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NCT Number: NCT06478238

Calcium Folinate 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.

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Key information

Conditions

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

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Shanghai 6th People's Hospita

Shanghai, Shanghai Municipality, China

Location status: Recruiting

Location contact

Shankai Yin

CONTACT

[email protected]

+8664369181

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Calcium folinate

Drug

Intravenous infusion and/or oral therapy

Primary outcomes

  1. GMFM-88

    Time frame: At the end of the 5-year follow-up period

    The change in the Gross Motor Function Measure-88 (GMFM-88) score from baseline (range: 0-264, higher scores mean a better outcome).

Secondary outcomes

  1. SPRS score

    Time frame: At the end of the 5-year follow-up period

    The change in the Spastic Paraplegia Rating Scale (SPRS) score from baseline (range: 0-52, higher scores mean a worse outcome).

  2. MMSE score

    Time frame: At the end of the 5-year follow-up period

    The change in the Mini-Mental State Examination (MMSE) score from baseline (range: 0-30, higher scores mean a better outcome).

  3. Laboratory indicators

    Time frame: At the end of the 5-year follow-up period

    The change in the Laboratory indicators (blood biochemistry, lipid metabolism, folate, etc) and the number of participants with abnormal laboratory indicators.

  4. Cranial CT/MRI

    Time frame: At the end of the 5-year follow-up period

    The change in the cranial CT/MRI from baseline.

  5. Gait examination

    Time frame: At the end of the 5-year follow-up period

    The change in the gait examination from baseline.

  6. MoCA score

    Time frame: At the end of the 5-year follow-up period

    The change in the Montreal Cognitive Assessment (MoCA) score from baseline (range: 0-30, higher scores mean a better outcome).

  7. High density electroencephalogram

    Time frame: At the end of the 5-year follow-up period

    The change in the high density electroencephalogram from baseline.

Sponsors and collaborators

Lead sponsor

Shanghai 6th People's Hospital

Other

Registry information

Official study title

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

Acronym: CFT-SPG56

Important dates

Study start
2024
Primary completion
2030
Study completion
2030
First posted
Jun 27, 2024
Registry last updated
Jun 27, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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