An Early Phase 1 interventional study of calcium folinate in Hereditary Spastic Paraplegia, sponsored by Shanghai 6th People's Hospital. Recruiting at 1 site in China. Per ClinicalTrials.gov, last updated 2024-06-27.
Sponsored by Shanghai 6th People's Hospital · Early Phase 1, Interventional, and Treatment
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.
704 studies on the registry are indexed under Muscle Spasticity; 149 are open to participants now.
This study's planned enrollment of 10 is below the median of 36 across 525 interventional studies indexed under Muscle Spasticity.
Browse Muscle Spasticity studies →Shanghai 6th People's Hospital is the lead sponsor of 69 studies on the registry; 40 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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.
Drug: calcium folinate
Intravenous infusion and/or oral therapy
GMFM-88
The change in the Gross Motor Function Measure-88 (GMFM-88) score from baseline (range: 0-264, higher scores mean a better outcome).
Time frame: At the end of the 5-year follow-up period
SPRS score
The change in the Spastic Paraplegia Rating Scale (SPRS) score from baseline (range: 0-52, higher scores mean a worse outcome).
Time frame: At the end of the 5-year follow-up period
MMSE score
The change in the Mini-Mental State Examination (MMSE) score from baseline (range: 0-30, higher scores mean a better outcome).
Time frame: At the end of the 5-year follow-up period
Laboratory indicators
The change in the Laboratory indicators (blood biochemistry, lipid metabolism, folate, etc) and the number of participants with abnormal laboratory indicators.
Time frame: At the end of the 5-year follow-up period
Cranial CT/MRI
The change in the cranial CT/MRI from baseline.
Time frame: At the end of the 5-year follow-up period
Gait examination
The change in the gait examination from baseline.
Time frame: At the end of the 5-year follow-up period
MoCA score
The change in the Montreal Cognitive Assessment (MoCA) score from baseline (range: 0-30, higher scores mean a better outcome).
Time frame: At the end of the 5-year follow-up period
High density electroencephalogram
The change in the high density electroencephalogram from baseline.
Time frame: At the end of the 5-year follow-up period
Plan to share: Undecided
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Shanghai 6th People's Hospital