Rehabilitation Program for Spastic Ataxia

Recruiting at 5 trial locations

Overseen By Andrée Hardy

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Université du Québec à Chicoutimi

Disqualifiers: Rehab program, Physical limitations, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

84 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a special 12-week rehabilitation program designed to help individuals with spastic ataxias, a condition that affects walking and balance. The study compares the effects of this new program, called IMPACT (rehabIlitation prograM for sPAstiC aTaxias), against regular care to determine if it reduces disease severity and to explore its implementation in real-world settings. Participants will either continue with their usual care or join the program, which includes therapy sessions and aquatherapy (water-based exercises) three times a week. Individuals with spastic ataxias who can stand and move with assistance may be suitable for this trial. As an unphased trial, this study provides a unique opportunity to contribute to innovative rehabilitation strategies that could enhance the quality of life for those with spastic ataxias.

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 prior data suggests that this rehabilitation program is safe for individuals with spastic ataxias?

Research has shown that the IMPACT rehabilitation program for spastic ataxias is safe and manageable for participants. In a previous study, participants experienced noticeable improvements in balance, walking, and daily activities without serious side effects. Other studies support these findings, indicating that personalized rehabilitation programs can effectively reduce symptoms while remaining safe and acceptable for patients. This program aims to help people with spastic ataxias, conditions that cause walking and balance problems, and has shown promising results in enhancing their quality of life. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the IMPACT rehabilitation program for spastic ataxias because it offers a structured, intensive approach that isn't typically available with the current standard of care. Unlike typical treatments that might involve sporadic physiotherapy and occupational therapy sessions, this program involves a consistent schedule of three sessions per week over 12 weeks, incorporating both traditional therapy and aquatherapy. This comprehensive and regular regimen could potentially lead to more significant improvements in mobility and daily functioning for patients with spastic ataxia.

What evidence suggests that the IMPACT rehabilitation program is effective for spastic ataxia?

Research has shown that a special rehabilitation program for spastic ataxias yields promising results. In earlier studies, participants experienced significant improvements in balance, walking, and daily activities after completing the program. Another study found that personalized home exercises reduced ataxia symptoms and improved balance. Evidence supports that rehabilitation can enhance posture in people with cerebellar ataxias. In this trial, participants in the intervention group will follow the IMPACT rehabilitation program, which may effectively lessen disease symptoms and improve daily life for those with spastic ataxias. ^{1 2 3 4 5}

Who Is on the Research Team?

Elise Duchesne, Ph D.

Principal Investigator

Université du Québec à Chicoutimi

Are You a Good Fit for This Trial?

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.

Be uncomfortable in a swimming pool

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• IMPACT - rehabIlitation prograM for sPAstiC aTaxias

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

A 12-week physical therapy program, including balance and strength exercises as well as Wii Fit training, led to significant improvements in motor performance and balance in a 9-year-old girl with ataxia-telangiectasia (AT).

The therapy resulted in positive changes in multiple assessments, including the Gross Motor Function Measurement and Pediatric Berg Balance Scale, indicating enhanced quality of life and participation in daily activities for the child.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of Physical Therapy on Ataxia-Telangiectasia: A Case Report. Unes, S., Tuncdemir, M., Eroglu-Ertugrul, NG., et al.[2023]

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]

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]

The Ataxia Global Initiative (AGI) aims to standardize clinical outcome assessments (COAs) for ataxia, creating a mandatory dataset for routine clinical consultations and an extended dataset for research, which will enhance trial readiness and data sharing.

The initiative emphasizes the need to develop the scale for the assessment and rating of ataxia (SARA) into a widely accepted tool for clinical trials, while also calling for more data on patient-reported outcomes and methods to measure meaningful changes in patient health.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06261424?cond=(SPINOCEREBELLAR%20ATAXIA,%20AUTOSOMAL%20RECESSIVE)%20OR%20(VPS41)&rank=6

Effects of a Supervised Rehabilitation Program on Disease ... 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 ...

2. withpower.com withpower.com/trial/phase-muscle-spasticity-1-2024-58bbb

Rehabilitation Program for Spastic Ataxia The study on the rehabilitation program for spastic ataxia of Charlevoix-Saguenay showed significant improvements in balance, walking, and daily activities ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11914335/

A Tailored Home-Based Training Program Improved Ataxia ... In this study, participants significantly improved their balance, ataxia severity, and ability to perform activities of daily living.

4. clinicaltrial.be clinicaltrial.be/en/details/425574?only_active=0&only_eligible=0&only_recruiting=0&per_page=20

Effects of a Supervised Rehabilitation Program on Disease... The rehabilitation program will last 12 weeks and consists of three training sessions of 60 minutes per week (2 sessions in a therapy room and 1 ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9648943/

Rehabilitation in patients with cerebellar ataxias - PMC They concluded that there was moderate level of evidence that rehabilitation was efficient in improving postural capacities of patients with cerebellar ataxia, ...