Autosomal Recessive Spastic Ataxia of Charlevoix-SaguenayPACE-ARSCSNCT05768750
A Home-based Rehabilitation in ARSACS
A study testing Rehabilitation in people with Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay, run by Université de Sherbrooke at 1 site in Canada.
Trial summary
48 participants (24 women and 24 men) with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) will participate in 2 phases : control phase (12-week usual care) and intervention phase (12-week home-based rehabilitation program). Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
- What is being tested?
- Rehabilitation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteCanada
- Study dates
- 1 Dec 2022to 1 Dec 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- ARSACS diagnosis must be confirmed by genetic analysis;
- Women and men, aged between 18 and 50 years old;
- Be able to perform the sit-to-stand transfer;
- Consent of the neurologist must be given to participate in this study;
- Must reside in the Saguenay-Lac-St-Jean region;
- Subjects must be able to give their consent freely and voluntarily.
Who cannot join
- Patients who already meet physical activity (PA) recommendations (150 min of moderate to high intensity PA/week) or already participate in a rehabilitation program are excluded;
- Remain in a care facility;
- Do not speak English or French;
- Have another diagnosis causing physical limitations;
- Are pregnant.
Trial locations and contacts
1 site in 1 country.
Canada1 site, 1 recruiting
- Recruiting
Groupe de recherche interdisciplinaire sur les maladies neuromusculaires
Saguenay, Quebec, G7X 7X2
Elise Duchesne, Ph.D., 418-545-5011 ext. 6148, elise1_duchesne@uqac.ca
Elise Duchesne, Ph.D.
About this study
Official title: A Home-based Rehabilitation Program to Counter the Motor Impairments and Activity Limitations Experienced by People With Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
48 participants (24 women and 24 men) with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) will participate in 2 phases : control phase (12-week usual care) and intervention phase (12-week home-based rehabilitation program). The participants will be evaluated at baseline, week 12 (end of control phase) and week 24 (end of intervention phase) to quantify the effects of an individualized home-based rehabilitation program. Participants will also participate on a focus group at the end of the program to evaluate the acceptability of the program and the perceived changes.
Read the detailed description
Co-creation phase:
Preceding the intervention, a co-creation phase involving healthcare professionals (3), patient-partners (2), and ARSACS researchers (3) will be conducted to develop the exercises for the rehabilitation program and the evaluation scale to determine the level of difficulty of each exercise assigned to participants.
Intervention phase:
Random sampling stratified by gender and level of indoor mobility (unassisted walking, assisted walking, wheelchair) from the Neuromuscular Clinic's registry of 48 participants (24 women and 24 men) will be conducted.
- Control phase: All participants will be asked to maintain their usual activities for 12 weeks.
- Intervention phase: All participants recruited to the project will complete the home-based rehabilitation program assigned to them for 12 weeks, unsupervised, 20 minutes 3 times a week. Follow-up calls by a physiotherapist will be conducted at weeks 2, 4, 6, 8, 10 and 12.
- Being tested
- Rehabilitation
- Keywords
- ARSACS
- Medical subject headings
- Spastic ataxia Charlevoix-Saguenay type
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RehabilitationOther
X
| Study group | Role | What they receive |
|---|---|---|
| Control followed by intervention phase | Experimental | All participants will participate in 2 phases : control phase (12-week usual care) followed by an intervention phase (12-week home-based rehabilitation program).
The rehabilitation program consists of three domains of exercises divided by levels of difficulty: sitting balance (16 levels), standing balance (21 levels) and sit-to-stand transfer (12 levels), including 67 exercises. Each individualized home-based rehabilitation program will include 3 to 6 exercises, for a total duration of 15 to 20 minutes. Receives: Other: Rehabilitation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in sitting balance
Measured with the Ottawa sitting scale, score range 0-40, a higher score indicates a better outcome
Measured: Baseline, week 12, week 24
Change in balance
Measured with the Berg Balance Scale, score range 0-56, a higher score indicates better balance
Measured: Baseline, week 12, week 24
Change in walking speed
Change in the walking speed, measured with the time to walk 10 meters at self-selected and maximum speed
Measured: Baseline, week 12, week 24
Secondary outcomes (18)
Change in the balance confidence
Measured with the modified Activities-specific Balance Confidence (ABC) Scale, score range 0-100, 0 indicates no confidence and 100 is full confidence
Measured: Baseline, week 12, week 24
Change in the number of falls
Number of self-reported falls in the last month
Measured: Baseline, week 12, week 24
Change in the lower limb coordination
Number of touched targets in a 30 second period with each foot, measured with the Lower Extremity MOtor COordination Test (LEMOCOT)
Measured: Baseline, week 12, week 24
Change in the number of sit-to-stand performed in 30 seconds
Number of complete sit-to-stand performed in 30 seconds without the help of arms
Measured: Baseline, week 12, week 24
Change in the time required to ascent 10 stairs
Time required to ascent 10 stairs, measured with the 10 Stairs Ascent
Measured: Baseline, week 12, week 24
Change in the time required to descent 10 stairs
Time required to descent 10 stairs, measured with the 10 Stairs Descent
Measured: Baseline, week 12, week 24
Change in the life space
Measured with the Life Space Assessment (LSA), score range 0-120, a higher score indicates higher level of mobility in the next five areas: outside the bedroom, outside the house, in the neighborhood, outside of the neighborhood but in town, and outside town during the past four weeks
Measured: Baseline, week 12, week 24
Change in the upper limb coordination
Number of finger touched targets in a 20 second period of each side, measured with the Standardized Finger-Nose Test (SFNT)
Measured: Baseline, week 12, week 24
Change in social participation
The performance in activities of daily living, measured with the Assessment of Life Habits (LIFE-H), score range 0-9
Measured: Baseline, week 12, week 24
Change in the Scale for the assessement and rating of ataxia (SARA)
Measured with the Scale for the assessment and rating of ataxia (SARA), score range 0-40, higher score indicates higher ataxia severity
Measured: Baseline, week 12, week 24
Measure of the usability of the program and hardware
Measured with the French System Usability Scale (F-SUS), original score range 0-40 converted to 0-100, a SUS score above a 68 would be considered above average and anything below 68 is below average
Measured: Week 24
Change in sleep components
The sleep components are measured with an actigraphy watch
Measured: Week 12, week 24
Change in mobility components
The changes in mobility components are measured with an actigraphy watch
Measured: Week 12, week 24
Change in the adductor hip muscle group spasticity
Change in the adductor hip muscle group spasticity, measured with the Modified Ashworth Scale
Measured: Baseline, week 12, week 24
Change in the knee extensor muscle group spasticity
Change in the knee extensor muscle group spasticity, measured with the Modified Ashworth Scale
Measured: Baseline, week 12, week 24
Change in the knee flexor muscle group spasticity
Change in the knee flexor muscle group spasticity, measured with the Modified Ashworth Scale
Measured: Baseline, week 12, week 24
Change in the plantar flexor muscle group spasticity
Change in the plantar flexor muscle group spasticity, measured with the Modified Ashworth Scale
Measured: Baseline, week 12, week 24
Sum of lower limb muscle spasticity
Sum of the spasticity results obtained for the adductor hip, knee extensor, knee flexor and plantar flexor muscle groups
Measured: Baseline, week 12, week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2023
- Study start1 Dec 2022
- Primary completion1 Dec 2024 (estimated)
- Study completion1 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Recessive Spastic Ataxia of Charlevoix-SaguenayStudies of the same condition, open and under way.
- More trials run by Université de SherbrookeThe 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
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Participants will participate in 2 phases : control phase (12-week usual care) followed by an intervention phase (12-week home-based rehabilitation program).
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Université de Sherbrooke
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Cynthia Gagnon, Université de Sherbrooke
- Investigators
- Elise Duchesne, Ph.D, Principal investigator, Université du Québec à Chicoutimi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05768750
- Sponsor's protocol ID
- 2021-047
- Acronym
- PACE-ARSCS
- Status last verified
- May 2023
- First submitted
- 3 Mar 2023
- Last updated
- 25 May 2023
- 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 25 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Home-based Rehabilitation Program to Counter the Motor Impairments and Activity Limitations Experienced by People With Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
- ClinicalTrials.gov ID
- NCT05768750
- Acronym / sponsor ID
- PACE-ARSCS / 2021-047
- Keywords
- ARSACS
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Université de SherbrookeUniversity, hospital or other
- Study start
- 1 Dec 2022 (actual)
- Primary completion
- 1 Dec 2024 (estimated)
- Study completion
- 1 Dec 2024 (estimated)
- First posted
- 14 Mar 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.
48 participants (24 women and 24 men) with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) will participate in 2 phases : control phase (12-week usual care) and intervention phase (12-week home-based rehabilitation program). The participants will be evaluated at baseline, week 12 (end of control phase) and week 24 (end of intervention phase) to quantify the effects of an individualized home-based rehabilitation program. Participants will also participate on a focus group at the end of the program to evaluate the acceptability of the program and the perceived changes.
Detailed description
Co-creation phase:
Preceding the intervention, a co-creation phase involving healthcare professionals (3), patient-partners (2), and ARSACS researchers (3) will be conducted to develop the exercises for the rehabilitation program and the evaluation scale to determine the level of difficulty of each exercise assigned to participants.
Intervention phase:
Random sampling stratified by gender and level of indoor mobility (unassisted walking, assisted walking, wheelchair) from the Neuromuscular Clinic's registry of 48 participants (24 women and 24 men) will be conducted.
- Control phase: All participants will be asked to maintain their usual activities for 12 weeks.
- Intervention phase: All participants recruited to the project will complete the home-based rehabilitation program assigned to them for 12 weeks, unsupervised, 20 minutes 3 times a week. Follow-up calls by a physiotherapist will be conducted at weeks 2, 4, 6, 8, 10 and 12.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Participants will participate in 2 phases : control phase (12-week usual care) followed by an intervention phase (12-week home-based rehabilitation program).
| Arm | Type | Receives |
|---|---|---|
| Control followed by intervention phase | Experimental | Other: RehabilitationAll participants will participate in 2 phases : control phase (12-week usual care) followed by an intervention phase (12-week home-based rehabilitation program). The rehabilitation program consists of three domains of exercises divided by levels of difficulty: sitting balance (16 levels), standing balance (21 levels) and sit-to-stand transfer (12 levels), including 67 exercises. Each individualized home-based rehabilitation program will include 3 to 6 exercises, for a total duration of 15 to 20 minutes. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- ARSACS diagnosis must be confirmed by genetic analysis;
- Women and men, aged between 18 and 50 years old;
- Be able to perform the sit-to-stand transfer;
- Consent of the neurologist must be given to participate in this study;
- Must reside in the Saguenay-Lac-St-Jean region;
Key exclusion criteria (5 listed)
- Patients who already meet physical activity (PA) recommendations (150 min of moderate to high intensity PA/week) or already participate in a rehabilitation program are excluded;
- Remain in a care facility;
- Do not speak English or French;
- Have another diagnosis causing physical limitations;
- Are pregnant.
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 |
|---|---|---|
| RehabilitationX | Other | Control followed by intervention phase |
Primary outcome
Change in sitting balance
Time frame: Baseline, week 12, week 24
Measured with the Ottawa sitting scale, score range 0-40, a higher score indicates a better outcome
Change in balance
Time frame: Baseline, week 12, week 24
Measured with the Berg Balance Scale, score range 0-56, a higher score indicates better balance
Change in walking speed
Time frame: Baseline, week 12, week 24
Change in the walking speed, measured with the time to walk 10 meters at self-selected and maximum speed
18 secondary outcomes
Change in the balance confidence
Time frame: Baseline, week 12, week 24
Change in the number of falls
Time frame: Baseline, week 12, week 24
Change in the lower limb coordination
Time frame: Baseline, week 12, week 24
Change in the number of sit-to-stand performed in 30 seconds
Time frame: Baseline, week 12, week 24
Change in the time required to ascent 10 stairs
Time frame: Baseline, week 12, week 24
Change in the time required to descent 10 stairs
Time frame: Baseline, week 12, week 24
Change in the life space
Time frame: Baseline, week 12, week 24
Change in the upper limb coordination
Time frame: Baseline, week 12, week 24
Change in social participation
Time frame: Baseline, week 12, week 24
Change in the Scale for the assessement and rating of ataxia (SARA)
Time frame: Baseline, week 12, week 24
Measure of the usability of the program and hardware
Time frame: Week 24
Change in sleep components
Time frame: Week 12, week 24
Change in mobility components
Time frame: Week 12, week 24
Change in the adductor hip muscle group spasticity
Time frame: Baseline, week 12, week 24
Change in the knee extensor muscle group spasticity
Time frame: Baseline, week 12, week 24
Change in the knee flexor muscle group spasticity
Time frame: Baseline, week 12, week 24
Change in the plantar flexor muscle group spasticity
Time frame: Baseline, week 12, week 24
Sum of lower limb muscle spasticity
Time frame: Baseline, week 12, week 24
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2023.
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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. This study is not recruiting at the moment.
Elise Duchesne, Ph.D principal investigator
Université du Québec à Chicoutimi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Rehabilitation in people with Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay, run by Université de Sherbrooke at 1 site in Canada.
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 6 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 25 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Elise Duchesne, Ph.D (418-545-5011 ext. 6148, elise1_duchesne@uqac.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in sitting balance (time frame: Baseline, week 12, week 24). The study lists 3 primary outcomes in all. 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 25 May 2023.