Clinical trial NCT06089863: Rehabilitation Program on Genetic and Degenerative Ataxia
Official title: Evaluation of the Effect of PAMPERO Rehabilitation Program in Collaboration With Patient Partner on the Symptom Intensity, Activity, and Quality of Life on Genetic and Degenerative Ataxia
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Ataxia
Degenerative Disease
Genetic Disease
Keywords: Genetic ataxia
Degenerative ataxia
Rehabilitation
Cerebellar ataxia
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2023-10-18
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.


Eligibility criteria: Inclusion Criteria:

* ≥ 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)

Non inclusion Criteria:

* 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

Primary outcomes:
measure: Scale for the Assessment and Rating of Ataxia (SARA)
timeFrame: Inclusion ; 3 months after the end of rehabilitation
Secondary outcomes:
measure: Scale for the Assessment and Rating of Ataxia (SARA)
timeFrame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 6 months after the end of rehabilitation
measure: Score of the Mini-BESTest scale
timeFrame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
measure: Eyes open postural surface
timeFrame: 5 weeks
measure: Displacement length of the center of mass as a function of time eyes open
timeFrame: 5 weeks
measure: Scale of Short Falls Efficacy Scale International (Short FES-I)
timeFrame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
measure: Number of weekly steps
timeFrame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
measure: Time of weekly sedentary activity
timeFrame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
measure: Light, moderate and vigorous weekly activity
timeFrame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
measure: Short Form Health Survey (SF-36)
timeFrame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
measure: Number of falls during the study
timeFrame: 4 weeks ; up to 3 months ; up to 6 months
measure: Number of patients who completed the entire PAMPERO program
timeFrame: 5 weeks
measure: Number of patients who stopped PAMPERO program
timeFrame: 5 weeks
measure: Time to occurrence of PAMPERO program stops
timeFrame: 5 weeks
measure: Reasons of PAMPERO program stops
timeFrame: 5 weeks
Other outcomes:
Locations:
city: Saint-Didier-au-Mont-d'Or
country: France
facility: Service de Rééducation Fonctionnelle (S.S.R.) Val Rosay
city: Saint-Genis-Laval
country: France
facility: Service de Rééducation Fonctionnelle (S.S.R.) de l'Hôpital Henry Gabrielle Hospices Civils de Lyon
city: Saint-Étienne
country: France
facility: Service de Médecine Physique et Réadaptation (M.P.R) de l'Hôpital Bellevue
Study officials:
References: