Cholesterol, HDLNCT01782027
Mendelian Reverse Cholesterol Transport Study
A study testing 3H-cholesterol bound to albumin in people with Cholesterol, HDL and Lipid Metabolism, Inborn Errors, run by University of Pennsylvania at 1 site in United States.
Trial summary
The purpose of this study is to investigate the use of radiolabeled particulate cholesterol administered intravenously in association with albumin, as a method to study reverse cholesterol transport (RCT) in people carrying mutations in genes known to affect high density lipoprotein (HDL) metabolism by analyzing… Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Cholesterol, HDL, Lipid Metabolism, Inborn Errors, Tangier Disease
- What is being tested?
- 3H-cholesterol bound to albumin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2012to 4 Sep 2024
- 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 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Men and women between the ages of 18 and 75
- Subjects must be:
- Carriers of functional mutations of genes encoding proteins affecting HDL metabolism;
- Healthy control subjects with HDL cholesterol levels within the normal range of the lab where screening tests are run, or at the discretion of the investigator, and matched for gender, race, age (± 5 years) to the patients.
- Negative screening pregnancy test if female of child bearing potential (females of child-bearing potential must be following a medically accepted form of contraception)
- Subjects must be able to comprehend and willing to provide a signed IRB approved Informed Consent Form.
- Subjects must be willing and able to comply with all study-related procedures.
Who cannot join
- Known cardiovascular disease, including coronary disease, cerebrovascular disease, or peripheral vascular disease (control subjects only)
- History of diabetes mellitus or fasting glucose > 126 mg/dL at the screening visit (control subjects only).
- Any current, unstable endocrine disease as assessed by collection of medical history during screening. Subjects with rare Mendelian disorders with thyroid disease that is well controlled by stable treatment may be considered for enrollment at the discretion of the principal investigator
- History of previous malignancy, other than basal cell or squamous cell carcinoma of the skin, from which the patient has been disease free for less than 5 years as assessed by collection of medical history during screening
- Current diagnosis of anemia as assessed by collection of medical history during screening or hemoglobin less than 10 g/dL as evaluated during safety lab at screening
- History of kidney disease or chronic renal insufficiency, as defined as estimated glomerular filtration rate (eGFR) < 60 ml/min/1.73m2 in control subjects and patients with other disorders of HDL metabolism and eGFR < 30 ml/min/1.73m2 in subjects with Lecithin-Cholesterol Acyltransferase (LCAT) deficiency.
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- Any active rheumatologic, pulmonary, or dermatologic disease or inflammatory condition as assessed by collection of medical history during screening, and judged by the investigator to be a major condition.
- Sustained uncontrolled hypertension (Systolic >160 mm Hg and/or Diastolic BP >100 mmHg) at screening. Blood pressure may be re-tested twice after initial assessment in the supine position at five minute intervals (for a total of 3 blood pressure assessments). The pressure elevation is considered sustained if either the systolic or the diastolic pressure values are outside the stated limits for all three assessments
- Use of warfarin, or any known coagulopathy and /or elevated prothrombin time/partial thromboplastin time (PT/PTT) >1.5 x upper limit of normal (ULN)
- Self-reported history of human immunodeficiency virus (HIV) positive
- History of previous organ transplantation, as assessed by collection of medical history during screening
- Clinical evidence of liver disease or liver injury as indicated by abnormal liver function tests such as alanine transaminase (ALT) or aspartate transaminase (AST) > 1.5x ULN, or self-reported history of positive Hepatitis B or Hepatitis C test result
- Any surgical procedure that occurred within the previous 3 months of the screening visit, as assessed by collection of medical history during screening, and judged by the investigator to be a major procedure.
- History of drug abuse (< 1 year), as assessed by collection of medical history during screening procedures
- Regular abuse of alcoholic beverages (> 2 drinks/day), as assessed by collection of medical history during screening procedures
- Self-reported participation in an investigational drug study within 6 weeks prior to the screening visit
- Serious or unstable medical or psychological conditions that, in the opinion of the investigator, would compromise the subject's safety or successful participation in the study will be excluded.
- Use of lipid lowering drugs expected to affect RCT (e.g. fibrates) within the 6 weeks prior to dosing or during the study, as assessed by collection of medical history during screening and concomitant medication checks at each study visit. Use of statins (stable dose for at least 30 days) is permitted.
- Male subjects who plan to conceive a child within 3 months of the conclusion of the study.
- Women who are pregnant or lactating or who are planning to become pregnant
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Validation Study Evaluating the Use of 3H-Cholesterol Bound to Albumin as a Method to Assess Reverse Cholesterol Transport in Subjects With Monogenic Diseases Affecting HDL Metabolism
The purpose of this study is to investigate the use of radiolabeled particulate cholesterol administered intravenously in association with albumin, as a method to study reverse cholesterol transport (RCT) in people carrying mutations in genes known to affect high density lipoprotein (HDL) metabolism by analyzing changes in the tracer activity in total plasma, lipoproteins fractions and feces.
Read the detailed description
The study will use 3H-cholesterol bound to albumin (particulate cholesterol) to assess the ability of HDL to transport cholesterol from the periphery to the liver to be eliminated. This process is called reverse cholesterol transport (RCT) and is one of the main mechanisms by which HDL protect against atherosclerotic cardiovascular disease. Mutations in some of the genes affecting HDL metabolism, may results in changes in RCT. The validation of a method assessing RCT is important for the development of new drugs which affect RCT and may result in useful treatments for atherosclerosis.
Subjects carrying mutations in genes known to affect HDL metabolism and healthy controls will be enrolled in the study. Changes in the tracer activity in total plasma, lipoproteins fractions and feces will be analyzed following the intravenous administration of radiolabeled particulate cholesterol.
- Conditions
- Cholesterol, HDL, Lipid Metabolism, Inborn Errors, Tangier Disease, LCAT Deficiency, Cholesteryl Ester Transfer Protein (CETP) Deficiency
- Being tested
- 3H-cholesterol bound to albumin
- Keywords
- healthy controls, Tangier Disease, Adenosine triphosphate-binding cassette transporter 1 (ABCA1), apoA-I, LCAT deficiency, Scavenger receptor BI (SRBI) deficiency, CETP deficiency
- Medical subject headings
- Lipid Metabolism, Inborn Errors, Tangier Disease, Lecithin Cholesterol Acyltransferase Deficiency, Cholesteryl Ester Transfer Protein Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3H-cholesterol bound to albuminDrug
Also called particulate cholesterol
up to 100 uCi of [3H]-cholesterol (containing approximately 0.2 mg of cholesterol) mixed with a solution containing human serum albumin will be administered as an intravenous bolus injection
| Study group | Role | What they receive |
|---|---|---|
| 3H-cholesterol | Experimental | Not described Receives: Drug: 3H-cholesterol bound to albumin |
What is being measured
The primary outcome is the main question the study is designed to answer.
determination of 3H cholesterol in plasma and lipoproteins
Measured: up to 192 hr
Secondary outcomes (1)
determination of 3H cholesterol and its metabolites in red blood cells over time
Measured: up to 192 hr
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2013 (estimated)
- Study startOct 2012
- Primary completion4 Sep 2024
- Study completion4 Sep 2024
Why it stopped: Lack of funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cholesterol, HDLStudies 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
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Marina Cuchel, MD, PhD, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01782027
- Sponsor's protocol ID
- 815075
- Status last verified
- Sep 2024
- First submitted
- 30 Jan 2013
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Validation Study Evaluating the Use of 3H-Cholesterol Bound to Albumin as a Method to Assess Reverse Cholesterol Transport in Subjects With Monogenic Diseases Affecting HDL Metabolism
- ClinicalTrials.gov ID
- NCT01782027
- Acronym / sponsor ID
- 815075
- Conditions
- Cholesterol, HDL, Lipid Metabolism, Inborn Errors, Tangier Disease, LCAT Deficiency, Cholesteryl Ester Transfer Protein (CETP) Deficiency
- Keywords
- healthy controls, Tangier Disease, Adenosine triphosphate-binding cassette transporter 1 (ABCA1), apoA-I, LCAT deficiency, Scavenger receptor BI (SRBI) deficiency, CETP deficiency
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of funding
- Enrolment
- 17 participants (actual)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Study start
- Oct 2012
- Primary completion
- 4 Sep 2024 (actual)
- Study completion
- 4 Sep 2024 (actual)
- First posted
- 1 Feb 2013 (estimated)
- 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.
The purpose of this study is to investigate the use of radiolabeled particulate cholesterol administered intravenously in association with albumin, as a method to study reverse cholesterol transport (RCT) in people carrying mutations in genes known to affect high density lipoprotein (HDL) metabolism by analyzing changes in the tracer activity in total plasma, lipoproteins fractions and feces.
Detailed description
The study will use 3H-cholesterol bound to albumin (particulate cholesterol) to assess the ability of HDL to transport cholesterol from the periphery to the liver to be eliminated. This process is called reverse cholesterol transport (RCT) and is one of the main mechanisms by which HDL protect against atherosclerotic cardiovascular disease. Mutations in some of the genes affecting HDL metabolism, may results in changes in RCT. The validation of a method assessing RCT is important for the development of new drugs which affect RCT and may result in useful treatments for atherosclerosis.
Subjects carrying mutations in genes known to affect HDL metabolism and healthy controls will be enrolled in the study. Changes in the tracer activity in total plasma, lipoproteins fractions and feces will be analyzed following the intravenous administration of radiolabeled particulate cholesterol.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 3H-cholesterol | Experimental | Drug: 3H-cholesterol bound to albumin |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Men and women between the ages of 18 and 75
- Subjects must be:
- Negative screening pregnancy test if female of child bearing potential (females of child-bearing potential must be following a medically accepted form of contraception)
- Subjects must be able to comprehend and willing to provide a signed IRB approved Informed Consent Form.
- Subjects must be willing and able to comply with all study-related procedures.
Key exclusion criteria (20 listed)
- Known cardiovascular disease, including coronary disease, cerebrovascular disease, or peripheral vascular disease (control subjects only)
- History of diabetes mellitus or fasting glucose > 126 mg/dL at the screening visit (control subjects only).
- Any current, unstable endocrine disease as assessed by collection of medical history during screening. Subjects with rare Mendelian disorders with thyroid disease that is well controlled by stable treatment may be considered for enrollment at the discretion of the principal investigator
- History of previous malignancy, other than basal cell or squamous cell carcinoma of the skin, from which the patient has been disease free for less than 5 years as assessed by collection of medical history during screening
- Current diagnosis of anemia as assessed by collection of medical history during screening or hemoglobin less than 10 g/dL as evaluated during safety lab at screening
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 |
|---|---|---|
| 3H-cholesterol bound to albuminAlso: particulate cholesterolup to 100 uCi of [3H]-cholesterol (containing approximately 0.2 mg of cholesterol) mixed with a solution containing human serum albumin will be administered as an intravenous bolus injection | Drug | 3H-cholesterol |
Primary outcome
determination of 3H cholesterol in plasma and lipoproteins
Time frame: up to 192 hr
1 secondary outcome
determination of 3H cholesterol and its metabolites in red blood cells over time
Time frame: up to 192 hr
Results and safety
The study was stopped early (reason given: Lack of funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
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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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing 3H-cholesterol bound to albumin in people with Cholesterol, HDL and Lipid Metabolism, Inborn Errors, run by University of Pennsylvania at 1 site in United States.
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 7 inclusion and 20 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: stopped early, as of the sponsor's last update on 19 Sep 2024. 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 United States. 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 was stopped early (reason given: Lack of funding). Results are not available in the source record.
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?
determination of 3H cholesterol in plasma and lipoproteins (time frame: up to 192 hr). 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 19 Sep 2024.