- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04101643
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
- Name: Ying Fu
- Phone Number: 13920263588
- Email: fuying1995@163.com
Study Locations
-
-
-
Fuzhou, China
- Recruiting
- Department of Neurology , First Affiliated Hospital Fujian Medical University
-
Contact:
- Ying Fu
- Email: fuying1995@163.com
-
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
12 years to 78 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
|
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.
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
- 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
- Molecular Mechanisms of Pharmacological Action
- Antimetabolites
- Anticholesteremic Agents
- Hypolipidemic Agents
- Lipid Regulating Agents
- Evolocumab
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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