Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias

NCT06261424 | Recruiting

• 1 other identifier: 2024-1426
• Study Type: interventional
• Target: 84
• Locations: 1 country
• Sites: 5

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.

Conditions

Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay; Spastic Paraplegia 7

Outcome Measures

Primary Outcomes (1)

• Severity of ataxia

  The severity of ataxia assessed using the Scale for the assessment and rating of Ataxia (SARA) has been chosen as the main variable based on several considerations. 1) it is one of the most widely used scales among recessive cerebellar ataxias in intervention studies, 2) it includes lower limbs motor impairments-induced mobility limitations items, 3) is significantly correlated with key impairments variables such as standing balance and walking speed, 4) and is a candidate for outcome measurement in foreseeable clinical trials. It has eight categories with accumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia) It showed evidence of adequate content validity and excellent construct validity and internal consistency in ARSACS and we recently validated the French version.

  64 weeks

Secondary Outcomes (36)

• Medication change

  64 weeks

• Weight

  64 weeks

• height

  64 weeks

• Physical activities

  64 weeks

• Walking speed.

  64 weeks

Study Arms (2)

Control group

NO 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.

Intervention group

EXPERIMENTAL

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).

Procedure: IMPACT - rehabIlitation prograM for sPAstiC aTaxias

Interventions

IMPACT - rehabIlitation prograM for sPAstiC aTaxias PROCEDURE

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 session in an adapted pool with multiple levels of water) for a total of 36 sessions. Participants will be divided into 7 subgroups of 6 participants and each sub-group will be supervised by one physiotherapist (PT) and one physiotherapy technologist for safety considerations. It will focus on these three domains: 1) postural control; balance, trunk-limbs and multi-joints control and coordination and 3) functional mobility. Each domain contains a standardized list of exercises with different difficulty levels and specific times allocation. All exercises will be executed by the participants at each session at the appropriate difficulty level. The initial level of difficulty for each exercise will be determined by the PT based on individual results of the pre-intervention assessment and on individual performance, fatigue level and safety.

Intervention group

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Laval University: lead
• McGill University: collaborator
• Université de Sherbrooke: collaborator
• Université du Québec a Montréal: collaborator
• University of Calgary: collaborator
• Corporation de recherche et d'action sur les maladies héréditaires (CORAMH): collaborator
• Cégep de Jonquière: collaborator
• Muscular Dystrophy Canada: collaborator
• University of Alberta: collaborator
• Integrated University Health and Social Services Center of the Capitale-Nationale: collaborator
• Centre intégré universitaire de santé et de services sociaux du Saguenay-Lac-St-Jean: collaborator

Study Sites (5)

CIUSSS de la Capitale-Nationale, Hôpital de Baie-Saint-Paul

Baie-Saint-Paul, Quebec, G3Z 0K3, Canada

NOT YET RECRUITING

CIUSSS de la Capitale-Nationale, Hôpital de La Malbaie

La Malbaie, Quebec, G5A 1T1, Canada

NOT YET RECRUITING

CIUSSS du Centre-Sud-de-l'Île-de-Montréal, installation Centre de réadaptation Lucie-Bruneau

Montreal, Quebec, H2H 2N8, Canada

RECRUITING

CIUSSS de la Capitale-Nationale, installation IRDPQ

Québec, Quebec, G1M 2S8, Canada

RECRUITING

Clinique des maladies neuromusculaires du Centre intégré universitaire de santé et de services sociaux (CIUSSS) du Saguenay-Lac-Saint-Jean, installation Hôpital Jonquière

Saguenay, Quebec, G7X 7X2, Canada

RECRUITING

Related Publications (1)

• 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.

  PMID: 36576926 BACKGROUND

MeSH Terms

Conditions

Spastic ataxia Charlevoix-Saguenay type; Spastic Paraplegia Type 7

Study Officials

• Elise Duchesne, Ph D.

  Université du Québec à Chicoutimi

  STUDY DIRECTOR

Central Study Contacts

Elise Duchesne, Ph D.

CONTACT

418-590-3552; educhesn@uqac.ca

Andrée Hardy

CONTACT

418 541-1234; guichetunique.slsj@ssss.gouv.qc.ca

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: CARE PROVIDER
• Masking Details: All participants will be assessed at their respective neuromuscular clinic (Saguenay, Quebec, Montreal or Calgary) by physiotherapists trained according to standard operating procedures (SOP). These physiotherapists will not be involved in the supervision of the program and will be blinded.
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Full Professor

Model Details: A multicentre and controlled trial design.

Study Record Dates

• First Submitted: December 21, 2023
• First Posted: February 15, 2024
• Study Start: February 1, 2024
• Primary Completion: April 1, 2026
• Study Completion (Estimated): March 1, 2027
• Last Updated: February 18, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF, CSR
• Time Frame: The research data will be kept for as long as it is useful for the advancement of scientific knowledge. These data will be share of other researchers around the world.
• Access Criteria: All these research projects will first be evaluated and approved by the Research Ethics Committee : Comité de recherche du Centre intégré universitaire de santé et de services sociaux du Saguenay-Lac-Saint-Jean. Also, this commitee make the follow-up for these research projects. The principal investigator of this research will respect privacy and confidentiality regulations in Quebec and in Canada for any sharing data research with other authorized persons in any countries.

Locations

CA (5)