Calcium Folinate Treatment of Spastic Paraplegia 56
ConditionHereditary Spastic Paraplegia
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorShanghai 6th People's Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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;
Trial design
Design model
Single group
Treatments tested in this trial
calcium folinate
DrugIntravenous infusion and/or oral therapy
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: calcium folinate treatment groupExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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 periodSecondary outcomes
1
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 period2
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 period3
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 period4
Cranial CT/MRI
The change in the cranial CT/MRI from baseline.
Time frame
At the end of the 5-year follow-up periodSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov