Table 2

Outcome measures and psychometrics properties

OutcomeMeasureDescriptionPsychometric properties
Motor functionm-FIM37
  • The m-FIM evaluates a person’s ability to perform motor activities of daily living.56 Items include performance in self-care, sphincter control and mobility.57

  • 13 items, each assessed against a 7-point ordinal scale.

  • Maximum score of 91 (complete independence) and a minimum of 13 (complete dependence).

  • High validity and inter-rater reliability36 58.

  • More responsive to change after rehabilitation than the total FIM score for individuals with FRDA.14

  • Exhibited strong correlations with level of disability in neurological populations and can predict amount of help required.36 59–61

Ataxia symptomsSARA39
  • The SARA is a semiquantitative clinical assessment of ataxia, measuring ataxia of upper limb, lower limb, gait, balance and speech.

  • Eight items; score range 0–40, with a higher score indicating more severe ataxia.39

  • Excellent inter-rater and test–retest reliability in individuals with ataxia.39

  • Excellent construct validity in ataxias of multiple aetiologies62 63.

BalanceBBS40
  • The BBS evaluates performance in sitting and standing balance activities.

  • 14 items; score ranging 0–56 with a higher score indicating better balance.

  • Responsive to change after intensive coordinative training in degenerative ataxias.17

  • Good intra- and inter-rater reliability when assessing balance in people with ataxia secondary to multiple sclerosis.64

Participant perceived benefitPGIC41
  • The PGIC is 7-point numerical rating scale measuring global benefit from the participant’s perspective.

  • Maximum score of 7 (a great deal better, and a considerable improvement that has made all the difference) and a minimum of 0 (no change).

  • Cut-off for clinically meaningful change will be 5 (moderately better, and a slight but noticeable change).

  • High face validity.65

  • Responsive to change following a 6 week rehabilitation programme in individuals with FRDA.14

  • Used as an external criterion for determining smallest detectible and clinically meaningful change after rehabilitation and 1 year of natural decline in individuals with multiple sclerosis and spinocerebellar ataxia respectively66 67.

Quality of lifeSF-36 v242
  • The SF-36 v2 measures self-perceived health-related quality of life.

  • 36 items; yields scores for eight multiitem dimensions and two summary scale scores (physical and mental health).42

  • Responsive to reduction in quality of life in individuals with ataxia68 69.

  • The physical component of the SF-36 v2 has been shown to be highly correlated with disease duration and ataxia severity in individuals with FRDA.68

  • The Sf-36 v1 has shown acceptable internal consistency among subscales in individuals with FRDA.70

Daily walking activity
  1. Average daily step count.

  2. Average daily distance walked.

  • Measured with the Fitbit Flex 2, a commercial grade tri-axial accelerometer worn on the wrist.

  • Worn for 24 hours per day for seven consecutive days.

  • A valid day=Fitbit Flex 2 worn for ≥90% of the day. Wear time will be recorded by participant self-report.

  • 3–5 days of accelerometer monitoring in adults is necessary to achieve a between day intra-class correlation of 0.80.71

  • Moderate validity for measuring physical activity relative to the Actigraph.72 Good to excellent significant positive correlations and agreement with the Actigraph, although it overestimates number of steps.73

  • Excellent reliability in an older population.74

Sitting balanceFIST43
  • The FIST is a clinical measure of sitting balance.43

  • 14 items; score ranging 0–56 with a higher score indicating better sitting balance.43

  • Excellent concurrent validity with the BBS and moderate to good validity with the m-FIM in adults with neurological deficits and impaired sitting balance.75

  • Excellent test–retest reliability in individuals with various neurological disorders76 77.

  • Responsive to change following rehabilitation and a minimal detectible change of 5.5 points.75

Postural control3D movement of the trunk in sitting and standing with eyes open and closed.
  • Measured with the BioKin system, a wireless motion capture device.44

  • Four test conditions include: sitting 30 s, no foot contact on the floor, arms out straight: (1) eyes open and (2) eyes closed; standing 30 s, feet together: (3) eyes open and (4) eyes closed.

  • An exploratory outcome used in this trial, not previously validated in this population.

  • BBS, Berg Balance Scale; 3D, three-dimensional; FIST, Function in Sitting Test; FRDA, Friedreich ataxia; m-FIM, motor domain of the functional independence measure; PGIC, Patient Global Impression of Change; SARA, Scale for the Assessment and Rating of Ataxia; SF-36 v2, Medical Outcomes Study 36 item Short-Form Health Survey V.2.