Calcium Folinate Treatment of Spastic Paraplegia 56 (CFT-SPG56)

Shanghai Jiao Tong University

Status and phase: Enrolling; Early Phase 1

Conditions: Hereditary Spastic Paraplegia

Treatments: Drug: calcium folinate

Study type: Interventional

Funder types: Other

Identifiers

NCT06478238

2024-058-(1)

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

calcium folinate treatment group

Experimental group

Description:

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.

Treatment:

Drug: calcium folinate