L-arginine for Spinocerebellar Ataxia Type 6
Author Information
Author(s): Ishihara Tomohiko, Tada Masayoshi, Kanemitsu Yoshitomi, Takahashi Yuji, Ishikawa Kinya, Ikenaka Kensuke, Hirano Makito, Yokota Takanori, Minakawa Eiko N., Saito Katsuhisa, Nagai Yoshitaka, Onodera Osamu
Primary Institution: Brain Research Institute, Niigata University, Niigata, Japan
Hypothesis
Does L-arginine improve symptoms in patients with spinocerebellar ataxia type 6?
Conclusion
L-arginine treatment showed a tendency to improve symptoms in patients with spinocerebellar ataxia type 6, but the results were not statistically significant.
Supporting Evidence
- The mean medication adherence rate was 97.2% in the L-arginine group.
- Two serious adverse reactions occurred in the L-arginine group, including one case of pneumonia (severe, death) and one case of abnormal liver function (moderate, recovery).
- The difference in SARA total score between the L-arginine and placebo groups was −1.52.
Takeaway
This study tested a medicine called L-arginine to see if it helps people with a certain brain problem called spinocerebellar ataxia type 6. It seemed to help a little, but not enough to be sure.
Methodology
A multicenter, randomized, double-blind, placebo-controlled phase 2 trial was conducted with 40 genetically confirmed SCA6 patients over 48 weeks.
Potential Biases
Potential bias due to the high placebo effect observed in neurodegenerative disease trials.
Limitations
The study had a small sample size and did not include patients with mild symptoms, which may limit the generalizability of the results.
Participant Demographics
Participants were aged 20 years or older, with a mean age of 66.3 years in the L-arginine group and 63.6 years in the placebo group.
Statistical Information
P-Value
0.0582
Confidence Interval
−3.10 to 0.06
Statistical Significance
p=0.0582
Digital Object Identifier (DOI)
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