Actively Recruiting
A Prospective Single Arm Clinical Trial of Calcium Folinate in the Treatment of Spastic Paraplegia 56
Led by Shanghai 6th People's Hospital · Updated on 2024-06-27
10
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Hereditary Spastic Paraplegia type 56 (SPG56) is a complex early-onset form of HSP caused by genetic mutations in the CYP2U1 gene. Currently, there is no standardized treatment for SPG56. This research aims to evaluate the safety and potential benefits of calcium folinate in SPG56 patients through a prospective, open-label, single-arm clinical trial sponsored by Shanghai 6th People's Hospital. Participants will receive calcium folinate treatment in two phases. Initially, calcium folinate is given intravenously for 5 consecutive days at 1 mg/kg/day in two divided doses. Following this, during hospitalization, treatment switches to oral calcium folinate at 2 mg/kg/day. Afterward, long-term oral calcium folinate at the same dose is continued. The trial will last for six years with a total of 10 participants. During the study, patients will undergo regular professional clinical evaluations. Researchers will measure outcomes such as the Gross Motor Function Measure (GMFM-88) after five years of follow-up. Additional assessments include the Spastic Paraplegia Rating Scale (SPRS), Mini-Mental State Examination (MMSE), laboratory tests, brain imaging (CT/MRI), gait analysis, Montreal Cognitive Assessment (MoCA), and high-density electroencephalogram at the five-year mark. Safety and compliance will also be monitored throughout the study period.
CONDITIONS
Brief 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)
- Diagnosis of spastic paraplegia type 56 (SPG56) confirmed by CYP2U1 pathogenic mutation
- Willingness and ability to participate and comply with the clinical trial
You will not qualify if you...
- Allergy to drugs involved in the study
- Presence of other neurological diseases that may affect treatment evaluation
- Serious medical conditions (heart disease, tumor, blood disease, liver disease, kidney disease) diagnosed within the past year
- Pregnancy, lactation, or inability to use appropriate contraception during the trial
- Participation in another investigational drug trial within the past 30 days
- Poor compliance or other factors unsuitable for clinical trial participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 5 years
Participants receive calcium folinate treatment starting with a 5-day intravenous infusion followed by oral therapy during hospitalization, then continue long-term oral medication.
Initial daily visits for 5 days during intravenous infusion, then ongoing oral medication with visits as scheduled by the study team
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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