Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia

NCT06692296 | Recruiting

• 2 other identifiers: 1129CET ANV 2024-97
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with Friedreich's Ataxia. The secondary objective is to assess the retention of the rehabilitation treatment effects over time. Enrolled patients will be randomized and assigned to one of two treatment groups for four weeks. Three assessments will be conducted for each patient: one before treatment (T0), one at the end of treatment (T1), and a follow-up assessment 90 days after T1 via telemedicine (T2). This study is:

• Exploratory: The study aims to explore the usefulness of the stabilometric platform in a rehabilitative setting in combination with conventional therapy;
• Not "first-in-human";
• Longitudinal: The study will investigate changes over time in motor and functional ability scales, as well as stabilometric platform parameters, within the same cohort;
• Randomized, controlled, open-label;
• Monocentric: The study will be conducted only at the "La Nostra Famiglia" Association in Conegliano and Pieve di Soligo - IRCCS Eugenio Medea in Conegliano - UOC Neuromotor Rehabilitation in Pieve di Soligo, Via Monte Grappa, 96, 31053 Pieve di Soligo TV;
• Prospective;
• Post-market.

Conditions

Friedreich Ataxia

Keywords

randomized controlled trial; Friedreich Ataxia; balance; rehabilitation; training; stabilometric platform; ataxia; therapeutic exercise; standing balance; Scale for the assessment and rating of ataxia; six minute walking test; functional reach test; timed up and go; Friedreich ataxia rating scale

Outcome Measures

Primary Outcomes (1)

• Score on the subscale 'Section E. Upright stability' of the modified Friedreich Ataxia Rating Scale.

  The outcome evaluates balance performance while sitting, standing, and walking. Lower scores indicate better balance performance, higher scores indicate more severe balance impairment.

  Enrolment (T0), end of treatment at four weeks (T1), follow-up at 12 weeks after T1 (T2).

Secondary Outcomes (7)

• Modified Friedreich's Ataxia Rating Scale (mFARS)

  Enrolment (T0), end of treatment at four weeks (T1), follow-up at 12 weeks after T1 (T2).

• Scale for Assessment and Rating for Ataxia (SARA).

  Enrolment (T0), end of treatment at four weeks (T1), follow-up at 12 weeks after T1 (T2)..

• Six minutes walking test (6MWT).

  Enrolment (T0), end of treatment at four weeks (T1).

• Timed Up and Go test (TUG).

  Enrolment (T0), end of treatment at four weeks (T1).

• Berg balance scale (BBS).

  Enrolment (T0), end of treatment at four weeks (T1).

Study Arms (2)

Platform Group

EXPERIMENTAL

Balance training including both conventional rehabilitation (24 sessions) and training over a stabilometric platform (20 sessions).

Behavioral: Balance training over stabilometric platform; Behavioral: Balance training by means of conventional rehabilitation

Conventional Group

ACTIVE COMPARATOR

Balance training including only conventional rehabilitation (44 sessions).

Behavioral: Balance training by means of conventional rehabilitation

Interventions

Balance training over stabilometric platform BEHAVIORAL

The treatment using the stabilometric platform (a total of 20 sessions) generally includes an initial phase (approximately 5 minutes) of training and warm-up in "monitor" mode, during which the patient becomes familiar with the equipment. The patient then moves on to "training library" mode, which includes the following subcategories: (1) Stabilometric tracings in both static and dynamic phases (10 minutes); (2) Dexterity exercises in both static and dynamic phases (10 minutes); (3) Imbalance exercises in both static and dynamic phases (10 minutes). The goal during this phase is to increase the patient's stability limits and provoke balance reactions through load shifts in all directions. The session concludes with 10 minutes of TecnoBody Exergames, which help to automate the functions learned within a more complex activity.

Also known as: PROKIN - PK252

Platform Group

Balance training by means of conventional rehabilitation BEHAVIORAL

Conventional rehabilitation to improve balance includes an initial phase of tissue mobilization, particularly of the foot structures (approximately 5 minutes per foot), in order to prepare them for the subsequent activation phase. This is followed by a phase focused on activating the anti-gravity muscles and aligning the trunk within the chosen setting (10 minutes). The treatment continues with balance training, both static and dynamic, load transfers, and dual-task exercises using external references (15 minutes). The session concludes with functional exercises relevant to the patient's daily life context (10 minutes).

Also known as: balance training

Conventional Group; Platform Group

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 14 years;
• Weight \< 150 kg;
• Genetic diagnosis of Friedreich's Ataxia;
• Subscores of SARA (Scale for the Assessment and Rating of Ataxia) as follows: (1) from 1 to 6 for the item 'Gait (walking ability)'; (2) from 1 to 3 for the item 'Stance (ability to maintain a stable posture)'; (3) ability to walk ≥10 metres with or without walking aids; (4) ability to stand unsupported; Stadiation of the Friedreich Ataxia Rating Scale between 2 and 4.

You may not qualify if:

• Age \< 14 years;
• Diagnosis of acquired ataxia;
• Subscores for items on the SARA scale of: (1) 0 or 7 for gait; (2) 0 or 4-6 for stance;
• Inability to walk;
• Ability to walk \< 10 meters;
• Standing position possible only with support;
• Other associated neurological conditions;
• Current major psychiatric disorder (psychosis and major depression).

Sponsors & Collaborators

• IRCCS Eugenio Medea: lead

Study Sites (1)

Scientific Institute, IRCCS E. Medea, Department of Pieve di Soligo, Treviso, Italy

Pieve di Soligo, Veneto, 31053, Italy

RECRUITING

Related Publications (12)

• Zesiewicz TA, Stephenson JB, Kim SH, Sullivan KL, Jahan I, Huang Y, Salemi JL, Wecker L, Shaw JD, Gooch CL. Longitudinal gait and balance decline in Friedreich's Ataxia: A pilot study. Gait Posture. 2017 Jun;55:25-30. doi: 10.1016/j.gaitpost.2017.03.019. Epub 2017 Mar 30.

  PMID: 28411441 BACKGROUND

• Prosperini L, Pozzilli C. The clinical relevance of force platform measures in multiple sclerosis: a review. Mult Scler Int. 2013;2013:756564. doi: 10.1155/2013/756564. Epub 2013 May 19.

  PMID: 23766910 BACKGROUND

• Podsiadlo D, Richardson S. The timed "Up & Go": a test of basic functional mobility for frail elderly persons. J Am Geriatr Soc. 1991 Feb;39(2):142-8. doi: 10.1111/j.1532-5415.1991.tb01616.x.

  PMID: 1991946 BACKGROUND

• Milne SC, Kim SH, Murphy A, Larkindale J, Farmer J, Malapira R, Danoudis M, Shaw J, Ramakrishnan T, Rasouli F, Yiu EM, Georgiou-Karistianis N, Tai G, Zesiewicz T, Delatycki MB, Corben LA. The Responsiveness of Gait and Balance Outcomes to Disease Progression in Friedreich Ataxia. Cerebellum. 2022 Dec;21(6):963-975. doi: 10.1007/s12311-021-01348-2. Epub 2021 Dec 2.

  PMID: 34855135 BACKGROUND

• Milne SC, Corben LA, Georgiou-Karistianis N, Delatycki MB, Yiu EM. Rehabilitation for Individuals With Genetic Degenerative Ataxia: A Systematic Review. Neurorehabil Neural Repair. 2017 Jul;31(7):609-622. doi: 10.1177/1545968317712469. Epub 2017 Jun 9.

  PMID: 28595509 BACKGROUND

• Januario F, Campos I, Amaral C. Rehabilitation of postural stability in ataxic/hemiplegic patients after stroke. Disabil Rehabil. 2010;32(21):1775-9. doi: 10.3109/09638281003734433.

  PMID: 20350209 BACKGROUND

• Ilg W, Milne S, Schmitz-Hubsch T, Alcock L, Beichert L, Bertini E, Mohamed Ibrahim N, Dawes H, Gomez CM, Hanagasi H, Kinnunen KM, Minnerop M, Nemeth AH, Newman J, Ng YS, Rentz C, Samanci B, Shah VV, Summa S, Vasco G, McNames J, Horak FB; Ataxia Global Initiative (AGI) working group Digital Motor Biomarkers. Quantitative Gait and Balance Outcomes for Ataxia Trials: Consensus Recommendations by the Ataxia Global Initiative Working Group on Digital-Motor Biomarkers. Cerebellum. 2024 Aug;23(4):1566-1592. doi: 10.1007/s12311-023-01625-2. Epub 2023 Nov 13.

  PMID: 37955812 BACKGROUND

• Hartley H, Cassidy E, Bunn L, Kumar R, Pizer B, Lane S, Carter B. Exercise and Physical Therapy Interventions for Children with Ataxia: A Systematic Review. Cerebellum. 2019 Oct;18(5):951-968. doi: 10.1007/s12311-019-01063-z.

  PMID: 31392562 BACKGROUND

• Cattaneo D, Jonsdottir J, Regola A, Carabalona R. Stabilometric assessment of context dependent balance recovery in persons with multiple sclerosis: a randomized controlled study. J Neuroeng Rehabil. 2014 Jun 10;11:100. doi: 10.1186/1743-0003-11-100.

  PMID: 24912561 BACKGROUND

• Burciu RG, Fritsche N, Granert O, Schmitz L, Sponemann N, Konczak J, Theysohn N, Gerwig M, van Eimeren T, Timmann D. Brain changes associated with postural training in patients with cerebellar degeneration: a voxel-based morphometry study. J Neurosci. 2013 Mar 6;33(10):4594-604. doi: 10.1523/JNEUROSCI.3381-12.2013.

  PMID: 23467375 BACKGROUND

• Bidichandani SI, Delatycki MB. Friedreich Ataxia. In: Adam MP, Pagon RA, Bird TD, Dolan CR, Stephens K, eds. GeneReviews™. Seattle: University of Washington; 2017.

  BACKGROUND

• Berg K, Wood-Dauphinee S, Williams JI. The Balance Scale: reliability assessment with elderly residents and patients with an acute stroke. Scand J Rehabil Med. 1995 Mar;27(1):27-36.

  PMID: 7792547 BACKGROUND

MeSH Terms

Conditions

Friedreich Ataxia; Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Gabriella Paparella, Medical Degree

  Scientific Institute, IRCCS E. Medea, Department of Conegliano, Treviso, Italy.

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gabriella Paparella, Medical Degree

CONTACT

00390438906330; gabriella.paparella@lanostrafamiglia.it

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: A monocentric, open-label randomized controlled trial.

Study Record Dates

• First Submitted: November 14, 2024
• First Posted: November 18, 2024
• Study Start: February 3, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: May 2, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Start date: January 2028; End date: January 2038.
• Access Criteria: Any researcher accredited by non-profit clinical and research centre.

Locations

IT (1)