SlowSCA: Slowing Down Disease Progression in Premanifest SCA: a Piloting Interventional Exergame Trial

Sponsor

University Hospital Tuebingen (Other)

Overall Status

Unknown status

CT.gov ID

NCT02867969

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

2.9

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a piloting study using continuous motor training provided via whole body-controlled video games (exergames) to establish proof-of-concept evidence that such training leads to motor and neural changes in pre-manifest subjects with spinocerebellar ataxias (SCA).

Condition or Disease	Intervention/Treatment	Phase
Spinocerebellar Ataxia	Other: Motor training	N/A

Detailed Description

In many neurodegenerative diseases, including spinocerebellar ataxias (SCA), large populations of neurons are already lost and compensatory resources exhausted at time of clinical diagnosis. This calls for early intervention strategies aiming to slow down disease progression already at the premanifest stage of the disease. Here we propose the world-first interventional study aiming to delay onset in a rare genetic neurodegenerative disease. Specifically, we propose a piloting study using continuous motor training provided via whole body-controlled video games (exergames) to establish proof-of-concept evidence that such training leads to motor and neural changes in pre-manifest SCA subjects. The subclinical effects will be unravelled within an intraindividual control study design by elaborated quantitative Video Management System (VICON®)-based movement analysis and structural and functional 3 Tesla (T) magnetic resonance imaging. This will provide unique insights in underlying motor and neural networks and compensatory strategies. If successful, this piloting trial will provide the basis for a rigorous international multi-center large-scale study in a larger SCA population. Moreover, it will stimulate complementary tandem projects on effects of motor training on neural functioning and molecular pathways in premanifest SCA mouse models.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

26 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Supportive Care

Official Title:

Slowing Down Disease Progression in Premanifest SCA: a Piloting Interventional Exergame Trial

Study Start Date :

Aug 1, 2016

Anticipated Primary Completion Date :

Feb 1, 2017

Anticipated Study Completion Date :

May 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
Other: Motor training The motor training comprise of demanding coordinative exercises based on commercially available developed by Microsoft Game Console (XBOX Kinect™) exergames that specifically target ataxia dysfunctions.	Other: Motor training The motor training will comprise of demanding coordinative exercises based on commercially available developed by Microsoft Game Console (XBOX Kinect™) exergames that specifically target ataxia dysfunctions. Other Names: Exergaming

Arm

Intervention/Treatment

Other: Motor training

The motor training comprise of demanding coordinative exercises based on commercially available developed by Microsoft Game Console (XBOX Kinect™) exergames that specifically target ataxia dysfunctions.

Other: Motor training

The motor training will comprise of demanding coordinative exercises based on commercially available developed by Microsoft Game Console (XBOX Kinect™) exergames that specifically target ataxia dysfunctions.

Other Names:

Exergaming

Outcome Measures

Primary Outcome Measures

Gait score [Day 43]
Decrease in a quantitative composite gait score consisting of body sway and gait variability in walking challenge conditions (mattress), assessed by VICON-based motion tracking

Secondary Outcome Measures

Brain networks captured [Day 43]
Changes in brain networks captured by specific neuroimaging focussing on brain grey matter volume (VBM), brain fibre tracking (DTI) and functional connectivity of the cerebellum with other brain regions (resting-state connectivity)
Quantitative movement parameters [Day 43]
Set of quantitative movement parameters for complex whole-body movements (gait, stand, fast sequences of goal-directed stepping movements) (VICON-based motion tracking).
Ataxia severity [Day 43]
Clinical ataxia severity according to the Scale for the Assessment and Rating of Ataxia (SARA)
Daily activity [Day 43]
Objectively measured level of daily activity in subjects' real-world settings (body-worn motions sensors; ActivePal®)
Brain-derived neurotrophic factor [Day 43]
Increase of serum brain-derived neurotrophic factor (BDNF)

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

adult persons (age 18-80 years) who (i) are a blood-related relative of an index subject with a known SCA 1,2,3 or 6 mutation and who (ii) have a score value of <8 points on the Scale for the Assessment and Rating of Ataxia (SARA)
full capacity to consent to study participation after extensive information (fully Informed Consent)

Exclusion Criteria:

Cognitive deficits which prevent full comprehension of the study requirements and/or training requirements
Comorbid diseases that place severe constraints on a continous training with whole-body controlled exergames (e.g. severe visual or hearing deficits; severe paresis; severe movement disorders other than ataxia)
Comorbid neurologic disease other than ataxia (e.g. prior stroke or brain trauma)
Pregnant or breast-feeding persons
Lack of capacity for full informed consent according to established psychiatric criteria for full informed consent

Magnetic Resonance Imaging (MRI) exclusion criteria:

cardiac pacemakers
artificial heart valves
metal prostheses
implanted magnetic metal parts (screws, plates and similar devices)
intrauterine devices (for instance, for contraceptive purposes)
shrapnel parts and other metal pieces
non-removable braces and metal dentures
non-removable acupuncture needles
insulin pumps and infusion ports
tattoos, eye shadow and other metal-containing make-up
The subject has decreased sensitivity to temperature and / or decreased tolerance towards elevated body temperatures or warming of body parts.