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

A Prospective Single Arm Clinical Trial of Calcium Folinate in the Treatment of Spastic Paraplegia 56

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 Overview

• Status: Recruiting
• Conditions: Hereditary Spastic Paraplegia
• Intervention / Treatment: Drug: calcium folinate

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Recruiting
      • Shanghai 6th People's Hospita
      • Contact: Shankai Yin; Phone Number: +8664369181; Email: slxck@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: calcium folinate treatment group; 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
SPRS score	The change in the Spastic Paraplegia Rating Scale (SPRS) score from baseline (range: 0-52, higher scores mean a worse outcome).	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).	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.	At the end of the 5-year follow-up period
Cranial CT/MRI	The change in the cranial CT/MRI from baseline.	At the end of the 5-year follow-up period
Gait examination	The change in the gait examination from baseline.	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).	At the end of the 5-year follow-up period
High density electroencephalogram	The change in the high density electroencephalogram from baseline.	At the end of the 5-year follow-up period

Collaborators and Investigators

• Sponsor: Shanghai 6th People's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): July 1, 2024
• Primary Completion (Estimated): May 31, 2030
• Study Completion (Estimated): May 31, 2030

Study Registration Dates

• First Submitted: June 8, 2024
• First Submitted That Met QC Criteria: June 21, 2024
• First Posted (Actual): June 27, 2024

Study Record Updates

• Last Update Posted (Actual): June 27, 2024
• Last Update Submitted That Met QC Criteria: June 21, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Congenital Abnormalities; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Manifestations; Heredodegenerative Disorders, Nervous System; Nervous System Malformations; Paralysis; Muscle Hypertonia; Polyneuropathies; Hereditary Sensory and Motor Neuropathy; Muscle Spasticity; Paraplegia; Spastic Paraplegia, Hereditary; Physiological Effects of Drugs; Protective Agents; Micronutrients; Vitamins; Calcium-Regulating Hormones and Agents; Antidotes; Vitamin B Complex; Leucovorin; Calcium; Levoleucovorin
• Other Study ID Numbers: 2024-058-(1)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No