A Prospective Single Arm Clinical Trial of Calcium Folinate in the Treatment of Spastic Paraplegia 56
Trial Snapshot
- Phase
- Early Phase 1
- Status
- Recruiting
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- GMFM-88
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
calcium folinate treatment group
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.
Intervention: calcium folinate (Drug)
Outcomes
Primary Outcomes
GMFM-88
Time Frame: 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 Outcomes
- High density electroencephalogram(At the end of the 5-year follow-up period)
- Gait examination(At the end of the 5-year follow-up period)
- MMSE score(At the end of the 5-year follow-up period)
- Laboratory indicators(At the end of the 5-year follow-up period)
- Cranial CT/MRI(At the end of the 5-year follow-up period)
- MoCA score(At the end of the 5-year follow-up period)
- SPRS score(At the end of the 5-year follow-up period)
Investigators
Li Cao
Head of the Department of Neurology
Shanghai 6th People's Hospital