Clinical trial NCT06261424: Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias
Official title: IMPACT, a Supervised Rehabilitation Program for Spastic Ataxias: A Rater-blinded, Randomized Controlled Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
Spastic Paraplegia 7
Lead sponsor: Université du Québec à Chicoutimi
Sponsor class: OTHER
Collaborators: name: McGill University
name: Université de Sherbrooke
name: Université du Québec a Montréal
name: Laval University
name: University of Calgary
name: Corporation de recherche et d'action sur les maladies héréditaires (CORAMH)
name: Cégep de Jonquière
name: Centre intégré universitaire de santé et de services sociaux du Saguenay-Lac-St-Jean
name: Muscular Dystrophy Canada
name: University of Alberta
name: Integrated University Health and Social Services Center of the Capitale-Nationale
First posted: 2024-02-15
Enrollment: 84
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: A multicentre and controlled trial design.
Primary purpose: SUPPORTIVE_CARE

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* have a confirmed genetic diagnosis of ARSACS or SPG7
* be able to maintain standing position and to transfert
* be authorized by their treating neurologist
* speak French or English
* be able to give informed consent.

Exclusion Criteria:

* have active participation in a rehabilitation program (self-reported information)
* have another condition causing physical limitations
* be uncomfortable in a swimming pool
* be pregnant.

Primary outcomes:
measure: Severity of ataxia
timeFrame: 64 weeks
Secondary outcomes:
measure: Medication change
timeFrame: 64 weeks
measure: Weight
timeFrame: 64 weeks
measure: height
timeFrame: 64 weeks
measure: Physical activities
timeFrame: 64 weeks
measure: Walking speed.
timeFrame: 64 weeks
measure: Standing balance
timeFrame: 64 weeks
measure: Balance confidence
timeFrame: 64 weeks
measure: Sitting balance
timeFrame: 64 weeks
measure: Activities of Daily Living
timeFrame: 64 weeks
measure: Muscle tone
timeFrame: 64 weeks
measure: Life habits
timeFrame: 64 weeks
measure: Community mobility
timeFrame: 7 consecutives days, 2 times
measure: Plasma
timeFrame: 64 weeks
measure: DNA
timeFrame: 64 weeks
measure: Serum
timeFrame: 64 weeks
measure: RNA
timeFrame: 64 weeks
measure: Urine
timeFrame: 64 weeks
measure: Saliva
timeFrame: 64 weeks
measure: Mobility Life-space
timeFrame: 64 weeks
measure: Ataxia impact scale
timeFrame: 64 weeks
measure: Lower limb coordination
timeFrame: 64 weeks
measure: Peak respiratory flow and cough
timeFrame: 64 weeks
measure: Fatigue
timeFrame: 64 weeks
measure: Falls.
timeFrame: 64 weeks
measure: Patient-reported impression of change.
timeFrame: 64 weeks
measure: Anxiety and Depression
timeFrame: 64 weeks
measure: Pain characteristics
timeFrame: 64 weeks
measure: Functional lower extremity strength
timeFrame: 64 weeks
measure: Focus group with intervention group participants
timeFrame: 1 day
measure: Focus groups with stakeholders
timeFrame: 12 weeks
measure: Lower limb muscle activation
timeFrame: 64 weeks
measure: Quality of life
timeFrame: 64 weeks
measure: CBA analyses and Willingness to pay
timeFrame: 64 weeks
measure: Gait's temporal parameters
timeFrame: 64 weeks
measure: Stance balance
timeFrame: 64 weeks
measure: Postural balance
timeFrame: 64 weeks
Other outcomes:
Locations:
city: Baie-Saint-Paul
country: Canada
facility: CIUSSS de la Capitale-Nationale, Hôpital de Baie-Saint-Paul
state: Quebec
city: La Malbaie
country: Canada
facility: CIUSSS de la Capitale-Nationale, Hôpital de La Malbaie
state: Quebec
city: Montréal
country: Canada
facility: CIUSSS du Centre-Sud-de-l'Île-de-Montréal, installation Centre de réadaptation Lucie-Bruneau
state: Quebec
city: Québec
country: Canada
facility: CIUSSS de la Capitale-Nationale, installation IRDPQ
state: Quebec
city: Saguenay
country: Canada
facility: Clinique des maladies neuromusculaires du Centre intégré universitaire de santé et de services sociaux (CIUSSS) du Saguenay-Lac-Saint-Jean, installation Hôpital Jonquière
state: Quebec
Study officials:
affiliation: Université du Québec à Chicoutimi
name: Elise Duchesne, Ph D.
role: STUDY_DIRECTOR
References:
citation: Lessard I, Masterman V, Cote I, Gagnon C, Duchesne E. A rehabilitation program to increase balance and mobility in ataxia of Charlevoix-Saguenay: An exploratory study. PLoS One. 2022 Dec 28;17(12):e0279406. doi: 10.1371/journal.pone.0279406. eCollection 2022.