PCSK9 Inhibitor Treatment for Patients With SPG5

November 21, 2021 updated by: Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University

PCSK9 Inhibitor Treatment for Patients With Hereditary Spastic Paraplegia Type 5

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Fuzhou, China
        • Recruiting
        • Department of Neurology , First Affiliated Hospital Fujian Medical University
        • Contact:
        • Principal Investigator:
          • Wan-Jin Chen

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years to 76 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Age 14-80 years
  • Probands with clinically manifest hereditary spastic paraplegia
  • Genetically confirmed diagnosis of SPG5

Exclusion Criteria:

  • Comprised treatment with statins 3 months prior to enrolment
  • Contraindications to PCSK9 inhibitor therapy
  • Pregnancy was excluded in women of childbearing age

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Evolocumab group
Eligible patients receive subcutaneous injections of evolocumab 420 mg
Drug: evolocumab
Eligible patients receive subcutaneous injections of evolocumab 420 mg

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The change of 27-hydroxycholesterol (27-OHC)
Time Frame: up to 4 weeks
Cholesterol is initially side chain oxidized and the resulting 27-hydroxycholesterol (27-OHC) are 7a-hydroxylated
up to 4 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

First Affiliated Hospital of Fujian Medical University

Investigators

  • Principal Investigator: Wan-Jin Chen, First Affiliated Hospital Fujian Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 29, 2019

Primary Completion (Anticipated)

January 3, 2023

Study Completion (Anticipated)

January 3, 2023

Study Registration Dates

First Submitted

September 22, 2019

First Submitted That Met QC Criteria

September 22, 2019

First Posted (Actual)

September 24, 2019

Study Record Updates

Last Update Posted (Actual)

November 23, 2021

Last Update Submitted That Met QC Criteria

November 21, 2021

Last Verified

November 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MRCTA, ECFAHofFMU[2019]209

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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