RAPP: Rehabilitation Program on Genetic and Degenerative Ataxia

Sponsor

Hospices Civils de Lyon (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT06089863

Collaborator

(none)

Enrollment

Locations

Arms

Anticipated Duration (Months)

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Ataxia Degenerative Disease Genetic Disease	Other: PAMPERO program	N/A

Study Design

Study Type:

Interventional

Anticipated Enrollment :

48 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Other

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

Anticipated Study Start Date :

Nov 1, 2023

Anticipated Primary Completion Date :

Apr 1, 2027

Anticipated Study Completion Date :

Jul 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: PAMPERO program group This program is an intensive multidisciplinary program rehabilitation including Physical, Occupational, Speech, Psychomotor therapy and Adapted Physical Activity.	Other: PAMPERO program 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
No Intervention: Usual Care intervention group 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.

Arm

Intervention/Treatment

Experimental: PAMPERO program group

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

Other: PAMPERO program

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

No Intervention: Usual Care intervention group

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.

Outcome Measures

Primary Outcome Measures

Scale for the Assessment and Rating of Ataxia (SARA) [Inclusion ; 3 months after the end of rehabilitation]
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.

Secondary Outcome Measures

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]
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.
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]
Measure of the balance evaluate by Mini-BESTest scale (Mini-Balance Evaluation Systems Test). Scores are from 0 to 28. An higher score is associated with a better outcome.
Eyes open postural surface [5 weeks]
Measure of the balance evaluate by the Eyes open postural surface
Displacement length of the center of mass as a function of time eyes open [5 weeks]
Measure of the balance evaluated by the displacement length of the center of mass as a function of time eyes open
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]
Fear of fall evaluated by to Scale of Short Falls Efficacy Scale International (Short FES-I). Scores are from 7 to 28. An higher score is associated with a worse outcome.
Number of weekly steps [Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation]
Number of steps during one week continuously measured by actimeter ActiGraph wGT3X-BT with EUROCOC (European Certificate of Conformity)
Time of weekly sedentary activity [Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation]
Time in minutes/day of sedentary activity during one week continuously measured by actimeter ActiGraph wGT3X-BT with EUROCOC
Light, moderate and vigorous weekly activity [Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation]
Light, moderate and vigorous activity during one week continuously measured by actimeter ActiGraph wGT3X-BT with EUROCOC
Short Form Health Survey (SF-36) [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 of the quality of life evaluate by Short Form Health Survey (SF-36). Scores are from 0 to 100. An higher score is associated with a better outcome.
Number of falls during the study [4 weeks ; up to 3 months ; up to 6 months]
Risk of falling evaluated by the number of falls during and outside the rehabilitation normalized on 4 weeks
Number of patients who completed the entire PAMPERO program [5 weeks]
Number of patients who completed the entire PAMPERO program (5 weeks)
Number of patients who stopped PAMPERO program [5 weeks]
Number of patients who stopped PAMPERO program
Time to occurrence of PAMPERO program stops [5 weeks]
Time to occurrence of PAMPERO program stops
Reasons of PAMPERO program stops [5 weeks]
Reasons of PAMPERO program stops

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Service de Rééducation Fonctionnelle (S.S.R.) Val Rosay	Saint-Didier-au-Mont-d'Or	France	69370
2	Service de Rééducation Fonctionnelle (S.S.R.) de l'Hôpital Henry Gabrielle Hospices Civils de Lyon	Saint-Genis-Laval	France	69230
3	Service de Médecine Physique et Réadaptation (M.P.R) de l'Hôpital Bellevue	Saint-Étienne	France	42100