Hereditary Spastic Paraplegia Type 5Phase 1/2NCT04101643
PCSK9 Inhibitor Treatment for Patients With SPG5
A Phase 1/2 study testing evolocumab in people with Hereditary Spastic Paraplegia Type 5, run by First Affiliated Hospital of Fujian Medical University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 to 80 years
- Conditions the study is for
- Hereditary Spastic Paraplegia Type 5
- What is being tested?
- evolocumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 29 Sep 2019to 3 Jan 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age 14-80 years
- Probands with clinically manifest hereditary spastic paraplegia
- Genetically confirmed diagnosis of SPG5
Who cannot join
- Comprised treatment with statins 3 months prior to enrolment
- Contraindications to PCSK9 inhibitor therapy
- Pregnancy was excluded in women of childbearing age
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Neurology , First Affiliated Hospital Fujian Medical University
Fuzhou
Ying Fu, fuying1995@163.com
Wan-Jin Chen
About this study
Official title: 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.
- Conditions
- Hereditary Spastic Paraplegia Type 5
- Being tested
- evolocumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
evolocumabDrug
Eligible patients receive subcutaneous injections of evolocumab 420 mg
| Study group | Role | What they receive |
|---|---|---|
| Evolocumab group | Experimental | Eligible patients receive subcutaneous injections of evolocumab 420 mg Receives: Drug: evolocumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change of 27-hydroxycholesterol (27-OHC)
Cholesterol is initially side chain oxidized and the resulting 27-hydroxycholesterol (27-OHC) are 7a-hydroxylated
Measured: up to 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2019
- Study start29 Sep 2019
- Primary completion3 Jan 2023 (estimated)
- Study completion3 Jan 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Spastic Paraplegia Type 5Studies of the same condition, open and under way.
- More trials run by First Affiliated Hospital of Fujian Medical UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University
- Investigators
- Wan-Jin Chen, Principal investigator, First Affiliated Hospital Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04101643
- Sponsor's protocol ID
- MRCTA, ECFAHofFMU[2019]209
- Status last verified
- Nov 2021
- First submitted
- 22 Sep 2019
- Last updated
- 23 Nov 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PCSK9 Inhibitor Treatment for Patients With Hereditary Spastic Paraplegia Type 5
- ClinicalTrials.gov ID
- NCT04101643
- Acronym / sponsor ID
- MRCTA, ECFAHofFMU[2019]209
- Conditions
- Hereditary Spastic Paraplegia Type 5
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Fujian Medical UniversityUniversity, hospital or other
- Study start
- 29 Sep 2019 (actual)
- Primary completion
- 3 Jan 2023 (estimated)
- Study completion
- 3 Jan 2023 (estimated)
- First posted
- 24 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Evolocumab group | Experimental | Drug: evolocumabEligible patients receive subcutaneous injections of evolocumab 420 mg |
Who may qualify
- Ages
- 14 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Age 14-80 years
- Probands with clinically manifest hereditary spastic paraplegia
- Genetically confirmed diagnosis of SPG5
Key exclusion criteria (3 listed)
- Comprised treatment with statins 3 months prior to enrolment
- Contraindications to PCSK9 inhibitor therapy
- Pregnancy was excluded in women of childbearing age
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| evolocumabEligible patients receive subcutaneous injections of evolocumab 420 mg | Drug | Evolocumab group |
Primary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wan-Jin Chen principal investigator
First Affiliated Hospital Fujian Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing evolocumab in people with Hereditary Spastic Paraplegia Type 5, run by First Affiliated Hospital of Fujian Medical University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ying Fu (13920263588, fuying1995@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change of 27-hydroxycholesterol (27-OHC) (time frame: up to 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Nov 2021.