Rehabilitation Program on Genetic and Degenerative Ataxia

NCT06089863 | Not Yet Recruiting

• 1 other identifier: 69HCL22_0912
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 3

Brief Summary

Cerebellar ataxia is a pathology linked to the lesion of the cerebellum or the afferent and/or efferent cerebellar pathways. The aetiology can be an acquired cerebral lesion, following a chemical poisoning or a genetic degenerative lesion (for example : Friedreich's ataxia, spinocerebellar ataxias, etc.). As reported by the latest estimate available, genetic degenerative cerebellar ataxias affect approximately 6,000 patients in France (Orpha.net). Symptoms suffered by ataxic patients are : problems and gait disorders along with difficulties in coordination resulting in ataxia, uncoordinated movements. These symptoms cause a decrease in the quality of life on patients with spinocerebellar ataxia. The symptoms improvement linked to the cerebellar syndrome is based on rehabilitation that can be supplemented by use of technical aids. Current scientific knowledge confirms that intensive rehabilitation by physiotherapy and occupational therapy in patients with degenerative ataxias improves cerebellar symptoms. Nevertheless, the choice rehabilitation technique stay at the appreciation of the therapist. From the observation, the investigators have designed an intensive multidisciplinary rehabilitation program, called PAMPERO, with partner patients member of two genetic degenerative ataxia patient organisations. This 5-weeks program has been used in clinic during 3 years on 28 patients. It appears to be the only one in France. The preliminary results show a positive effect on ataxia symptom. Nevertheless, the duration of the benefice over time and the effect on the quality of life stay unknown. However, the quality of life is mainly affected by the participation restriction due to the risk of falling. The most frequent complaint from partner patient is the diminution of the social interaction resulting of the incapacity to move without risk. The present protocol aimed at evaluating the Rehabilitation Program in collaboration with partner patient on the symptom intensity, activity and quality of life on genetic and degenerative ataxia. This PAMPERO program's effect will be assessed by comparing the difference of Intensity of symptom measured by to Scale for the Assessment and Rating of Ataxia (SARA) at inclusion and 3 months after the end of rehabilitation.

Conditions

Ataxia; Degenerative Disease; Genetic Disease

Keywords

Genetic ataxia; Degenerative ataxia; Rehabilitation; Cerebellar ataxia

Outcome Measures

Primary Outcomes (1)

• Scale for the Assessment and Rating of Ataxia (SARA)

  Intensity of symptom measured by to Scale for the Assessment and Rating of Ataxia (SARA). Scores are from 0 to 42. An higher score is associated with a worse outcome.

  Inclusion ; 3 months after the end of rehabilitation

Secondary Outcomes (14)

• Scale for the Assessment and Rating of Ataxia (SARA)

  Inclusion ; Immediately or 1 week after the end of rehabilitation ; 6 months after the end of rehabilitation

• Score of the Mini-BESTest scale

  Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation

• Eyes open postural surface

  5 weeks

• Displacement length of the center of mass as a function of time eyes open

  5 weeks

• Scale of Short Falls Efficacy Scale International (Short FES-I)

  Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation

Study Arms (2)

PAMPERO program group

EXPERIMENTAL

This program is an intensive multidisciplinary program rehabilitation including Physical, Occupational, Speech, Psychomotor therapy and Adapted Physical Activity.

Other: PAMPERO program

Usual Care intervention group

NO INTERVENTION

Usual Rehabilitation of the patient after the inclusion. 0 to 2 physical therapy sessions and 0 to 2 Speech therapy sessions each week usually constitute this program. This rehabilitation highly depends on the patients usual preferences. Some patients have no rehabilitation in the daily care while some have more.

Interventions

PAMPERO program OTHER

This program is an intensive multidisciplinary program rehabilitation including Physical, Occupational, Speech, Psychomotor therapy and Adapted Physical Activity. The duration of this program is 5 weeks and each week is divided as follow : * 5 hours of physical therapy : individual session with the objective to improve the dynamic balance and coordination on the aim to gain confidence of moving, gripping capacity and interaction with object * 5 hours of Adapted Physical activity : group session to promote pairing with muscle building priority * 1 hour of Speech therapy : group session to educate and promote prevention strategy about dysphagia rick * 2 hours of Occupational therapy : group session to bring adaptive solution of autonomy lost with material propose, home organisation or gesture * 2 hours of Hydrotherapy : group session activity performed in water to assist rehabilitation

PAMPERO program group

Eligibility Criteria

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

You may qualify if:

• ≥ 18 years
• Patients with genetic and degenerative cerebellar ataxia
• Diagnostic of cerebellar ataxia confirmed by anatomic MRI
• Affiliated to a social insurgence regime or similar
• Patients who have given their free, informed and express consent
• Walking patients (who can walk unsupervised on flat ground with the help of proper technique : categories greater than 4 on the New Functional Ambulation Classification scale)
• Patients who have already benefited from a hospitalization of more than 3 weeks for rehabilitation during the last 12 months
• Patients participating simultaneously in another research whose objective would be the evaluation of a therapy, medicinal or not, likely to improve neurological or functional recovery
• Pregnants, parturient or breastfeeding
• Patients deprived of their liberty by a judicial or administrative decision
• Psychiatric care patients
• Patients admitted to Patients admitted to a health or social establishment for purposes other than research for purposes other than research
• Major patients protected by the Law

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

Study Sites (3)

Service de Rééducation Fonctionnelle (S.S.R.) Val Rosay

Saint-Didier-au-Mont-d'Or, 69370, France

Location

Service de Médecine Physique et Réadaptation (M.P.R) de l'Hôpital Bellevue

Saint-Etienne, 42100, France

Location

Service de Rééducation Fonctionnelle (S.S.R.) de l'Hôpital Henry Gabrielle Hospices Civils de Lyon

Saint-Genis-Laval, 69230, France

Location

MeSH Terms

Conditions

Ataxia; Genetic Diseases, Inborn; Cerebellar Ataxia

Condition Hierarchy (Ancestors)

Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases

Central Study Contacts

Lucas PIGNON

CONTACT

04 78 86 50 58; lucas.pignon@chu-lyon.fr

Christelle MAROLHO

CONTACT

04 72 11 57 68; christelle.marolho@chu-lyon.fr

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 9, 2023
• First Posted: October 18, 2023
• Study Start: February 1, 2024
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): October 1, 2027
• Last Updated: January 17, 2024

Record last verified: 2023-10

Locations

FR (3)