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Recruiting NCT06478238

Calcium Folinate Treatment of Spastic Paraplegia 56

Early Phase I Interventional Hereditary Spastic Paraplegia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: calcium folinate.
Who it may be relevant to
Registry conditions: Hereditary Spastic Paraplegia. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Prospective Single Arm Clinical Trial of Calcium Folinate in the Treatment of Spastic Paraplegia 56

Overview

SPG56 is one of the complicated and early-onset HSP subtypes caused by genetic mutations in CYP2U1. So far, there is no standardized and specific clinical therapy for SPG56. The goal of this clinical trial is to explore the efficacy and safety of calcium folinate in the treatment of SPG56 patients. This study is prospective, open-label and single arm and this trial will last for 6 years. A total of 10 patients will participate and they will receive calcium folinate treatment and professional clinical evaluation regularly.

Interventions

  • Drug calcium folinate
    Intravenous infusion and/or oral therapy

Primary outcome measures

  • GMFM-88 [Time frame: At the end of the 5-year follow-up period]
Secondary outcome measures (7)
  • SPRS score [Time frame: At the end of the 5-year follow-up period]
  • MMSE score [Time frame: At the end of the 5-year follow-up period]
  • Laboratory indicators [Time frame: At the end of the 5-year follow-up period]
  • Cranial CT/MRI [Time frame: At the end of the 5-year follow-up period]
  • Gait examination [Time frame: At the end of the 5-year follow-up period]
  • MoCA score [Time frame: At the end of the 5-year follow-up period]
  • High density electroencephalogram [Time frame: At the end of the 5-year follow-up period]

Eligibility criteria

Inclusion criteria

  • Patients meet the clinical diagnostic standard of hereditary spastic paraplegia (HSP);
  • Spastic paraplegia type 56 (SPG56) was diagnosed by CYP2U1 pathogenic mutation;
  • Patients are willing to participate in clinical trials and able to understand and comply with the research program.

Exclusion criteria

  • Patients are allergic to the drugs involved in the study;
  • Other neurological diseases likely affecting the evaluation of study treatment;
  • Other medical conditions such as: heart disease, tumor, blood disease, liver disease, kidney disease, etc. in the past 1 year;
  • Pregnancy or lactating women or subjects who are unable to use appropriate contraception during the trial;
  • Participating in another study drug trial and used the investigational drug in the past 30 days;
  • Subjects have poor compliance or other factors that are not suitable for participating in the clinical trial.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Shanghai 6th People's Hospita — Shanghai

Identifiers

NCT: NCT06478238 · 2024-058-(1)

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗