Effects of acetyl-DL-leucine on cerebellar ataxia (ALCAT trial): study protocol for a multicenter, multinational, randomized, double-blind, placebo-controlled, crossover phase III trial

Table 2 Schedule of enrolment, interventions, and assessments

	Enrollment		Treatment period 1				Treatment period 2			Close-out
	Before	Eligibility Screening Visit 1^c	Visit 2 (Baseline)	Visit 3 2 weeks after V2	Visit 4 6 weeks after V2	Wash-out 4 weeks	Visit 5	Visit 6 2 weeks after V5	Visit 7 6 weeks after V5	Follow-up Visit 8 4 weeks after V7
Timeline (days)		0/-28	0	14	42	70	70	84	112	140
Informed consent^a	X
Inclusion/exclusion criteria		X
Medical history, including demographics & medications		X
Documentation of physiotherapy/speech therapy		X	X^b	X	X		X	X	X	X
Neurological examination		X
Blood tests		X^d			X^d		X^d		X^d
Randomization			X
Dispensing of trial drug			X				X
Return of trial drug					X				X
Compliance check				X	X			X	X
Patient questionnaires (EQ-5D-5 L, BDI-II, FSS)			X	X	X		X	X	X	X
Ataxia rating scale: SARA		X	X^b	X	X		X	X	X	X
Ataxia rating scale: SCAFI			X	X	X		X	X	X	X
Documentation of (S)AEs		X	X^b	X	X		X	X	X	X
Documentation of concomitant medication (drug history)			X	X	X		X	X	X	X

A delay of -3 and +5 days is acceptable for visits 2 and 5, for all other visits a delay of ± 5 days is acceptable
^aprior to first study-specific intervention and allocation
^bIf visit 1 and visit 2 take place at the same time, the ataxia rating scale (SARA), documentation of physiotherapy/speech therapy and documentation of (S)AE are assessed only once
^cIf patients are on medication due to cerebellar symptoms at visit 1, a 4-week wash-out (screening period) prior to randomization at visit 2 is required (adherence will again be checked before randomization at V2). Otherwise, visit 1 and visit 2 coincide
^dincl. negative pregnancy test for women of childbearing potential

ISSN: 1471-2377