Rehabilitation Program for Spastic Ataxia

Recruiting at 4 trial locations

Overseen By Andrée Hardy

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: Université du Québec à Chicoutimi

Disqualifiers: Rehab program, Physical limitations, Pregnancy, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

Spastic ataxias are a group of diseases causing symptoms such as walking difficulties and balance impairments that lead to a high risk of falls. No pharmacological treatments exist to treat these diseases. Unfortunately, little effort is made to develop non-pharmacological treatments specific to spastic ataxias despite the detrimental impact of the disease on several aspects of an individual's life and the high cost of falls for society each year. The three objectives of this project are: 1) to determine the effect of a 12-week rehabilitation program on disease severity as compared with usual care for individuals with spastic ataxias; 2) to identify which factors can help (or not) the implementation of the program in the clinical settings ("reel world"); and 3) to explore the cost-benefits of IMPACT \[rehabIlitation prograM for sPAstiC aTaxias\]. The team has developed the program to specifically target symptoms present in these patients and was previously pilot-tested. Based on the results obtained in this pilot project, positive effects are expected concerning the disease severity of participants. The investigators want, with this project, provide to health care professionals an option to offer better-suited services to people living with spastic ataxia worldwide.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It focuses on a rehabilitation program rather than medication changes.

What data supports the effectiveness of the treatment IMPACT - rehabIlitation prograM for sPAstiC aTaxias?

A study on a similar rehabilitation program for people with spastic ataxia showed significant improvements in balance, walking speed, and daily activities. Participants also reported better posture and mobility, suggesting that such programs can positively impact daily life. ^{1 2 3 4 5}

Is the Rehabilitation Program for Spastic Ataxia safe for humans?

The study on the rehabilitation program for spastic ataxia of Charlevoix-Saguenay showed significant improvements in balance, walking, and daily activities without mentioning any safety concerns, suggesting it is generally safe for humans. ^{2 3 4 6 7}

How is the IMPACT rehabilitation program for spastic ataxia different from other treatments?

The IMPACT rehabilitation program is unique because it combines group-supervised sessions in a gym and pool to improve balance, trunk control, and walking abilities in people with spastic ataxia. This approach focuses on enhancing daily living activities and functional mobility, which is not commonly addressed in standard treatments for this condition. ^{2 4 7 8 9}

Research Team

Elise Duchesne, Ph D.

Principal Investigator

Université du Québec à Chicoutimi

Eligibility Criteria

This trial is for individuals with spastic ataxias, a group of conditions causing walking difficulties and balance problems. Participants should have these symptoms but not be undergoing any other specific treatments for them during the trial.

Inclusion Criteria

Be authorized by their treating neurologist

Be able to give informed consent.

I have a genetic diagnosis of ARSACS or SPG7.

Exclusion Criteria

Have another condition causing physical limitations

Be pregnant.

I am actively participating in a rehabilitation program.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants in the intervention group will follow a 12-week rehabilitation program, 3 times a week (two sessions in a therapy room and one session of aquatherapy).

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, with various assessments conducted over a 64-week period.

64 weeks

Treatment Details

Interventions

• IMPACT - rehabIlitation prograM for sPAstiC aTaxias (Behavioural Intervention)

Trial Overview The study tests a 12-week supervised rehabilitation program called IMPACT, designed to reduce disease severity in spastic ataxias compared to usual care. It also examines how well this program can be implemented in clinical settings and its cost-effectiveness.

Participant Groups

Group I: Intervention group Experimental Treatment 1 Intervention

The intervention group will follow a 12-week rehabilitation program, 3 times a week (two sessions in a therapy room and one session of aquatherapy).

Group II: Control group Active Control 1 Intervention

The control group will receive their usual care and will be asked to continue their usual activities and exercises for the whole project duration. Only one clinical guide exists for ataxia interventions and no specific guidelines for physiotherapy and occupational therapy interventions are specified in this guide in terms of frequency, duration and type of interventions that are effective with this population. Patients generally have an annual follow-up with a doctor or a clinical nurse at a Neuromuscular Diseases Clinic. Patients are then referred if needed for follow-up with the physiotherapist and occupational therapist. Typically, patients are referred to physiotherapy for mobility-related needs such as the introduction of a walking aid, orthotic and sometimes teaching an exercise program. In occupational therapy, patients are referred for needs such as the introduction of technical aids, home adaptation or the introduction of a wheelchair.

Who Is Running the Clinical Trial?

Université du Québec à Chicoutimi

Lead Sponsor

Trials

7

Recruited

320+

Centre intégré universitaire de santé et de services sociaux du Saguenay-Lac-St-Jean

Collaborator

Trials

1

Recruited

80+

Integrated University Health and Social Services Center of the Capitale-Nationale

Collaborator

Trials

5

Recruited

580+

Corporation de recherche et d'action sur les maladies héréditaires (CORAMH)

Collaborator

Trials

1

Recruited

80+

University of Alberta

Collaborator

Trials

957

Recruited

437,000+

Muscular Dystrophy Canada

Collaborator

Trials

4

Recruited

170+

Cégep de Jonquière

Collaborator

Trials

1

Recruited

80+

Université du Québec a Montréal

Collaborator

Trials

28

Recruited

5,600+

University of Calgary

Collaborator

Trials

827

Recruited

902,000+

Université de Sherbrooke

Collaborator

Trials

317

Recruited

79,300+

Findings from Research

Rehabilitation interventions for spinocerebellar ataxia showed significant improvements in balance and gait, with statistical significance for posture (P<.008) and gait (P<.02).

The interventions also led to notable reductions in speech disorders (P=.02), depressive symptoms (P<.0001), and accidental falls (P<.005), indicating a broader impact on quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Effect of physical rehabilitation in patients with hereditary spinocerebellar ataxia. A systematic review]. Marchal-Muñoz, A., García-Aranda, M., Marchal-Muñoz, M., et al.[2021]

An 8-week group rehabilitation program for individuals with Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS) led to significant improvements in balance, walking speed, and daily living activities, indicating its efficacy in enhancing motor control.

All participants showed measurable improvements in at least two outcome measures, and most reported positive changes in balance and mobility, suggesting that this type of rehabilitation could greatly benefit their quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A rehabilitation program to increase balance and mobility in ataxia of Charlevoix-Saguenay: An exploratory study. Lessard, I., Masterman, V., Côté, I., et al.[2023]

The Action Research Arm Test (ARAT) has been shown to have strong interrater reliability and moderate construct validity for assessing upper extremity activity limitations in individuals with ataxia, based on a study of 22 participants.

While the ARAT correlates well with body function and structure measures, it does not correlate with participation measures, highlighting its specific focus on upper extremity activity rather than overall participation in daily tasks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Can the ARAT Be Used to Measure Arm Function in People With Cerebellar Ataxia? Reoli, R., Cherry-Allen, K., Therrien, A., et al.[2023]

The Person-Reported Ataxia Impact Scale (PRAIS) is a newly developed 38-item questionnaire designed to assess the impact of autosomal recessive cerebellar ataxias from the perspective of those affected, based on a study involving 125 participants.

PRAIS demonstrated strong validity and reliability, effectively distinguishing between different stages of mobility and levels of autonomy, making it a valuable tool for both clinical and research applications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Development of a New Patient-Reported Outcome Measure in Recessive Ataxias: The Person-Reported Ataxia Impact Scale. Tremblay, M., Brais, B., Asselin, V., et al.[2023]

In a study of 19 adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) over 2 years, significant declines were observed in lower limb functions, mobility, and balance, indicating a progressive deterioration in these areas.

Despite the overall decline in mobility and balance, upper limb coordination and fine dexterity remained stable, suggesting that ARSACS primarily affects lower limb functions over time.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An exploratory natural history of ataxia of Charlevoix-Saguenay: A 2-year follow-up. Gagnon, C., Lessard, I., Lavoie, C., et al.[2023]

A study involving 45 children with ataxia showed that a 6-month home-based rehabilitation program, including vibration-assisted therapy, significantly improved motor function, as measured by the Gross Motor Function Measure (GMFM-66).

The improvements in motor skills were maintained or further developed after an additional 6 months, particularly in children with non-progressive ataxia, indicating the long-term efficacy of this rehabilitation approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Motor Function Improvement in Children with Ataxia Receiving Interval Rehabilitation, Including Vibration-Assisted Hometraining: A Retrospective Study. Martakis, K., Stark, C., Alberg, E., et al.[2022]

A systematic review of 20 studies involving 40 children with ataxia found that while exercise and physical therapy interventions showed promising results, the studies were of low methodological quality and lacked randomized controlled trials (RCTs).

Due to the small sample sizes and variability in interventions and outcomes, no definitive conclusions can be drawn about the effectiveness of these therapies for children with ataxia, highlighting the need for more rigorous, child-centered research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Exercise and Physical Therapy Interventions for Children with Ataxia: A Systematic Review. Hartley, H., Cassidy, E., Bunn, L., et al.[2020]

References

1. Spain pubmed.ncbi.nlm.nih.gov

[Effect of physical rehabilitation in patients with hereditary spinocerebellar ataxia. A systematic review]. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

A rehabilitation program to increase balance and mobility in ataxia of Charlevoix-Saguenay: An exploratory study. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Can the ARAT Be Used to Measure Arm Function in People With Cerebellar Ataxia? [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Effectiveness of Physical Therapy on Ataxia-Telangiectasia: A Case Report. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

The Development of a New Patient-Reported Outcome Measure in Recessive Ataxias: The Person-Reported Ataxia Impact Scale. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

An exploratory natural history of ataxia of Charlevoix-Saguenay: A 2-year follow-up. [2023]

8. Germany pubmed.ncbi.nlm.nih.gov

Motor Function Improvement in Children with Ataxia Receiving Interval Rehabilitation, Including Vibration-Assisted Hometraining: A Retrospective Study. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Exercise and Physical Therapy Interventions for Children with Ataxia: A Systematic Review. [2020]