Rehabilitation Program on Genetic and Degenerative Ataxia (RAPP)

Civil Hospices of Lyon

Status: Not yet enrolling

Conditions: Degenerative Disease; Ataxia; Genetic Disease

Treatments: Other: PAMPERO program

Study type: Interventional

Funder types: Other

Identifiers

NCT06089863

69HCL22_0912

Details and patient eligibility

About

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.

Enrollment: 48 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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48 participants in 2 patient groups

PAMPERO program group

Experimental group

Description:

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

Treatment:

Other: PAMPERO program

Usual Care intervention group

No Intervention group

Description:

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.