Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA)

Table 2 Visit schedule

Study Period	Screening	Phase A 1 month	Phase B 23 months
Visit No.		1	2	3	4	5	6	7
Activity		Baseline	Randomization 1 month	4 month	8 month	12 month	18 month	EOS 24 month
Visit window +/− calendar days		Baseline	±3 days	±1 week	±1 week	±1 week	±1 week	±1 week
Informed consent	X
Inclusion/Exclusion criteria^a	X
Medical history	X
Pregnancy test	X	X	X	X	X	X	X	X
Demography	X	X
Vital signs^b, respiratory rate, resting pulse, height and weight	X	X	X	X	X	X	X	X
Blood pressure^b	X	X	X	X	X	X	X	X
Body temperature	X	X	X	X	X	X	X	X
Physical examination	X	X	X	X	X	X	X	X
Electrocardiogram (ECG)		X	X	X	X	X	X	X
Echocardiogram (ECHO)		X				X		X
Quest. about exercise (QPE)		X	X	X	X	X	X	X
Clinical measures^c SCAFI, CCFS, mFARS, ADL, EQ. 5D, MoCA, CGI-C, PGI-C		X	X	X	X	X	X	X
Scale for the Assessment and Rating of Ataxia (SARA)	X	X	X	X	X	X	X	X
Standard laboratory evaluation^d	X	X	X	X	X	X	X	X
Up-regulation of Frataxin (FXN) protein level		X	X			X		X
Gene expression of Frataxin (FXN) gene	X	X				X		X
RNA-Sequencing of Frataxin gene		X				X		X
PK sampling (nicotinamide level)	X	X	X	x		X		X
MRI (brain and spine)		X				X		X
Concomitant medication	X	X	X	X	X	X	X	X
Adverse events evaluation		X	X	X	X	X	X	X

^a might include genetic testing, if participants are not included in the EFACTS registry
^b after at least 15 min of resting
^c ADL Activity of daily living, CCFS Composite cerebellar functional severity, CGI-C Clinician’s global impression-change scale, EQ. 5D EuroQol five dimensions questionnaire, INAS Inventory of non-ataxia signs, MoCA Montreal cognitive assessment, PGI-C Patient’s global impression-change scale, SCAFI Spinocerebellar ataxia functional index, EOS End of Study
^d Hematology (total and differential blood count) and blood chemistry

ISSN: 2524-3489