Calcium Folinate Treatment of Spastic Paraplegia 56

Early Phase 1RecruitingNCT06478238

Shanghai 6th People's Hospital10 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hereditary Spastic Paraplegia

Interventions

calcium folinateDRUG

Intravenous infusion and/or oral therapy

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: 1. Patients meet the clinical diagnostic standard of hereditary spastic paraplegia (HSP); 2. Spastic paraplegia type 56 (SPG56) was diagnosed by CYP2U1 pathogenic mutation; 3. Patients are willing to participate in clinical trials and able to understand and comply with the research program. Exclusion Criteria: 1. Patients are allergic to the drugs involved in the study; 2. Other neurological diseases likely affecting the evaluation of study treatment; 3. Other medical conditions such as: heart disease, tumor, blood disease, liver disease, kidney disease, etc. in the past 1 year; 4. Pregnancy or lactating women or subjects who are unable to use appropriate contraception during the trial; 5. Participating in another study drug trial and used the investigational drug in the past 30 days; 6. Subjects have poor compliance or other factors that are not suitable for participating in the clinical trial.

Locations (1)

Shanghai 6th People's Hospita

Shanghai, Shanghai Municipality, China

RECRUITING

Outcomes

Primary Outcomes

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).