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Table 1 Trial outcome measurement schedule

From: Cognitive behaviour therapy plus aerobic exercise training to increase activity in patients with myotonic dystrophy type 1 (DM1) compared to usual care (OPTIMISTIC): study protocol for randomised controlled trial

   

Measurement point

Priority

outcome measure

Time to complete

Visit 1 Screening

Visit 2 Baseline a

Visit 3 (5 months)

Visit 4 (10 months)

Visit 5 (16 months)

1

Demographics

 

X

    
 

General medical history

 

X

X

   

1

Concomitant medications & therapies

 

X

X

X

X

X

 

Adverse events

  

X

X

X

X

1

Confirm genetically proven classical or adult DM1/specific DM1 history

 

X

X

   
 

Primary outcome measure

      

1

DM1-Activ

5 min

 

X

X

X

X

 

Secondary outcome measures

      
 

Activity

      

5

MDHI

20 min

 

X

X

X

X

2

6-minute walk test/BORG Scale

10 min

X

Xa

X

X

X

13

Actometer data

For 14-days after visit

X

Xa

X

X

X

 

Fatigue and sleepiness

      

3

Checklist individual strength (CIS) fatigue

5 min

X

Xa

X

X

X

11

Fatigue and daytime sleepiness scale (FDSS)

5 min

 

X

X

X

X

 

Quality of Life

      

6

Individualised neuromuscular quality of life (InQoL)

20 min

 

X

X

X

X

 

Mood

      

4

Beck depression inventory-fast screen (BDI-FS):

10 min

X

Xa

X

X

X

 

Cognitive Measures

      

8

Apathy evaluation scale

10 min

 

X

X

X

X

12

Stroop

5 min

 

X

X

X

X

 

Potential Effect Modifiers

      

9

MIRS

2 min

X

Xa

X

X

X

14

McGill pain questionnaire

10 min

 

X

X

X

X

15

Trail making

10 min

 

X

X

X

X

21

Adult social behaviour questionnaire (ASBQ)

10 min

 

X

X

X

X

 

Marital satisfaction (VAS)

  

X

X

X

X

16

CBT: self-efficacy scale for fatigue (SES-28);

4 min

 

X

X

X

 

17

CBT: fatigue catastrophising scale (FCS)

4 min

 

X

X

X

 

18

CBT: focusing on symptoms (IMmQ)

4 min

 

X

X

X

 

19

CBT: Illness acceptance scale

4 min

 

X

X

X

 

20

CBT: social support (SSL-D)/Social Support list interactions (SSL-I)/Social support list negative interactions (SSL-N)

4 min

 

X

X

X

 
 

Sickness impact profile (subscale sleep)

  

X

   
 

Biomarkers

      

7

Whole blood 10 ml sample x3 (10 ml for DNA; 10 ml for RNA and 10 ml serum).

5 min

 

X

 

X

X

22

Urine sample (10 ml or more) x1

5 min

 

X

 

X

X

 

Carers measures

      
 

Caregivers strain index (CSI)

  

X

X

X

X

 

ASBQ (Other)

10min

 

X

X

X

X

 

Marital satisfaction (VAS) (can be a partner).

  

X

X

X

X

 

Will not be performed if carer is a non-partner.

      
 

Apathy evaluation scale (AES)

  

X

X

X

X

  1. aVisit 1 and Visit 2 may be combined if appropriate. No duplication of tests/questionnaires performed