Striatin genotype-based, mineralocorticoid receptor antagonist-driven clinical trial: study rationale and design

Pharmacogenet Genomics. 2021 Jun 1;31(4):83-88. doi: 10.1097/FPC.0000000000000425.

Abstract

Objectives: In human studies and genetically altered mouse studies, variants in the striatin gene (STRN) are associated with increased blood pressure (BP) and aldosterone on a liberal salt diet. This clinical trial is based on the presumed mechanism for striatin-associated HTN - increased aldosterone. It is designed to determine if participants with the STRN risk alleles will have a greater BP reduction on a liberal salt diet with a specific, mechanism-based therapy - a mineralocorticoid receptor antagonist, eplerenone - as compared with a nonspecific anti-hypertensive therapy - amlodipine.

Methods: One hundred five hypertensive adults with the STRN risk alleles (SNP rs2540923 carriers or rs888083 homozygotes) will be enrolled in a 12-week, double-blind, dose-escalation, clinical trial. After a minimum 2-week washout period and baseline assessment of BP on a liberal salt diet, participants will be randomized to either daily eplerenone or amlodipine. Participants will take daily at-home BP recordings as a safety check. After 4 and 8 weeks of drug therapy, BP will be measured by the study team and medication will be increased, if needed, to achieve a participant goal BP of <140/90 mmHg.Anticipated results We anticipate that STRN risk allele carriers will demonstrate a greater reduction in BP with eplerenone and will require a lower dose of eplerenone to reach goal BP as compared with amlodipine.

Conclusion: This is a proof-of-concept clinical trial. Positive results support the feasibility of performing genetically-defined, mechanistically-driven trials in HTN. Clinically, it would suggest that genetic biomarkers can identify individuals highly responsive to specific treatment.

Trial registration: ClinicalTrials.gov NCT03683069.

Publication types

  • Clinical Trial
  • Research Support, N.I.H., Extramural

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aldosterone / blood
  • Alleles
  • Amlodipine / administration & dosage
  • Animals
  • Blood Pressure / drug effects
  • Blood Pressure / genetics*
  • Calmodulin-Binding Proteins / genetics*
  • Clinical Trials as Topic
  • Eplerenone / administration & dosage*
  • Genetic Predisposition to Disease
  • Humans
  • Hypertension / blood
  • Hypertension / drug therapy*
  • Hypertension / genetics
  • Hypertension / pathology
  • Male
  • Membrane Proteins / genetics*
  • Mice
  • Middle Aged
  • Mineralocorticoid Receptor Antagonists / administration & dosage*
  • Nerve Tissue Proteins / genetics*
  • Polymorphism, Single Nucleotide / genetics
  • Receptors, Mineralocorticoid / genetics
  • Young Adult

Substances

  • Calmodulin-Binding Proteins
  • Membrane Proteins
  • Mineralocorticoid Receptor Antagonists
  • Nerve Tissue Proteins
  • Receptors, Mineralocorticoid
  • STRN protein, human
  • Amlodipine
  • Aldosterone
  • Eplerenone

Associated data

  • ClinicalTrials.gov/NCT03683069