Calcium Folinate Treatment of Spastic Paraplegia 56
- Registration Number
- NCT06478238
- Lead Sponsor
- Shanghai 6th People's Hospital
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 10
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description calcium folinate treatment group calcium folinate Drug: calcium folinate Phase I: calcium folinate infusion intravenously for 5 consecutive days at a dose of 1mg/kg/day in two divided doses per day. Then it was changed to oral administration at a dose of 2mg/kg/day during hospitalization. Phase II: long-term oral medication at a dose of 2mg/ kg/day in two daily doses.
- Primary Outcome Measures
Name Time Method 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).
- Secondary Outcome Measures
Name Time Method High density electroencephalogram At the end of the 5-year follow-up period The change in the high density electroencephalogram from baseline.
Gait examination At the end of the 5-year follow-up period The change in the gait examination from baseline.
MMSE score At the end of the 5-year follow-up period The change in the Mini-Mental State Examination (MMSE) score from baseline (range: 0-30, higher scores mean a better outcome).
Laboratory indicators At the end of the 5-year follow-up period The change in the Laboratory indicators (blood biochemistry, lipid metabolism, folate, etc) and the number of participants with abnormal laboratory indicators.
Cranial CT/MRI At the end of the 5-year follow-up period The change in the cranial CT/MRI from baseline.
MoCA score At the end of the 5-year follow-up period The change in the Montreal Cognitive Assessment (MoCA) score from baseline (range: 0-30, higher scores mean a better outcome).
SPRS score At the end of the 5-year follow-up period The change in the Spastic Paraplegia Rating Scale (SPRS) score from baseline (range: 0-52, higher scores mean a worse outcome).
Trial Locations
- Locations (1)
Shanghai 6th People's Hospita
🇨🇳Shanghai, Shanghai, China