1Department of Neurology, Samsung Seoul Hospital, Sungkyunkwan University School of Medicine, Seoul, Korea
2Department of Neurology, SMG-SNU Boramae Medical Center, Seoul, Korea
3Department of Neurology, Seoul National University College of Medicine, Seoul, Korea
4Department of Neurology, College of Medicine, The Catholic University of Korea, Seoul, Korea
5Department of Neurology, Korea University Anam Hospital, Korea University College of Medicine, Seoul, Korea
6Department of Neurology, Chonnam National University Hospital, Chonnam National University Medical School, Gwangju, Korea
7Department of Neurology, Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, Korea
8Department of Neurology, Korea University Guro Hospital, Korea University College of Medicine, Seoul, Korea
Copyright © 2025 The Korean Movement Disorder Society
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Conflicts of Interest
The authors have no financial conflicts of interest.
Funding Statement
This study was funded by HLB Pharmaceutical Co., LTD. (Seoul, Korea) (HLB-004).
Author contributions
Conceptualization: all authors. Data curation: all authors. Formal analysis: all authors. Funding acquisition: all authors. Investigation: all authors. Methodology: all authors. Project administration: all authors. Resources: all authors. Software: all authors. Supervision: all authors. Validation: all authors. Visualization: all authors. Writing—original draft: all authors. Writing—review & editing: Seong-Beom Koh.
Variables |
Values |
p value | |
---|---|---|---|
Taltirelin group (n=75) | Control group (n=74) | ||
Age (yr) | 56.60±10.55 | 52.51±12.17 | 0.0265* |
Sex | 0.9385 | ||
Men | 40 (53.33) | 39 (52.70) | |
Women | 35 (46.67) | 35 (47.30) | |
Genetic inheritance of ataxia | 0.6927 | ||
Hereditary ataxia | 41 (54.67) | 45 (60.81) | |
SCA | 37 (49.33) | 42 (56.76) | |
Other types of hereditary ataxia | 4 (5.33) | 3 (4.05) | |
Spastic ataxia of Charlevoix-Saguenay | 0 (0.00) | 1 (1.35) | |
Hereditary spastic ataxia | 1 (1.33) | 0 (0.00) | |
DRPLA gene | 1 (1.33) | 0 (0.00) | |
Chromosome analysis | 1 (1.33) | 0 (0.00) | |
Unknown | 1 (1.33) | 2 (2.70) | |
Non-hereditary ataxia | 34 (45.33) | 29 (39.19) | |
ILOCA | 34 (45.33) | 29 (39.19) | |
Disease duration (yr) | 2.33 (0.07–16.47) | 3.23 (0.00–22.50) | 0.4165 |
K-MMSE scores | 28.03±1.69 | 27.93±2.07 | 0.7894 |
Outcome measures | Taltirelin group (n=75) | Control group (n=74) | p value |
---|---|---|---|
Primary efficacy outcome measure | |||
Changes in K-SARA scores at 24 weeks from baseline | -0.51±2.79 | 0.36±2.62 | 0.0321* |
Secondary efficacy outcome measures | |||
Changes in K-SARA scores at 12 weeks from baseline | -0.47±3.05 | 0.05±2.66 | 0.1151 |
Changes in CGI-S scores at 24 weeks from baseline | 0.08±0.56 | 0.03±0.47 | 0.4925 |
Changes in CGI-I scores at 24 weeks from 4 weeks | 0.15±0.65 | 0.31±0.57 | 0.1170 |
Changes in CGI-E scores at 24 weeks from 4 weeks | -0.08±0.65 | -0.16±0.57 | 0.3440 |
Changes in K-SCOPA-AUT scores at 24 weeks from baseline | -2.27±6.09 | -0.45±5.34 | 0.2349 |
Changes in EQ-5D-5L scores at 24 weeks from baseline | |||
EQ-5D-5L descriptive system | 0.00±0.16 | -0.02±0.13 | 0.3159 |
EQ VAS | 1.92±21.72 | -3.88±18.32 | 0.1539 |
Changes in Tinetti balance and gait scores at 24 weeks from baseline | 0.03±4.02 | -0.34±3.25 | 0.5874 |
Spatio-temporal parameters of gait | |||
Spatial parameters | |||
Changes in step length at 24 weeks from baseline (cm) | |||
Left | -1.25±8.93 | -0.95±7.22 | >0.9999 |
Right | -1.52±8.09 | -1.00±6.00 | 0.9320 |
Changes in CoV of step length at 24 weeks from baseline (%) | |||
Left | 0.51±7.43 | 3.44±13.29 | 0.2066 |
Right | 1.86±14.76 | -3.07±11.17 | 0.5682 |
Changes in stride length at 24 weeks from baseline (cm) | |||
Left | -2.96±17.19 | -1.65±12.23 | 0.9008 |
Right | -2.89±15.96 | -2.37±12.11 | 0.8286 |
Changes in CoV of stride length at 24 weeks from baseline (%) | |||
Left | 3.51±16.33 | -0.68±5.78 | 0.6273 |
Right | 1.29±9.67 | -0.19±5.30 | 0.4704 |
Changes in base of support at 24 weeks from baseline (cm) | |||
Left | 0.34±4.41 | 0.29±3.87 | 0.8388 |
Right | 0.41±4.40 | 0.43±3.65 | 0.9895 |
Changes in CoV of base of support at 24 weeks from baseline (%) | |||
Left | -4.72±15.87 | -2.12±12.16 | 0.9112 |
Right | -3.44±13.87 | -2.50±10.98 | 0.7930 |
Temporal parameters | |||
Changes in walking velocity at 24 weeks from baseline (cm/s) | -1.14±14.74 | -3.15±13.28 | 0.5651 |
Changes in cadence at 24 weeks from baseline (step/min) | 1.49±11.81 | -4.41±16.06 | 0.2761 |
Changes in proportion in gait cycle of swing time at 24 weeks from baseline (%GC) | |||
Left | -1.37±7.11 | -1.48±7.34 | 0.2077 |
Right | -0.62±6.27 | -1.42±5.07 | 0.3792 |
Changes in proportion in gait cycle of stance time at 24 weeks from baseline (%GC) | |||
Left | 1.36±7.10 | 3.00±6.20 | 0.1023 |
Right | -0.06±7.48 | 1.41±5.05 | 0.2997 |
Changes in proportion in gait cycle of single support time at 24 weeks from baseline (%GC) | |||
Left | -0.89±6.53 | -1.41±5.03 | 0.4584 |
Right | -1.36±7.16 | -1.69±7.43 | 0.1380 |
Changes in proportion in gait cycle of double support time at 24 weeks from baseline (%GC) | |||
Left | -0.36±10.25 | 5.77±14.20 | 0.1346 |
Right | 0.69±7.91 | 4.02±8.93 | 0.1850 |
Values are presented as mean±standard deviation.
* p value<0.05.
K-SARA, Korean version of Scale for the Assessment and Rating of Ataxia; CGI-S, Clinical Global Impression Scale-Severity; CGI-I, Clinical Global Impression Scale-Improvement; CGI-E, Clinical Global Impression Scale-Efficacy Index; K-SCOPA-AUT, Korean version of Scales of Outcome in Parkinson’s Disease Autonomic; EQ-5D-5L, five-level version of the EuroQol five-dimensional questionnaire; EQ, European quality; VAS, visual analogue scale; CoV, coefficient of variation; GC, gait cycle.
Comments on this article
Variables | Values |
p value | |
---|---|---|---|
Taltirelin group (n=75) | Control group (n=74) | ||
Age (yr) | 56.60±10.55 | 52.51±12.17 | 0.0265 |
Sex | 0.9385 | ||
Men | 40 (53.33) | 39 (52.70) | |
Women | 35 (46.67) | 35 (47.30) | |
Genetic inheritance of ataxia | 0.6927 | ||
Hereditary ataxia | 41 (54.67) | 45 (60.81) | |
SCA | 37 (49.33) | 42 (56.76) | |
Other types of hereditary ataxia | 4 (5.33) | 3 (4.05) | |
Spastic ataxia of Charlevoix-Saguenay | 0 (0.00) | 1 (1.35) | |
Hereditary spastic ataxia | 1 (1.33) | 0 (0.00) | |
DRPLA gene | 1 (1.33) | 0 (0.00) | |
Chromosome analysis | 1 (1.33) | 0 (0.00) | |
Unknown | 1 (1.33) | 2 (2.70) | |
Non-hereditary ataxia | 34 (45.33) | 29 (39.19) | |
ILOCA | 34 (45.33) | 29 (39.19) | |
Disease duration (yr) | 2.33 (0.07–16.47) | 3.23 (0.00–22.50) | 0.4165 |
K-MMSE scores | 28.03±1.69 | 27.93±2.07 | 0.7894 |
Outcome measures | Taltirelin group (n=75) | Control group (n=74) | p value |
---|---|---|---|
Primary efficacy outcome measure | |||
Changes in K-SARA scores at 24 weeks from baseline | -0.51±2.79 | 0.36±2.62 | 0.0321 |
Secondary efficacy outcome measures | |||
Changes in K-SARA scores at 12 weeks from baseline | -0.47±3.05 | 0.05±2.66 | 0.1151 |
Changes in CGI-S scores at 24 weeks from baseline | 0.08±0.56 | 0.03±0.47 | 0.4925 |
Changes in CGI-I scores at 24 weeks from 4 weeks | 0.15±0.65 | 0.31±0.57 | 0.1170 |
Changes in CGI-E scores at 24 weeks from 4 weeks | -0.08±0.65 | -0.16±0.57 | 0.3440 |
Changes in K-SCOPA-AUT scores at 24 weeks from baseline | -2.27±6.09 | -0.45±5.34 | 0.2349 |
Changes in EQ-5D-5L scores at 24 weeks from baseline | |||
EQ-5D-5L descriptive system | 0.00±0.16 | -0.02±0.13 | 0.3159 |
EQ VAS | 1.92±21.72 | -3.88±18.32 | 0.1539 |
Changes in Tinetti balance and gait scores at 24 weeks from baseline | 0.03±4.02 | -0.34±3.25 | 0.5874 |
Spatio-temporal parameters of gait | |||
Spatial parameters | |||
Changes in step length at 24 weeks from baseline (cm) | |||
Left | -1.25±8.93 | -0.95±7.22 | >0.9999 |
Right | -1.52±8.09 | -1.00±6.00 | 0.9320 |
Changes in CoV of step length at 24 weeks from baseline (%) | |||
Left | 0.51±7.43 | 3.44±13.29 | 0.2066 |
Right | 1.86±14.76 | -3.07±11.17 | 0.5682 |
Changes in stride length at 24 weeks from baseline (cm) | |||
Left | -2.96±17.19 | -1.65±12.23 | 0.9008 |
Right | -2.89±15.96 | -2.37±12.11 | 0.8286 |
Changes in CoV of stride length at 24 weeks from baseline (%) | |||
Left | 3.51±16.33 | -0.68±5.78 | 0.6273 |
Right | 1.29±9.67 | -0.19±5.30 | 0.4704 |
Changes in base of support at 24 weeks from baseline (cm) | |||
Left | 0.34±4.41 | 0.29±3.87 | 0.8388 |
Right | 0.41±4.40 | 0.43±3.65 | 0.9895 |
Changes in CoV of base of support at 24 weeks from baseline (%) | |||
Left | -4.72±15.87 | -2.12±12.16 | 0.9112 |
Right | -3.44±13.87 | -2.50±10.98 | 0.7930 |
Temporal parameters | |||
Changes in walking velocity at 24 weeks from baseline (cm/s) | -1.14±14.74 | -3.15±13.28 | 0.5651 |
Changes in cadence at 24 weeks from baseline (step/min) | 1.49±11.81 | -4.41±16.06 | 0.2761 |
Changes in proportion in gait cycle of swing time at 24 weeks from baseline (%GC) | |||
Left | -1.37±7.11 | -1.48±7.34 | 0.2077 |
Right | -0.62±6.27 | -1.42±5.07 | 0.3792 |
Changes in proportion in gait cycle of stance time at 24 weeks from baseline (%GC) | |||
Left | 1.36±7.10 | 3.00±6.20 | 0.1023 |
Right | -0.06±7.48 | 1.41±5.05 | 0.2997 |
Changes in proportion in gait cycle of single support time at 24 weeks from baseline (%GC) | |||
Left | -0.89±6.53 | -1.41±5.03 | 0.4584 |
Right | -1.36±7.16 | -1.69±7.43 | 0.1380 |
Changes in proportion in gait cycle of double support time at 24 weeks from baseline (%GC) | |||
Left | -0.36±10.25 | 5.77±14.20 | 0.1346 |
Right | 0.69±7.91 | 4.02±8.93 | 0.1850 |
SOC/PT | Safety set taltirelin group (n=77) |
Safety set control group (n=80) |
p value | ||
---|---|---|---|---|---|
n (%) | E | n (%) | E | ||
AE | 0.3828 | ||||
Total incidences | 22 (28.57) | 33 | 18 (22.50) | 22 | |
Injury, poisoning and procedural complications | 7 (9.09) | 8 | 2 (2.50) | 3 | |
Rib fracture | 2 (2.60) | 3 | 0 (0.00) | 0 | |
Limb injury | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Fall | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Ligament sprain | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Hand fracture | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Procedural pain | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Skin laceration | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Spinal column injury | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Head injury | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Psychiatric disorders | 2 (2.60) | 2 | 4 (5.00) | 4 | |
Insomnia | 2 (2.60) | 2 | 3 (3.75) | 3 | |
Rapid eye movement sleep behaviour disorder | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Skin and subcutaneous tissue disorders | 2 (2.60) | 2 | 4 (5.00) | 4 | |
Pruritus | 1 (1.30) | 1 | 1 (1.25) | 1 | |
Keloid scar | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Rash | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Urticaria | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Psoriasis | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Gastrointestinal disorders | 4 (5.19) | 4 | 2 (2.50) | 2 | |
Abdominal pain upper | 1 (1.30) | 1 | 1 (1.25) | 1 | |
Gastric ulcer | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Nausea | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Dyspepsia | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Dry mouth | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Nervous system disorders | 5 (6.49) | 5 | 1 (1.25) | 1 | |
Dizziness | 3 (3.90) | 3 | 0 (0.00) | 0 | |
Somnolence | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Seizure | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Headache | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Investigations | 2 (2.60) | 2 | 2 (2.50) | 2 | |
Blood creatine phosphokinase increased | 0 (0.00) | 0 | 2 (2.50) | 2 | |
Thyroxine free increased | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Liver function test abnormal | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Musculoskeletal and connective tissue disorders | 2 (2.60) | 2 | 1 (1.25) | 1 | |
Back pain | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Muscular weakness | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Arthralgia | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Infections and infestations | 2 (2.60) | 2 | 0 (0.00) | 0 | |
Nasopharyngitis | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Otitis externa | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Metabolism and nutrition disorders | 1 (1.30) | 1 | 1 (1.25) | 1 | |
Decreased appetite | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Hyperlipidaemia | 1 (1.30) | 1 | 0 (0.00) | 0 | |
General disorders and administration site conditions | 1 (1.30) | 1 | 1 (1.25) | 1 | |
Gait disturbance | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Feeling hot | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Reproductive system and breast disorders | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Menstruation irregular | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Renal and urinary disorders | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Haematuria | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Hepatobiliary disorders | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Hepatotoxicity | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Congenital, familial and genetic disorders | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Congenital cystic kidney disease | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Ear and labyrinth disorders | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Vertigo | 1 (1.30) | 1 | 0 (0.00) | 0 | |
Vascular disorders | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Hypertension | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Surgical and medical procedures | 0 (0.00) | 0 | 1 (1.25) | 1 | |
Removal of internal fixation | 0 (0.00) | 0 | 1 (1.25) | 1 |
Values are presented as mean±standard deviation, number (%), or median with the range unless otherwise indicated. SCA, spinocerebellar ataxia; ILOCA, idiopathic late onset cerebellar ataxia; K-MMSE, Korean Mini-Mental State Examination.
Values are presented as mean±standard deviation. K-SARA, Korean version of Scale for the Assessment and Rating of Ataxia; CGI-S, Clinical Global Impression Scale-Severity; CGI-I, Clinical Global Impression Scale-Improvement; CGI-E, Clinical Global Impression Scale-Efficacy Index; K-SCOPA-AUT, Korean version of Scales of Outcome in Parkinson’s Disease Autonomic; EQ-5D-5L, five-level version of the EuroQol five-dimensional questionnaire; EQ, European quality; VAS, visual analogue scale; CoV, coefficient of variation; GC, gait cycle.
SOC, system organ class; PT, preferred term; E, number of events; AE, adverse event.
JEE YOUNG LEE
January 30, 2025