Acipimox in Mitochondrial Myopathy (AIMM): study protocol for a randomised, double-blinded, placebo-controlled, adaptive design trial of the efficacy of acipimox in adult patients with mitochondrial myopathy

Table 3 Schedule of events

Trial procedure	Day −28 to day 0			Day 1 to day 84	Day 81 to day 90
	Screen	Baseline		Active treatment period	End of treatment visit (week 12)	End of trial follow-up (week 16)
	Screen	Part 1	Part 2	Week 0 to week 12
Visit Window date	Day −28 to −2^a	Day −28 to 0^c		+/7 days	Day 81–90^f	+28 days to +35 days
Informed consent	X
Demographics	X
Medical history	X
Medication review	X	X	X		X
Eligibility blood samples^b	X
Pregnancy test^d	X
Contraceptive counselling	X
Eligibility confirmation	X
Fatigue Impact Scale			X		X
Re-confirm Consent		X	X	X	X
Activity Monitor		X		Xⁱ
Physical/Vital signs	X				X
Urine sample			X		X
Blood samples^e			X		X
Cardiopulmonary exercise test (Optional)			X		X
Functional outcomes			X		X
NMDAS			X		X
Questionnaires			X		X
Needle Muscle biopsy			X		X
Randomisation^g			X
IMP and nIMP dispense			X
Adverse event reporting				X	X	X
IMP/nIMP Packaging returned					X
Telephone follow-up^h				X

^aAll screening assessments must be completed prior to baseline visit
^bEligibility screening bloods will be taken if routine blood results are not available from The Newcastle upon Tyne Hospitals NHS Foundation Trust within the previous 3-month period
^cAll baseline part 1 assessments must be completed prior to all baseline part 2 and randomisation
^dFemales of childbearing potential only
^eBlood samples: creatinine clearance (U&Es), full blood count (for platelets), liver enzymes (ALT, AST, Gamma GT)
^fAll part 1 assessments must be completed prior to all part 2
^gRandomisation will only take place once all assessments have been completed
^hFollow-up phone calls will take place at weeks 1, 2, 4, 8 and 10 (week 10 is only applicable for participants involved in activity monitoring)
ⁱAn activity monitor will be sent out at week 10 so participants can wear the monitor for the last 7 days prior to the end of treatment visit

ISSN: 1745-6215