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Table 2 Visit schedule

From: 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)

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 ±3 days ±1 week ±1 week ±1 week ±1 week ±1 week
Informed consent X        
Inclusion/Exclusion criteriaa X        
Medical history X        
Pregnancy test X X X X X X X X
Demography X X       
Vital signsb, respiratory rate, resting pulse, height and weight X X X X X X X X
Blood pressureb 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 measuresc
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 evaluationd 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
  1. a might include genetic testing, if participants are not included in the EFACTS registry
  2. b after at least 15 min of resting
  3. 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
  4. d Hematology (total and differential blood count) and blood chemistry