NCT04101643

PCSK9 Inhibitor Treatment for Patients With SPG5

PCSK9 Inhibitor Treatment for Patients With Hereditary Spastic Paraplegia Type 5

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: First Affiliated Hospital of Fujian Medical University · Academic / Other
• Sex: All
• Age: 14 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

Spastic paraplegia type 5 (SPG5) is a rare subtype of hereditary spastic paraplegia, a highly heterogeneous group of neurodegenerative disorders defined by progressive neurodegeneration of the corticospinal tract motor neurons. SPG5 is caused by recessive mutations in the gene CYP7B1 encoding oxysterol-7a-hydroxylase. This enzyme is involved in the degradation of cholesterol into primary bile acids. CYP7B1 deficiency has been shown to lead to accumulation of neurotoxic oxysterols. Oxysterols were found to impair metabolic activity and viability of human cortical neurons at concentrations found in SPG5 patients, indicating that elevated levels of oxysterols might be key pathogenic factors in SPG5. Monoclonal antibodies that inhibit proprotein convertase subtilisin-kexin type 9 (PCSK9) have emerged as a new class of drugs that effectively lower cholesterol levels. Evolocumab, a member of this class, is a fully human monoclonal antibody that reduces LDL cholesterol levels by approximately 60%. We thus performed this interventional trial with Evolocumab 420 mg for SPG5 patients.

Conditions

• Hereditary Spastic Paraplegia Type 5

Interventions

Type	Name	Description
DRUG	evolocumab	Eligible patients receive subcutaneous injections of evolocumab 420 mg

Timeline

• Start date: 2019-09-29
• Primary completion: 2023-01-03
• Completion: 2023-01-03
• First posted: 2019-09-24
• Last updated: 2021-11-23

Locations

1 site across 1 country: China