Hypercholesterolemia, FamilialNCT04722068
Regeneron 1331 Kinetics Sub-Study HoFH
A study testing Kinetics test in people with Hypercholesterolemia, Familial, run by University of Pennsylvania at 2 sites in 2 countries.
Trial summary
To evaluate the lipoprotein kinetics of subjects enrolled in the R1500-CL-1331 clinical trial (NCT02265952) to assess the mechanism by which the evinacumab may affect lipid levels in HoFH subjects. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypercholesterolemia, Familial
- What is being tested?
- Kinetics test
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 7 Oct 2016to 1 Jan 2021
- Phase
- Interventional
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and women 18 years or older at the time of the screening visit
- Diagnosis of HoFH by one of the following:
- Documented mutation or mutations in both LDLR alleles
- Documented presence of homozygous or compound heterozygous mutations in Apo B and/or PCSK9
- Documented skin fibroblast LDLR activity <20% of normal
- Willing to consistently maintain his/her usual diet for the duration of the study
- Willing and able to comply with clinic visits and study-related procedures
- Provide signed informed consent
Who cannot join
- Background medical LMT (if applicable) that has not been stable for at least 4 weeks (6 weeks for fibrates) prior to the screening visit (week -2) (unless participating in the run-in period to stabilize)
- Having undergone lipid apheresis within 4 weeks prior to the screening visit (week -2) (unless participating in the run-in period to wash out)
- Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins. Note: Patients on thyroid replacement therapy can be included if the dosage of thyroxine has been stable for at least 12 weeks prior to screening visit (week -2)
- Use of systemic corticosteroids, unless used as replacement therapy for pituitary/adrenal disease with a stable regiment for at least 6 weeks prior to the screening visit (week -2) Note: Topical, intra-articular, nasal, inhaled, and ophthalmic steroid therapies are not considered as "systemic" and are allowed
- History of a myocardial infarction, unstable angina leading to hospitalization, coronary bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the screening visit (week -2)
- Known history of HIV and a CD4 count less than 350c/mL Note: Patients with a history of HIV can be included, as long as they:
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- have a CD4 count greater than 350c/mL;
- have an undetectable viral load for at least 12 months prior to screening;
- have no opportunistic infections or active tuberculosis for at least 12 months prior to screening;
- are not receiving prophylaxis for opportunistic infections; and have been on stable antiretroviral therapy for at least 12 months prior to screening
- Use of another investigational drug or therapy within 30 days or at least 5 half-lives (whichever is longer) prior to screening visit (week -2). Includes patients who were involved in a prior clinical trial and are not sure of their treatment assignment.
- Previous participation in any clinical trial of REGN1500
- Conditions/situation such as:
- Any clinically significant abnormality identified at the time of screening that, in the judgement of the investigator or any subinvestigator, would preclude safe completion of the study or constrain endpoints assessment; eg. major systemic diseases, patients with short life expectancy
- Patient considered by the investigator or any subinvestigator as inappropriate for this study for any reason, eg: deemed unable to meet specific protocol requirements, such as scheduled visits. Deemed unable to tolerate injections as per the patient or the investigator. Investigator or any subinvestigator, pharmacist, study coordinator, other study staff or relative thereof directly involved in the conduct of the protocol, etc. Presence of any other conditions (eg, geographic or social), either actual or anticipated, that the investigator feels would restrict or limit the patient's participation for the duration of the study
- Laboratory findings during screening period:
- Positive test for Hepatitis B surface antigen and/or Hepatitis C antibody
- Positive serum beta-human chorionic gonadotropin or urine pregnancy test in women of childbearing potential
- Triglycerides greater than 400 mg/dL ( >3.95 mmol/L) (1 repeat lab is allowed)
- Estimated glomerular filtration rate <30 mL/min/1.73m^2 according to 4-variable Modification of Diet in Renal Disease Study equation (calculated by central lab)
- Alanine amintransferase (ALT) or aspartate aminotransferase (AST) >3 x the upper limit of normal (ULN) (1 repeat lab is allowed)
- Creatine phosphokinase (CPK) >3 x ULN (1 repeat lab is allowed)
- Known hypersensitivity to monoclonal antibody therapeutics
- History of hypersensitivity reaction to doxycycline or similar compound
- Pregnant or breast-feeding women
- Sexually active men, or women of childbearing potential, who are unwilling to utilize adequate contraception for at least 6 months after the last dose of study drug.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Netherlands1 site
Department of Vascular Medicine Amsterdam UMC
Amsterdam, AZ, 1105
About this study
Official title: A Kinetic Study Investigating Lipoprotein Metabolism Before and After the Administration of REGN1500, an ANGPTL3 Inhibitor, in Patients With Homozygous Familial Hypercholesterolemia. A Sub-study for Subjects Enrolled in the R1500-CL-1331 Clinical Trial
To evaluate the lipoprotein kinetics of subjects enrolled in the R1500-CL-1331 clinical trial (NCT02265952) to assess the mechanism by which the evinacumab may affect lipid levels in HoFH subjects.
Read the detailed description
Subjects enrolled in the R1500-CL-1331 clinical trial (NCT0226595) will participate in a study visit before and after administration of the ANGPTL3 inhibitor REGN1500, evinacumab) for the evaluation of its effect on lipoprotein kinetics.
- Conditions
- Hypercholesterolemia, Familial
- Being tested
- Kinetics test
- Keywords
- Lipid metabolism, Homozygous Familial Hypercholesterolemia, Evinacumab
- Medical subject headings
- Hyperlipoproteinemia Type II, Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Kinetics testOther
Subjects will participate in two study visits to evaluate lipoprotein kinetics before and after administration of evinacumab.
| Study group | Role | What they receive |
|---|---|---|
| Volunteers with known HoFH and particpated in R1500-CL-1331 clinical trial | Experimental | Not described Receives: Other: Kinetics test |
What is being measured
The primary outcome is the main question the study is designed to answer.
Fractional catabolic rate (FCR) pools/day
First visit to be performed prior to first evinacumab administration; second visit to be performed approximately 4-6 weeks after evinacumab i.v. administration. About 8 weeks between the 2 visits.
Measured: completion of study, average of 8 weeks
Production rate (PR) of lipoproteins mg/kg/day
First visit to be performed prior to first evinacumab administration; second visit to be performed approximately 4-6 weeks after evinacumab i.v. administration. About 8 weeks between the 2 visits.
Measured: completion of study, average of 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2021
- Study start7 Oct 2016
- Primary completion30 Nov 2016
- Study completion1 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hypercholesterolemia, FamilialStudies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe 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 N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Collaborators
- Regeneron Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Marina Cuchel, MD, PhD, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04722068
- Sponsor's protocol ID
- 1331-Kin
- Status last verified
- May 2021
- First submitted
- 12 Jan 2021
- Last updated
- 17 May 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Reeskamp LF, Millar JS, Wu L, Jansen H, van Harskamp D, Schierbeek H, Gipe DA, Rader DJ, Dallinga-Thie GM, Hovingh GK, Cuchel M. ANGPTL3 Inhibition With Evinacumab Results in Faster Clearance of IDL and LDL apoB in Patients With Homozygous Familial Hypercholesterolemia-Brief Report. Arterioscler Thromb Vasc Biol. 2021 May 5;41(5):1753-1759. doi: 10.1161/ATVBAHA.120.315204. Epub 2021 Mar 11.
PubMed 33691480
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Kinetic Study Investigating Lipoprotein Metabolism Before and After the Administration of REGN1500, an ANGPTL3 Inhibitor, in Patients With Homozygous Familial Hypercholesterolemia. A Sub-study for Subjects Enrolled in the R1500-CL-1331 Clinical Trial
- ClinicalTrials.gov ID
- NCT04722068
- Acronym / sponsor ID
- 1331-Kin
- Conditions
- Hypercholesterolemia, Familial
- Keywords
- Lipid metabolism, Homozygous Familial Hypercholesterolemia, Evinacumab
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Collaborators
- Regeneron Pharmaceuticals
- Study start
- 7 Oct 2016 (actual)
- Primary completion
- 30 Nov 2016 (actual)
- Study completion
- 1 Jan 2021 (actual)
- First posted
- 25 Jan 2021 (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.
To evaluate the lipoprotein kinetics of subjects enrolled in the R1500-CL-1331 clinical trial (NCT02265952) to assess the mechanism by which the evinacumab may affect lipid levels in HoFH subjects.
Detailed description
Subjects enrolled in the R1500-CL-1331 clinical trial (NCT0226595) will participate in a study visit before and after administration of the ANGPTL3 inhibitor REGN1500, evinacumab) for the evaluation of its effect on lipoprotein kinetics.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Volunteers with known HoFH and particpated in R1500-CL-1331 clinical trial | Experimental | Other: Kinetics test |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Men and women 18 years or older at the time of the screening visit
- Diagnosis of HoFH by one of the following:
- Willing to consistently maintain his/her usual diet for the duration of the study
- Willing and able to comply with clinic visits and study-related procedures
- Provide signed informed consent
Key exclusion criteria (26 listed)
- Background medical LMT (if applicable) that has not been stable for at least 4 weeks (6 weeks for fibrates) prior to the screening visit (week -2) (unless participating in the run-in period to stabilize)
- Having undergone lipid apheresis within 4 weeks prior to the screening visit (week -2) (unless participating in the run-in period to wash out)
- Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins. Note: Patients on thyroid replacement therapy can be included if the dosage of thyroxine has been stable for at least 12 weeks prior to screening visit (week -2)
- Use of systemic corticosteroids, unless used as replacement therapy for pituitary/adrenal disease with a stable regiment for at least 6 weeks prior to the screening visit (week -2) Note: Topical, intra-articular, nasal, inhaled, and ophthalmic steroid therapies are not considered as "systemic" and are allowed
- History of a myocardial infarction, unstable angina leading to hospitalization, coronary bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the screening visit (week -2)
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 |
|---|---|---|
| Kinetics testSubjects will participate in two study visits to evaluate lipoprotein kinetics before and after administration of evinacumab. | Other | Volunteers with known HoFH and particpated in R1500-CL-1331 clinical trial |
Primary outcome
Fractional catabolic rate (FCR) pools/day
Time frame: completion of study, average of 8 weeks
First visit to be performed prior to first evinacumab administration; second visit to be performed approximately 4-6 weeks after evinacumab i.v. administration. About 8 weeks between the 2 visits.
Production rate (PR) of lipoproteins mg/kg/day
Time frame: completion of study, average of 8 weeks
First visit to be performed prior to first evinacumab administration; second visit to be performed approximately 4-6 weeks after evinacumab i.v. administration. About 8 weeks between the 2 visits.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2021.
- Derived from this study: PubMed 33691480Reeskamp LF, Millar JS, Wu L, Jansen H, van Harskamp D, Schierbeek H, Gipe DA, Rader DJ, Dallinga-Thie GM, Hovingh GK, Cuchel M. ANGPTL3 Inhibition With Evinacumab Results in Faster Clearance of IDL and LDL apoB in Patients With Homozygous Familial Hypercholesterolemia-Brief Report. Arterioscler Thromb Vasc Biol. 2021 May 5;41(5):1753-1759. doi: 10.1161/ATVBAHA.120.315204. Epub 2021 Mar 11.
- Individual participant dataSharing planned: No
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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.
Marina Cuchel, MD, PhD principal investigator
University of Pennsylvania
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Kinetics test in people with Hypercholesterolemia, Familial, run by University of Pennsylvania at 2 sites in 2 countries.
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 8 inclusion and 26 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: finished, as of the sponsor's last update on 17 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Netherlands. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Fractional catabolic rate (FCR) pools/day (time frame: completion of study, average of 8 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04722068, which also lists 1 linked publication.
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 17 May 2021.