Calcium Folinate Treatment of Spastic Paraplegia 56

NCT06478238 | Recruiting Early Phase 1 DMC

• 1 other identifier: 2024-058-(1)
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hereditary Spastic Paraplegia

Outcome Measures

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).

  At the end of the 5-year follow-up period

Secondary Outcomes (7)

• SPRS score

  At the end of the 5-year follow-up period

• MMSE score

  At the end of the 5-year follow-up period

• Laboratory indicators

  At the end of the 5-year follow-up period

• Cranial CT/MRI

  At the end of the 5-year follow-up period

• Gait examination

  At the end of the 5-year follow-up period

Study Arms (1)

calcium folinate treatment group

EXPERIMENTAL

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

Interventions

calcium folinate DRUG

Intravenous infusion and/or oral therapy

calcium folinate treatment group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;
• Participating in another study drug trial and used the investigational drug in the past 30 days;
• Subjects have poor compliance or other factors that are not suitable for participating in the clinical trial.

Sponsors & Collaborators

• Shanghai 6th People's Hospital: lead

Study Sites (1)

Shanghai 6th People's Hospita

Shanghai, Shanghai Municipality, China

RECRUITING

MeSH Terms

Conditions

Spastic Paraplegia, Hereditary

Interventions

Leucovorin

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Formyltetrahydrofolates; Tetrahydrofolates; Folic Acid; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Coenzymes; Enzymes and Coenzymes

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of the Department of Neurology

Study Record Dates

• First Submitted: June 8, 2024
• First Posted: June 27, 2024
• Study Start: July 1, 2024
• Primary Completion (Estimated): May 31, 2030
• Study Completion (Estimated): May 31, 2030
• Last Updated: June 27, 2024

Record last verified: 2024-06

Locations

CN (1)