Actively Recruiting
A Prospective Single-Arm Study of Calcium Folinate Treatment for Hereditary Spastic Paraplegia 56 Evaluating Safety and Effects Over Six Years
Led by Shanghai 6th People's Hospital · Updated on 2024-06-27
10
Participants Needed
1
Research Sites
308 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating the effects of calcium folinate in patients diagnosed with Spastic Paraplegia 56 (SPG56), a complicated and early-onset form of hereditary spastic paraplegia caused by genetic mutations in the CYP2U1 gene. Currently, there is no standardized or specific treatment for SPG56, making this prospective clinical trial important for exploring potential therapy options. This is an early phase 1, open-label, single-arm study designed to assess both safety and efficacy over a long-term period. Participants in this study will receive calcium folinate through intravenous infusion and/or oral therapy. The treatment plan consists of a single study arm where all enrolled patients will receive the investigational treatment. A total of 10 patients will be treated and monitored closely throughout the trial, which spans approximately 6 years. Regular professional clinical evaluations will be conducted to monitor the participants' responses to the treatment. During the study, participants will undergo ongoing clinical assessments, including evaluations using the GMFM-88 scale at the end of the 5-year follow-up period to measure motor function. Safety and efficacy will be continuously monitored throughout the trial duration. Patients will be regularly evaluated to ensure adherence and to track any changes in their condition over time. This long-term follow-up aims to provide comprehensive data on the effects of calcium folinate in SPG56 patients.
CONDITIONS
Official Title
Calcium Folinate Treatment of Spastic Paraplegia 56
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients meet the clinical diagnostic standard of hereditary spastic paraplegia (HSP)
- Spastic paraplegia type 56 (SPG56) diagnosed by CYP2U1 pathogenic mutation
- Patients are willing to participate and able to understand and comply with the study program
You will not qualify if you...
- Allergy to drugs involved in the study
- Other neurological diseases that may affect evaluation of treatment
- Medical conditions such as heart disease, tumor, blood disease, liver disease, kidney disease in the past year
- Pregnancy, lactation, or inability to use appropriate contraception during the trial
- Participation in another investigational drug trial or use of investigational drug in past 30 days
- Poor compliance or other factors making the participant unsuitable for the trial
AI-Screening
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Trial Site Locations
Total: 1 location
1
Shanghai 6th People's Hospita
Shanghai, Shanghai Municipality, China
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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