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Completed

NCT Number: NCT04912609

Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11)

Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.

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Key information

Conditions

Hereditary Spastic Paraplegia Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Sensory and Motor Neuropathy Heredodegenerative Disorders, Nervous System Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies Spastic Paraplegia Type 11 Spastic Paraplegia, Hereditary Spastic paraplegia 11, autosomal recessive

Age range

10 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

IRCCS Fondazione Stella Maris

Pisa, PI, 56128, Italy

About this study

Several experiments on subjects affected by neurodegenerative diseases with dysfunction of the autophagic-lysosomal system show that trehalose improves the pathological phenotype. This evidence indicates that trehalose could be used in patients with SPG11 to try to prevent the accumulation of glycosphingolipids at the lysosomal level and induce the genesis of new lysosomes. This study aims to record clinical data of 20 patients with SPG11 who take trehalose during 12 months.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Confirmed diagnosis of SPG11
  • Written signed informed consent

Exclusion criteria

  • Diagnosis of other concomitant neurodegenerative diseases
  • taking other experimental drugs within 30 days of the first Study visit (T0) and during the study
  • Refusal to sign informed consent

Treatment and study plan

Primary outcomes

  1. Changes from baseline in Spastic Paraplegia Rating Scale (SPRS) at 6 and 12 months

    Time frame: At baseline, month 6, month 12

    Assess changes in score of the Spastic Paraplegia Rating Scale (SPRS) over ± 10%

Secondary outcomes

  1. Changes in glycosphingolipids and gangliosides plasmatic levels

    Time frame: At baseline, month 6, month 12

    Assess changes in glycosphingolipids and gangliosides plasmatic levels over ± 10%

Sponsors and collaborators

Lead sponsor

IRCCS Fondazione Stella Maris

Other

Registry information

Official study title

An Observational Study in Subjects With Spastic Paraplegia Type 11 Taking Trehalose

Acronym: 3AL-SPG11

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Jun 3, 2021
Registry last updated
Aug 3, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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