Table 4. Drug trials in patients with mainly dominant ataxias.
Study | Ataxia | Daily dose | Study design | Study period | No. of patients | Outcome |
---|---|---|---|---|---|---|
Ristori et al. [69] | Cerebellar ataxias of different etiologies | 100 mg riluzole | Randomised, double-blind, placebo-controlled | 8 weeks | 40 | Significant drop in ICARS score (at least 5 points) especially static function, kinetic functions, dysarthria |
Zesiewicz et al. [70] | SCA3 | 1 mg varenicline twice daily | Randomized, double-blind, placebo-controlled, (crossover) | 4 weeks titration, 4 weeks 1 mg twice daily | 20 | Significant improvement of SARA scores for gait, stance, rapid alternating movements, timed walking test, no crossover phase because of drop-outs |
Strupp et al. [73] | Cerebellar ataxia of different etiology | 5 g/day acetyl-dl-leucine | Open label | 1 week | 13 (5 SAO A, 1 SCA2, 1 SCA1,2ADCA, 4AOA) | SARA improvement (16.1 vs. 12.8); improvement in SCAFI and EuroQo1-5d-3l |
Velazquez-Perez et al. [76] | SCA2 | 50 mg ZnSO(4) daily or placebo | Randomized, double-blind, placebo-controlled | 6 months | 36 | Significant increase Zn levels in the CSF, non-significant decrease in SARA subscores for gait, posture, stance and rapid alternating movements, reduction of lipid's oxidative damage, reduction of saccadic latency in the zinc-treated group |
Assadietal. [157] | FRDA, CA, OPCA | 30 mg buspirone HC1 twice daily | Double-blind, placebo controlled, crossover | 3 months | 20 (10 SCA, 6 CA, 4 FA) | No evidence of an effect of buspirone HC1 on ICARS |
Schulte et al. [158] | SCA3 | Combination of trimethoprim 160 mg+sulfamethoxazole 800 mg, twice daily for 2 weeks, followed by a combination of trimethoprim 80 mg+sulfamethoxazole, 400 mg, twice daily for 5.5 months | Randomised, double-blind, placebo-controlled, crossove (2 weeks washout) | 6 months r | 22 | No significant improvement in ataxia ranking scale, self-assessment score, static posturography, and results of motor performance testing after 2 weeks and 6 months. No significant effects on the visual system using the achromatic Vision Contrast Test System and the Farnsworth-Munsell 100-hue test for color discrimination |
Yabe et al. [74] | SCA6 | 250 or 500 mg acetazolamide daily | Open-label pilot study | 22 months | 9 | Significant improvement in the ataxia rating scale (ICARS) and body sway analysis by stabilometry until week 48, non-significant effect after 88 weeks |
Botez et al. [75] | FRDA, OPCA | 200 mg amantadine vs. placebo | Randomized, double-blind, placebo-controlled | 3–4 months | 30 | Efficacy evaluation via visual and auditory reaction time and movement time with right and left hand according to Hamsher and Benton real method. Measurement of homovanillinic acid levels in the CSF |
FRDA Friedreich's ataxia, CA cerebellar atrophy, OPCA olivopontocerebellar atrophy, SCA spinocerebellar ataxia (SCA1 spinocerebellar ataxia type 1, SCA2 spinocerebellar ataxia type 2 etc.), CSF cerebrospinal fluid, POLG mutation in the polymerase gamma gene, SAOA sporadic ataxia of unknown origin, ADCA autosomal dominant cerebellar ataxia, AOA ataxia with ocular apraxia, SCAFI SCA functional index (consisting of the PATA test, 8 m walking time, nine-hole PEG test), EQ-VAS visual analog scale of the EQ-5D