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 |