A Phase 2 interventional study of 2-Hydroxybenzylamine and Placebo in Familial Hypercholesterolemia, sponsored by Vanderbilt University Medical Center. Recruiting at 1 site in United States. Open to participants aged 18 Years to 69 Years. Per ClinicalTrials.gov, last updated 2026-08-07.
Sponsored by Vanderbilt University Medical Center · Phase 2, Interventional, and Prevention
The Investigators will test the hypothesis that 2-HOBA will reduce modification of HDL and LDL and improve HDL function in humans with heterozygous FH. The Investigators plan to first study subjects with Familial Hypercholesterolemia (FH), treating them with 750 mg of 2-HOBA or placebo every 8 hours for 6 weeks.
245 studies on the registry are indexed under Hyperlipoproteinemia Type II; 52 are open to participants now.
This study's planned enrollment of 72 is below the median of 86 across 170 interventional studies indexed under Hyperlipoproteinemia Type II.
Browse Hyperlipoproteinemia Type II studies →Vanderbilt University Medical Center is the lead sponsor of 824 studies on the registry; 164 are open to participants now.
Of its 122 completed or terminated interventional studies of FDA-regulated products, 91 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks.
Drug: 2-Hydroxybenzylamine
Placebo- three tabs TID (po) for 6 weeks.
Other: Placebo
2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks.
Also known as: 2-HOBA
Placebo 250 mg three tabs TID (po) for 6 weeks.
2-HOBA increases HDL cholesterol efflux capacity.
Change in HDL cholesterol efflux capacity will be measured by macrophage cholesterol efflux assay.
Time frame: Baseline to week 6
2-HOBA reduces modification of HDL by Isolevuglandin (Iso-LG).
Measurement of the Iso-LG-lysine lactam by mass spectrometry.
Time frame: Baseline to week 6
2-HOBA reduces modification of HDL by malondialdehyde (MDA).
Measurement of dilysyl-MDA cross-links by mass spectrometry.
Time frame: Baseline to week 6
Change in HDL anti-inflammatory function in an in vitro assay of macrophage cytokine production (IL-1B, TNFa, IL-6).
Measurement of changes in LPS-stimulated macrophage cytokine production(IL-1B, TNFa, IL-6).
Time frame: Baseline to week 6
Change in HDL anti-oxidant function in an in vitro assay of macrophage reactive oxygen species production.
Measurement of changes in H2O2-stimulated macrophage reactive oxygen species
Time frame: Baseline to week 6
Change in HDL microRNA and small noncoding ribonucleic acid (sRNA) composition
HDL microRNA and sRNA will be measured through high-throughput sequencing with quantitative polymerase chain reaction (qPCR) validation.
Time frame: Baseline to week 6
Effects of 2-HOBA on HDL and LDL subpopulation sizes
HDL and LDL subpopulation sizes and particle numbers will be measured by NMR
Time frame: Baseline to week 6
Plan to share: Yes — Individual participant data that underlie the results reported will be made available (including data dictionaries) after de-identification.
Supporting information: Study protocol, Sap, Icf, Analytic code
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Vanderbilt University Medical Center