany) |
| 2026-07-14 | PublicationCentral adiposity is associated with poorer functional capacity in women with knee osteoarthritis: a cross-sectional study with exploratory adjustment for pain intensity and radiographic severity. | Rheumatology international |
| 2026-07-14 | PublicationEffects of Pulmonary Rehabilitation Nursing on Pulmonary Function and Exercise Capacity in Patients With COPD: A Meta-Analysis. | Nursing in critical care |
| 2026-07-18 | PublicationDoes combining walked distance and oxygenation obtained during the six-minute walk test improve mortality prediction in interstitial lung disease? | Heart & lung : the journal of critical care |
| 2026-07-18 | PublicationComparison of the Effects of Telerehabilitation-Based Motor and Cognitive Dual-Task Exercises in Patients With Parkinson's Disease-A Randomized Controlled Study. | Journal of aging and physical activity |
| 2026-07-22 | PublicationFeasibility of Real-Time Autonomous Functional Electrical Stimulation for Freezing of Gait. | The European journal of neuroscience |
| 2026-07-22 | PublicationA mobile learning environment: a preliminary study of a developmentally progressive modified ride-on car in infants with Down Syndrome. | Disability and rehabilitation. Assistive technology |
| 2026-08-13 | PublicationThe Effect of Whole-Body Cryotherapy on Exercise Capacity of Healthy Training Men. | Journal of clinical medicine |
| 2026-08-13 | PublicationPrecision and Error Propagation in Static MEMS-IMU Inertial Navigation: A Stochastic Time-Series Analysis. | Sensors (Basel, Switzerland) |
| 2026-08-16 | PublicationNigral biochemical and structural alterations following subacute exposure to MPTP in mice. | Toxicology reports |
| 2026-08-16 | PublicationA robotic perturbation trainer for transverse-plane gait perturbations in pediatric cerebral palsy. | MethodsX |
Début phase actuelle
Non disponible dans les sources synchronisées
Essais cliniques liés
1 essai(s)
Début phase actuelle
Non disponible dans les sources synchronisées
| NCT | Titre | Phase | Statut administratif | Pays | Sponsor |
|---|---|---|---|---|---|
| NCT04694456 | Pro-inflammatory Cytokines in Facioscapulohumeral Muscular Dystrophy (CYTOKINE-FSH) | NA | COMPLETED | France | Centre Hospitalier Universitaire de Nice |
Publications liées
19 publication(s)Publications liées
19 publication(s)| Titre | PMID | DOI | Journal | Date |
|---|---|---|---|---|
| Nigral biochemical and structural alterations following subacute exposure to MPTP in mice. Voir source | 42502326 | 10.1016/j.toxrep.2026.102314 | Toxicology reports | |
| A robotic perturbation trainer for transverse-plane gait perturbations in pediatric cerebral palsy. Voir source | 42502775 | 10.1016/j.mex.2026.104051 | MethodsX | |
| The Effect of Whole-Body Cryotherapy on Exercise Capacity of Healthy Training Men. Voir source | 42590172 | 10.3390/jcm15156070 | Journal of clinical medicine | |
| Precision and Error Propagation in Static MEMS-IMU Inertial Navigation: A Stochastic Time-Series Analysis. Voir source | 42590462 | 10.3390/s26154685 | Sensors (Basel, Switzerland) | |
| Feasibility of Real-Time Autonomous Functional Electrical Stimulation for Freezing of Gait. Voir source | 42484421 | 10.1111/ejn.70633 | The European journal of neuroscience | |
| A mobile learning environment: a preliminary study of a developmentally progressive modified ride-on car in infants with Down Syndrome. Voir source | 42485077 | 10.1080/17483107.2026.2703296 | Disability and rehabilitation. Assistive technology | |
| Does combining walked distance and oxygenation obtained during the six-minute walk test improve mortality prediction in interstitial lung disease? Voir source | 42468472 | 10.1016/j.hrtlng.2026.102885 | Heart & lung : the journal of critical care | |
| Comparison of the Effects of Telerehabilitation-Based Motor and Cognitive Dual-Task Exercises in Patients With Parkinson's Disease-A Randomized Controlled Study. Voir source | 42468927 | 10.1123/japa.2026-0190 | Journal of aging and physical activity | |
| [Real-world testing of digital health: the INSPIRE Living Lab : An interdisciplinary clinical innovation platform]. Voir source | 42446712 | 10.1007/s00132-026-04807-y | Orthopadie (Heidelberg, Germany) | |
| Central adiposity is associated with poorer functional capacity in women with knee osteoarthritis: a cross-sectional study with exploratory adjustment for pain intensity and radiographic severity. Voir source | 42443640 | 10.1007/s00296-026-06245-7 | Rheumatology international | |
| Effects of Pulmonary Rehabilitation Nursing on Pulmonary Function and Exercise Capacity in Patients With COPD: A Meta-Analysis. Voir source | 42444418 | 10.1111/nicc.70579 | Nursing in critical care | |
| Multimodal Integration of Gait, Balance, and Infrared Thermography Enhances Machine-Learning Classification of Knee Osteoarthritis: A Cross-Sectional Study. Voir source | 42435928 | 10.1016/j.apmr.2026.06.029 | Archives of physical medicine and rehabilitation | |
| A comparison of conservative versus surgical treatment in patients with foot drop due to peroneal nerve entrapment: results of a prematurely terminated randomized controlled trial. Voir source | 42436915 | 10.1016/j.bas.2026.106160 | Brain & spine | |
| The Impact of Exercise Tolerance Assessed by 6-Min Walking Distance on Clinical Outcomes in Patients With Interstitial Lung Disease Associated With Connective Tissue Diseases. Voir source | 42423508 | 10.1111/1756-185x.70765 | International journal of rheumatic diseases | |
| Pulmonary function and physical fitness in obese children and adolescents: a comparison of impulse oscillometry, spirometry, and 6-min walk test. Voir source | 42423832 | 10.1007/s00431-026-07240-7 | European journal of pediatrics | |
| Feasibility and behavioral impact of a wearable-supported digital health intervention on attitudes and behaviors toward sleep and physical activity: A proof-of-concept study in Kobe City. Voir source | 42416388 | 10.1177/20552076261466410 | Digital health | |
| Five-Year Outcomes of Paclitaxel-Coated Balloons in Patients with Diabetes from the BIOLUX P-III Single-Arm Study. Voir source | 42416524 | 10.2147/VHRM.S595482 | Vascular health and risk management | |
| Yiqi Fumai Lyophilized Injection for Improving Exercise Tolerance in Chronic Heart Failure: Protocol for a Prospective Cohort Study. Voir source | 42413004 | 10.2196/91051 | JMIR research protocols | |
| The reliability and validity of the 30-second chair sit-to-stand test and the five-repetition sit-to-stand test in individuals following lumbar spinal surgery. Voir source | 42414666 | 10.1007/s00586-026-10182-1 | European spine journal : official publication of the European Spine Society, the European Spinal Deformity Society, and the European Section of the Cervical Spine Research Society |
Sources officielles
liens de rechercheDernière mise à jour des données : 2026-03-20
Ces sources sont proposées pour vérification. Les informations de la fiche doivent être confirmées dans les registres officiels ou les publications originales.