Evidence map›Paper›PMID 41428099›Full record

Trial reportNeurogenetics2025

Miglustat does not impact clinical progression in patients with spastic paraplegia type 11.

Serena Mero, Ivana Ricca, Salvatore Rossi, Sabato Mellone, Olimpia Musumeci, Gianmarco Dalla Zanna, Elena Michelucci, Silvia Bagnoli, Benedetta Nacmias, Alessandra Tessa and 3 more

Abstract readClinical Trial, Phase II
PubMed Publisher
In one paragraph

Trial report in Neurogenetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Serena Mero *Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit, IRCCS Fondazione Stella Maris, via dei Giacinti 2, Pisa, Italy.
Ivana Ricca *Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit, IRCCS Fondazione Stella Maris, via dei Giacinti 2, Pisa, Italy.
Salvatore RossiDepartment of Neurosciences, Università Cattolica del Sacro Cuore, Rome, Italy.
Sabato MellonePersonal Health Systems Lab, University of Bologna, Bologna, Italy.
Olimpia MusumeciUnit of Neurology and Neuromuscular Disorders, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Gianmarco Dalla ZannaDepartment of Neurosciences, Università Cattolica del Sacro Cuore, Rome, Italy.
Elena MichelucciCNR-Istituto di Fisiologia Clinica, Pisa, Italy.
Silvia BagnoliNeurology Unit, Department of Neuroscience, Psychology, Drug Research and Child Health, University of Florence, Florence, Italy.
Benedetta NacmiasNeurology Unit, Department of Neuroscience, Psychology, Drug Research and Child Health, University of Florence, Florence, Italy.
Alessandra TessaMolecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit, IRCCS Fondazione Stella Maris, via dei Giacinti 2, Pisa, Italy.
Silvia RocchiccioliCNR-Istituto di Fisiologia Clinica, Pisa, Italy.
Gabriella SilvestriDepartment of Neurosciences, Università Cattolica del Sacro Cuore, Rome, Italy.
Filippo M SantorelliMolecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit, IRCCS Fondazione Stella Maris, via dei Giacinti 2, Pisa, Italy. filippo3364@gmail.com.

Funding

Ministero della Salute Ricerca Corrente 2024
6 · The paper itself

Abstract

Spastic paraplegia type 11 (SPG11) is a progressive neurological condition with no treatment. Possible involvement of abnormal ganglioside metabolism has been reported in mouse and human cells. Preclinical data in zebrafish and fruit fly models of the disease indicate that miglustat can improve biochemical parameters and locomotion. We assessed the short-term safety of oral miglustat in patients with SPG11. This was an open-label, single center, non-randomized, phase II study. All patients received miglustat orally (4 weeks 100 mg/tid and 8 weeks 200 mg/tid). The primary outcome was the frequency of adverse events (AEs), particularly and severe AEs (SAEs), emerging in the course of the study. As secondary outcomes we measured clinical severity scores, functional tests, and biomarkers at 3 and 6 months. Twelve-week treatment with miglustat was adequately tolerated, with only minor AEs and no SAEs reported. With a few exceptions, the treatment did not seem to impact neuromotor function in patients with SPG11. Although it showed an acceptable safety profile and no SAEs, miglustat did not significantly modify plasma sphingolipid and ganglioside profiles. Its potential therapeutic impact remains low.

Indexed as

1-DeoxynojirimycinSpastic Paraplegia, HereditaryAdultAgedDisease ProgressionFemaleHumansMaleMiddle AgedProteinsTreatment OutcomeYoung Adult1-DeoxynojirimycinmiglustatProteinsSPG11 protein, humanDrug safetyMiglustatSpastic paraplegia type 11

Identifiers

PMID41428099

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.