Lipid DisorderPhase 2NCT04128969
Causal Mechanisms in Adolescent Arterial Stiffness
A Phase 2 study testing CS+ and CS- in people with Lipid Disorder and Dyslipidemias, run by Baylor College of Medicine at 1 site in United States.
Trial summary
Hardening of the blood vessels, called arterial stiffness, is a risk factor for future heart disease and its causes are unclear. The proposed study will 1) randomly assign adolescents at high risk of stiffening blood vessels to take a protein supplement called carnitine and study its effects on arterial stiffening and… Read more about this study
- Who is it for?
- Ages 11 to 21 years
- Conditions the study is for
- Lipid Disorder, Dyslipidemias, Aortic Stiffness
- What is being tested?
- CS+ and CS-
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Feb 2020to 31 Aug 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 11 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 11-21 year old adolescents
- males and females
- all ethnicities and races
- fasting serum triglyceride levels over 130 and less than 500 mg/dL
- fasting low density lipoprotein cholesterol (LDL-C) less than 160mg/dL.
Who cannot join
- known seizure disorder
- renal failure patients requiring renal replacement therapy like dialysis or renal transplant
- diabetes mellitus type 1 or 2
- congenital heart disease requiring surgical or catheterization intervention
- current pregnancy or planned pregnancy during the active study participation
- incarceration/institutionalized/wards of the state
- known metabolic disorders that require carnitine therapy
- nonadherence to study protocol during run-in phase defined as possessing 25% more than the expected remainder of placebo supplement pro-rated to the day of assessment
Trial locations and contacts
1 site in 1 country.
About this study
Hardening of the blood vessels, called arterial stiffness, is a risk factor for future heart disease and its causes are unclear. The proposed study will 1) randomly assign adolescents at high risk of stiffening blood vessels to take a protein supplement called carnitine and study its effects on arterial stiffening and 2) study carnitine related genes for their effect on arterial stiffening. The study will definitively establish a role for carnitine action as a cause of stiffening blood vessels and signal a way to treat or prevent stiffening.
Read the detailed description
Aortic stiffness measured in adolescence or adulthood determines current hypertension, predicts future incidence of hypertension, and future atherosclerotic cardiovascular disease (ASCVD) events. International hypertension guidelines list severe aortic stiffness as grounds to intensify anti-hypertensive pharmacotherapy. Mechanisms of arterial stiffness beyond aging and obesity warrant further elucidation. In our preliminary data from adolescents attending weight-loss summer camps arterial stiffness improvement was not associated with weight change but was with change in circulating carnitine. Carnitine influences fatty acid oxidation and carbohydrate metabolism. Carnitine could therefore link to arterial stiffness through insulin resistance which in turn affects cellular tone, vascular fibrosis, modification of lipids or glucose metabolism, and/or advanced glycation end products. This proposal leverages 2 instrumental variable study designs to infer a causal relation between carnitine and arterial stiffness. First, in 90 youth 11-21 years old at risk of arterial stiffening due to high serum triglycerides(TG), we will conduct a mechanistic, double blinded, randomized controlled trial for the effect of 6 months of oral carnitine supplementation (CS+, n=45) versus placebo (CS-, n=45) on aortic stiffness measured as carotid femoral pulse wave velocity (CFPWV); serum fatty acid oxidation biomarkers by metabolomics analysis; insulin resistance as homeostatic model assessment of insulin resistance (HOMA-IR); and TG. Aim 1 is to compare CS+ versus CS- on change in arterial stiffness and monitor adverse events. The hypothesis CS+ is associated with lower arterial stiffening, and CS+ effect is not modified by sex or race/ethnicity. Aim 2 is to compare the effect of CS+ versus CS- on fatty acid metabolism, insulin resistance, and lipids. The hypothesis is that CS+ alters long chain fatty acid beta oxidation, measured as lower long chain acylcarnitines, which in turn improves (HOMA-IR), and in turn decreases TG levels. This causal chain will be disentangled for direct versus indirect effects on CFPWV change. Second, naturally randomly assorted carnitine single nucleotide polymorphisms (SNPs) noted above will be used to characterize the relationship of carnitine to arterial stiffness and stratify the effectiveness of CS+.Aim 3a is to obtain the direct effect of carnitine on arterial stiffness using Mendelian randomization of SNPs associated with serum carnitine as instrumental variables with the hypothesis these variant SNPs are associated with lower arterial stiffness, supporting a causal inference. Aim 3b is to identify effect modification of CS+ vs CS- on arterial stiffness by examining if a carnitine genetic risk score will modify the effect of CS+ on change in arterial stiffness. This proposal with 2 instrumental variable projects would evaluate a causal role for carnitine in arterial stiffness at a point when the life course trajectory to hypertension can be modified. The study will also investigate the role of carnitine in insulin resistance and dyslipidemia at this same age, which may serve as grounds for future therapeutic clinical trials. Discovering genetically mediated causes of arterial stiffness or other outcomes may facilitate targeting of future therapies on susceptible youth before atherosclerotic changes are irreversible.
- Conditions
- Lipid Disorder, Dyslipidemias, Aortic Stiffness, Insulin Resistance Syndrome, Metabolic Syndrome, Pediatric Obesity
- Being tested
- CS+, CS-
- Keywords
- dyslipidemia, arterial stiffness, pediatric obesity, metabolic syndrome, insulin resistance
- Medical subject headings
- Lipid Metabolism Disorders, Dyslipidemias, Metabolic Syndrome, Pediatric Obesity, Insulin Resistance
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CS+Dietary supplement
Oral carnitine supplementation
CS-Dietary supplement
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Carnitine supplementation (CS+) | Experimental | Carnitine supplementation in liquid form, sugar free. Receives: Dietary Supplement: CS+ |
| Placebo (CS-) | Placebo comparator | Placebo comparator liquid similar in appearance and taste to CS+. Receives: Dietary Supplement: CS- |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Carotid Femoral Pulse Wave Velocity
Carotid Femoral Pulse Wave Velocity will be measured noninvasively using applanation tonometry.
Measured: 6 months
Secondary outcomes (2)
Change in Fasting Triglyceride
Fasting serum triglycerides to be measured using conventional techniques.
Measured: 6 months
Change in Insulin Resistance
Insulin resistance will be assessed using fasting serum glucose and fasting serum insulin to calculate homeostatic model assessment of insulin resistance.
Measured: 6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2019
- Study start1 Feb 2020
- Primary completion1 Jun 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lipid DisorderStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Justin Zachariah, Baylor College of Medicine
- Investigators
- Justin P Zachariah, MD MPH, Principal investigator, Study Principal Investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04128969
- Sponsor's protocol ID
- H-45557
- Other IDs
- R01HL148217
- Status last verified
- Dec 2025
- First submitted
- 11 Oct 2019
- Last updated
- 10 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Causal Mechanisms in Adolescent Arterial Stiffness
- ClinicalTrials.gov ID
- NCT04128969
- Acronym / sponsor ID
- H-45557
- Conditions
- Lipid Disorder, Dyslipidemias, Aortic Stiffness, Insulin Resistance Syndrome, Metabolic Syndrome, Pediatric Obesity
- Keywords
- dyslipidemia, arterial stiffness, pediatric obesity, metabolic syndrome, insulin resistance
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 1 Feb 2020 (actual)
- Primary completion
- 1 Jun 2026 (estimated)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 16 Oct 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.
Hardening of the blood vessels, called arterial stiffness, is a risk factor for future heart disease and its causes are unclear. The proposed study will 1) randomly assign adolescents at high risk of stiffening blood vessels to take a protein supplement called carnitine and study its effects on arterial stiffening and 2) study carnitine related genes for their effect on arterial stiffening. The study will definitively establish a role for carnitine action as a cause of stiffening blood vessels and signal a way to treat or prevent stiffening.
Detailed description
Aortic stiffness measured in adolescence or adulthood determines current hypertension, predicts future incidence of hypertension, and future atherosclerotic cardiovascular disease (ASCVD) events. International hypertension guidelines list severe aortic stiffness as grounds to intensify anti-hypertensive pharmacotherapy. Mechanisms of arterial stiffness beyond aging and obesity warrant further elucidation. In our preliminary data from adolescents attending weight-loss summer camps arterial stiffness improvement was not associated with weight change but was with change in circulating carnitine. Carnitine influences fatty acid oxidation and carbohydrate metabolism. Carnitine could therefore link to arterial stiffness through insulin resistance which in turn affects cellular tone, vascular fibrosis, modification of lipids or glucose metabolism, and/or advanced glycation end products. This proposal leverages 2 instrumental variable study designs to infer a causal relation between carnitine and arterial stiffness. First, in 90 youth 11-21 years old at risk of arterial stiffening due to high serum triglycerides(TG), we will conduct a mechanistic, double blinded, randomized controlled trial for the effect of 6 months of oral carnitine supplementation (CS+, n=45) versus placebo (CS-, n=45) on aortic stiffness measured as carotid femoral pulse wave velocity (CFPWV); serum fatty acid oxidation biomarkers by metabolomics analysis; insulin resistance as homeostatic model assessment of insulin resistance (HOMA-IR); and TG. Aim 1 is to compare CS+ versus CS- on change in arterial stiffness and monitor adverse events. The hypothesis CS+ is associated with lower arterial stiffening, and CS+ effect is not modified by sex or race/ethnicity. Aim 2 is to compare the effect of CS+ versus CS- on fatty acid metabolism, insulin resistance, and lipids. The hypothesis is that CS+ alters long chain fatty acid beta oxidation, measured as lower long chain acylcarnitines, which in turn improves (HOMA-IR), and in turn decreases TG levels. This causal chain will be disentangled for direct versus indirect effects on CFPWV change. Second, naturally randomly assorted carnitine single nucleotide polymorphisms (SNPs) noted above will be used to characterize the relationship of carnitine to arterial stiffness and stratify the effectiveness of CS+.Aim 3a is to obtain the direct effect of carnitine on arterial stiffness using Mendelian randomization of SNPs associated with serum carnitine as instrumental variables with the hypothesis these variant SNPs are associated with lower arterial stiffness, supporting a causal inference. Aim 3b is to identify effect modification of CS+ vs CS- on arterial stiffness by examining if a carnitine genetic risk score will modify the effect of CS+ on change in arterial stiffness. This proposal with 2 instrumental variable projects would evaluate a causal role for carnitine in arterial stiffness at a point when the life course trajectory to hypertension can be modified. The study will also investigate the role of carnitine in insulin resistance and dyslipidemia at this same age, which may serve as grounds for future therapeutic clinical trials. Discovering genetically mediated causes of arterial stiffness or other outcomes may facilitate targeting of future therapies on susceptible youth before atherosclerotic changes are irreversible.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Carnitine supplementation (CS+) | Experimental | Dietary Supplement: CS+Carnitine supplementation in liquid form, sugar free. |
| Placebo (CS-) | Placebo comparator | Dietary Supplement: CS-Placebo comparator liquid similar in appearance and taste to CS+. |
Who may qualify
- Ages
- 11 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 11-21 year old adolescents
- males and females
- all ethnicities and races
- fasting serum triglyceride levels over 130 and less than 500 mg/dL
- fasting low density lipoprotein cholesterol (LDL-C) less than 160mg/dL.
Key exclusion criteria (8 listed)
- known seizure disorder
- renal failure patients requiring renal replacement therapy like dialysis or renal transplant
- diabetes mellitus type 1 or 2
- congenital heart disease requiring surgical or catheterization intervention
- current pregnancy or planned pregnancy during the active study participation
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 |
|---|---|---|
| CS+Oral carnitine supplementation | Dietary supplement | Carnitine supplementation (CS+) |
| CS-Placebo | Dietary supplement | Placebo (CS-) |
Primary outcome
Change in Carotid Femoral Pulse Wave Velocity
Time frame: 6 months
Carotid Femoral Pulse Wave Velocity will be measured noninvasively using applanation tonometry.
2 secondary outcomes
Change in Fasting Triglyceride
Time frame: 6 months
Change in Insulin Resistance
Time frame: 6 month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2025.
- Individual participant dataSharing planned: No
Related trials for Lipid Disorder
RecruitingObservational study
Late Subclinical Cardiovascular Disease in Testicular Cancer Survivors
Testicular Cancer, Survivorship
Not yet recruiting
Plasma Lipids-Dependent Vitamin E Metabolism During Dynamic Hyperlipidemia
Lipid Metabolism Disorders
RecruitingPhase 3
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Not yet recruitingPhase 1/2
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Heterozygous Familial Hypercholesterolemia (HeFH)
Not yet recruiting
Metabolic Effects of an Ophiocordyceps Sinensis- and CoQ10-Based Formulation in Mild-to-Moderate Hypercholesterolemia
Hypercholesterolemia, Lipid Metabolism Disorders
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.
Justin P Zachariah, MD MPH principal investigator
Study Principal Investigator
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CS+ and CS- in people with Lipid Disorder and Dyslipidemias, run by Baylor College of Medicine 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 5 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 10 Dec 2025. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
This study is still under way. Results are not available in the source record yet.
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?
Change in Carotid Femoral Pulse Wave Velocity (time frame: 6 months). 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 10 Dec 2025; AzTrials last refreshed its copy on 2 Nov 2026.