Ziclague^® (Alpinia Zerumbet oil) in patients with hereditary spastic paraplegia - the randomized controlled ZISPAST trial.

IF 3.5 2区医学 Q2 GENETICS & HEREDITY

Orphanet Journal of Rare Diseases Pub Date : 2025-09-24 DOI:10.1186/s13023-025-04007-5

Fabricio Diniz de Lima, Katiane Raisa Servelhere, Maria Fernanda Ribeiro Bittar, Carelis González-Salazar, Alberto Rolim Muro Martinez, Tatiana Benaglia, Benilton de Sá Carvalho, José Luiz Pedroso, Orlando Graziani Povoas Barsottini, Anamarli Nucci, Marcondes Cavalcante França

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引用次数: 0

Abstract

Background: Spasticity is a hallmark of hereditary spastic paraplegia (HSP) and contributes to gait impairment. Alpinia zerumbet oil (Ziclague^®) is a topical anti-spastic agent approved in Brazil, but not yet explored in HSP. Then, it was designed a randomized, placebo-controlled, double-blind, crossover trial to evaluate the efficacy and safety of Ziclague^® in patients with HSP: the ZISPAST trial.

Methods: Each participant was randomly assigned to receive 0.8 mL of Ziclague^® dermal applications (0.064 mL of Alpinia Zerumbet equally divided in each adductor magnus and each triceps surae) or placebo 0.9%. The primary endpoint was change from baseline in self-selected gait velocity and secondary endpoints included changes in maximal gait velocity, walking endurance, spasticity, muscle strength, Spastic Paraplegia Rating Scale, pain, fatigue, quality of life and post-treatment perceived change and general impression. Adverse events (AE) were also recorded.

Results: Fifty-seven patients were enrolled, 37 (64.9%) of whom were men and 50 (87.7%) with pure phenotype. Mean age was 44 (± 11.6; range, 22 to 74), mean age of onset 23 (± 16.6; range, < 1 to 62) and mean disease duration 21 (± 13.1; range, 2 to 54) years. Compared to baseline, there were no significant between-group differences in primary and secondary outcomes. There were few AEs, all of them mild. Incidence of AE was similar between treatment arms (p = 0.56).

Conclusions: Ziclague^® was safe in patients with HSP, but it was not able to improve gait velocity considering methods and protocol used.

Trial registration number: U1111-1218-2539. Registered 28 August 2018, https://ensaiosclinicos.gov.br/rg/RBR-83xh37 .

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Ziclague®（Alpinia Zerumbet oil）用于遗传性痉挛性截瘫患者的随机对照ZISPAST试验

背景：痉挛是遗传性痉挛性截瘫（HSP）的标志，并有助于步态障碍。Alpinia zerumbet oil （Ziclague®）是巴西批准的外用抗痉挛剂，但尚未在HSP中进行探索。然后，设计了一项随机、安慰剂对照、双盲、交叉试验来评估Ziclague®对HSP患者的疗效和安全性：ZISPAST试验。方法：每个参与者被随机分配接受0.8 mL Ziclague®皮肤涂抹剂（0.064 mL Alpinia Zerumbet平均分配于每个大内收肌和每个表面三头肌）或0.9%安慰剂。主要终点是自选步态速度的基线变化，次要终点包括最大步态速度、步行耐力、痉挛、肌肉力量、痉挛性截瘫评定量表、疼痛、疲劳、生活质量和治疗后感知变化和总体印象的变化。不良事件（AE）也被记录。结果：共纳入57例患者，其中男性37例（64.9%），纯表型50例（87.7%）。平均年龄44岁（±11.6，范围22 ~ 74），平均发病年龄23岁（±16.6，范围< 1 ~ 62），平均病程21岁（±13.1，范围2 ~ 54）。与基线相比，两组间主要和次要结局无显著差异。有几个ae，都是轻微的。AE的发生率在治疗组之间相似（p = 0.56）。结论：Ziclague®对HSP患者是安全的，但考虑到所采用的方法和方案，它不能改善步态速度。试验登记号：U1111-1218-2539。2018年8月28日注册，https://ensaiosclinicos.gov.br/rg/RBR-83xh37。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Orphanet Journal of Rare Diseases 医学-医学：研究与实验

CiteScore

6.30

自引率

8.10%

发文量

418

审稿时长

4-8 weeks

期刊介绍： Orphanet Journal of Rare Diseases is an open access, peer-reviewed journal that encompasses all aspects of rare diseases and orphan drugs. The journal publishes high-quality reviews on specific rare diseases. In addition, the journal may consider articles on clinical trial outcome reports, either positive or negative, and articles on public health issues in the field of rare diseases and orphan drugs. The journal does not accept case reports.