Table 2 Overview of trial schedule

Procedure

Screen Visit

Baseline

Visit 1

(Local site)

Baseline Visit 2 (KCH)

Week 4

Visit 1 (Local)

Week 4 Visit 2 (KCH)

Week 12

Visit 1 (Local)

Within 4 weeks of Screening visit

4 weeks ± 4 days from IMP Administration

12 weeks ± 14 days from IMP Administration

Patient information and informed consent

X

Inclusion / Exclusion criteria

X

Demographic data and medical history

X

Concomitant medications

X

X

X

X

X

X

Vital Signs

X

X

X

X

X

X

Weight

X

Height, weight, BMI, waist-to-hip ratio

X

X

X

IMP administration

X

Exercise prescription

X

Full blood count

X

X

X

Ferritin & TSAT

X

X

X

U + E, Creatinine, LFTs, CRP

X

X

X

eGFR, Phosphate

X

X

X

Soluble Transferrin Receptor and stored sera/plasma/whole blood (EDTA)

X

X

X

Exercise capacity (6MWD, VO₂ peak)

X

X

X

Muscle strength (isokinetic dynamometry)

X

X

X

Fatigue Severity Score (Chalder Fatigue Questionnaire)

X

X

X

Functional impairment (WSAS Questionnaire)

X

X

X

Quality of life (KDQOL-36)

X

X

X

Functional capacity (STS60)

X

X

X

MRI spectroscopy (subset of participants)

X

X

Procedure

Screen Visit

Baseline

Visit 1

(Local site)

Baseline Visit 2 (KCH)

Week 4

Visit 1 (Local)

Week 4 Visit 2 (KCH)

Week 12

Visit 1 (Local)

Within 4 weeks of Screening visit

4 weeks ± 4 days of IMP Administration

12 weeks ± 14 days of Baseline visit

Muscle metabolism via muscle biopsy (subset of participants)

X

X

Qualitative Interview

X

X (if no exercise)

X

Adverse Events

X

X

X

Whole blood DNA (EDTA) (optional)

X