AtaxiaRAPPNCT06089863
Rehabilitation Program on Genetic and Degenerative Ataxia
A study testing PAMPERO program in people with Ataxia and Degenerative Disease, run by Hospices Civils de Lyon at 3 sites in France.
Trial 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.). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ataxia, Degenerative Disease, Genetic Disease
- What is being tested?
- PAMPERO program
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 3 sitesFrance
- Study dates
- 1 Feb 2024to 1 Oct 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ≥ 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)
Show all 14Show fewer
- 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
Who cannot join
None listed.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
France3 sites
Service de Rééducation Fonctionnelle (S.S.R.) Val Rosay
Saint-Didier-au-Mont-d'Or, 69370
Julie MD Di Marco, 04 72 53 21 65 ext. +33, julie.dimarco@ugecam.assurance-maladie.fr
Service de Médecine Physique et Réadaptation (M.P.R) de l'Hôpital Bellevue
Saint-Etienne, 42100
Pascal MD Giraux, 04 77 12 77 57 ext. +33, pascal.giraux@univ-st-etienne.fr
Service de Rééducation Fonctionnelle (S.S.R.) de l'Hôpital Henry Gabrielle Hospices Civils de Lyon
Saint-Genis-Laval, 69230
Lucas PIGNON, 04 78 86 50 58 ext. +33, lucas.pignon@chu-lyon.fr
About this study
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
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.
Read more
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
- Being tested
- PAMPERO program
- Keywords
- Genetic ataxia, Degenerative ataxia, Rehabilitation, Cerebellar ataxia
- Medical subject headings
- Ataxia, Genetic Diseases, Inborn, Cerebellar Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PAMPERO programOther
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
| Study group | Role | What they receive |
|---|---|---|
| PAMPERO program group | Experimental | This program is an intensive multidisciplinary program rehabilitation including Physical, Occupational, Speech, Psychomotor therapy and Adapted Physical Activity. Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Inclusion ; 3 months after the end of rehabilitation
Secondary outcomes (14)
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.
Measured: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 6 months after the end of rehabilitation
Score of the Mini-BESTest scale
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.
Measured: 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
Measure of the balance evaluate by the Eyes open postural surface
Measured: 5 weeks
Displacement length of the center of mass as a function of time eyes open
Measure of the balance evaluated by the displacement length of the center of mass as a function of time eyes open
Measured: 5 weeks
Scale of Short Falls Efficacy Scale International (Short FES-I)
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.
Measured: 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 weekly steps
Number of steps during one week continuously measured by actimeter ActiGraph wGT3X-BT with EUROCOC (European Certificate of Conformity)
Measured: 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 of weekly sedentary activity
Time in minutes/day of sedentary activity during one week continuously measured by actimeter ActiGraph wGT3X-BT with EUROCOC
Measured: 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 weekly activity
Light, moderate and vigorous activity during one week continuously measured by actimeter ActiGraph wGT3X-BT with EUROCOC
Measured: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
Short Form Health Survey (SF-36)
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.
Measured: 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 falls during the study
Risk of falling evaluated by the number of falls during and outside the rehabilitation normalized on 4 weeks
Measured: 4 weeks ; up to 3 months ; up to 6 months
Number of patients who completed the entire PAMPERO program
Number of patients who completed the entire PAMPERO program (5 weeks)
Measured: 5 weeks
Number of patients who stopped PAMPERO program
Number of patients who stopped PAMPERO program
Measured: 5 weeks
Time to occurrence of PAMPERO program stops
Time to occurrence of PAMPERO program stops
Measured: 5 weeks
Reasons of PAMPERO program stops
Reasons of PAMPERO program stops
Measured: 5 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2023
- Study start1 Feb 2024 (estimated)
- Primary completion1 Jul 2027 (estimated)
- Study completion1 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AtaxiaStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06089863
- Sponsor's protocol ID
- 69HCL22_0912
- Acronym
- RAPP
- Status last verified
- Oct 2023
- First submitted
- 9 Oct 2023
- Last updated
- 17 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06089863
- Acronym / sponsor ID
- RAPP / 69HCL22_0912
- Conditions
- Ataxia, Degenerative Disease, Genetic Disease
- Keywords
- Genetic ataxia, Degenerative ataxia, Rehabilitation, Cerebellar ataxia
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 1 Feb 2024 (estimated)
- Primary completion
- 1 Jul 2027 (estimated)
- Study completion
- 1 Oct 2027 (estimated)
- First posted
- 18 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PAMPERO program group | Experimental | Other: PAMPERO programThis program is an intensive multidisciplinary program rehabilitation including Physical, Occupational, Speech, Psychomotor therapy and Adapted Physical Activity. |
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- ≥ 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
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PAMPERO programThis 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 | Other | PAMPERO program group |
Primary outcome
Scale for the Assessment and Rating of Ataxia (SARA)
Time frame: 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.
14 secondary outcomes
Scale for the Assessment and Rating of Ataxia (SARA)
Time frame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 6 months after the end of rehabilitation
Score of the Mini-BESTest scale
Time frame: 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
Time frame: 5 weeks
Displacement length of the center of mass as a function of time eyes open
Time frame: 5 weeks
Scale of Short Falls Efficacy Scale International (Short FES-I)
Time frame: 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 weekly steps
Time frame: 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 of weekly sedentary activity
Time frame: 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 weekly activity
Time frame: Inclusion ; Immediately or 1 week after the end of rehabilitation ; 3 months after the end of rehabilitation ; 6 months after the end of rehabilitation
Short Form Health Survey (SF-36)
Time frame: 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 falls during the study
Time frame: 4 weeks ; up to 3 months ; up to 6 months
Number of patients who completed the entire PAMPERO program
Time frame: 5 weeks
Number of patients who stopped PAMPERO program
Time frame: 5 weeks
Time to occurrence of PAMPERO program stops
Time frame: 5 weeks
Reasons of PAMPERO program stops
Time frame: 5 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PAMPERO program in people with Ataxia and Degenerative Disease, run by Hospices Civils de Lyon at 3 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 17 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lucas PIGNON (04 78 86 50 58 ext. +33, lucas.pignon@chu-lyon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Scale for the Assessment and Rating of Ataxia (SARA) (time frame: Inclusion ; 3 months after the end of rehabilitation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jan 2024.