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.
Eligibility
Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Patients meet the clinical diagnostic standard of hereditary spastic paraplegia (HSP);
2. Spastic paraplegia type 56 (SPG56) was diagnosed by CYP2U1 pathogenic mutation;
3. Patients are willing to participate in clinical trials and able to understand and comply with the research program.
Exclusion Criteria:
1. Patients are allergic to the drugs involved in the study;
2. Other neurological diseases likely affecting the evaluation of study treatment;
3. Other medical conditions such as: heart disease, tumor, blood disease, liver disease, kidney disease, etc. in the past 1 year;
4. Pregnancy or lactating women or subjects who are unable to use appropriate contraception during the trial;
5. Participating in another study drug trial and used the investigational drug in the past 30 days;
6. Subjects have poor compliance or other factors that are not suitable for participating in the clinical trial.
Primary outcome measure(s)
GMFM-88 — At the end of the 5-year follow-up period The change in the Gross Motor Function Measure-88 (GMFM-88) score from baseline (range: 0-264, higher scores mean a better outcome).
Trial sites (1)
Facility
City
Region
Status
Shanghai 6th People's Hospita
Shanghai
Shanghai Municipality
Recruiting
More Shanghai 6th People's Hospital trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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