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PCSK9 Inhibitor Treatment for Patients With SPG5

NCT ID: NCT04101643

Last Updated: 2021-11-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-09-29

Study Completion Date

2023-01-03

Brief Summary

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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.

Detailed Description

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Conditions

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Hereditary Spastic Paraplegia Type 5

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Evolocumab group

Eligible patients receive subcutaneous injections of evolocumab 420 mg

Group Type EXPERIMENTAL

evolocumab

Intervention Type DRUG

Eligible patients receive subcutaneous injections of evolocumab 420 mg

Interventions

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evolocumab

Eligible patients receive subcutaneous injections of evolocumab 420 mg

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

14 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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First Affiliated Hospital of Fujian Medical University

OTHER

Sponsor Role lead

Responsible Party

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Wan-Jin Chen

Neurology department

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Wan-Jin Chen

Role: PRINCIPAL_INVESTIGATOR

First Affiliated Hospital Fujian Medical University

Locations

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Department of Neurology , First Affiliated Hospital Fujian Medical University

Fuzhou, , China

Site Status RECRUITING

Countries

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China

Central Contacts

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Ying Fu

Role: CONTACT

Phone: 13920263588

Email: [email protected]

Facility Contacts

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Ying Fu

Role: primary

Other Identifiers

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MRCTA, ECFAHofFMU[2019]209

Identifier Type: -

Identifier Source: org_study_id