Using obeticholic acid to treat Alport syndrome

Obeticholic acid as a novel treatment for Alport syndrome

['FUNDING_FELLOWSHIP'] · GEORGETOWN UNIVERSITY · NIH-10894922

This study is looking at whether a medication called obeticholic acid, which is already used for other health issues, can help treat Alport syndrome, a genetic condition that affects the kidneys, by testing it on special mice and seeing how it works alone and with other treatments.

Quick facts

Phase['FUNDING_FELLOWSHIP']
Study typeNih_funding
SexAll
SponsorGEORGETOWN UNIVERSITY (nih funded)
Locations1 site (WASHINGTON, UNITED STATES)
Trial IDNIH-10894922 on ClinicalTrials.gov

What this research studies

This research investigates the potential of obeticholic acid, a drug already approved for other uses, as a new treatment for Alport syndrome, a hereditary condition that leads to chronic kidney disease. The study will utilize a transgenic mouse model to evaluate the effectiveness of obeticholic acid, both alone and in combination with existing treatments like ramipril and bardoxolone methyl. By exploring this novel approach, the research aims to provide new therapeutic options for patients suffering from this rare disease.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with Alport syndrome or related hereditary nephritis who are experiencing chronic kidney disease.

Not a fit: Patients with other forms of kidney disease unrelated to Alport syndrome may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to a new treatment option for patients with Alport syndrome, potentially improving kidney health and delaying the need for kidney transplants.

How similar studies have performed: While the use of obeticholic acid in this context is novel, there have been successful applications of similar approaches in treating chronic kidney disease with other agents.

Where this research is happening

WASHINGTON, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Conditions: Alport syndrome, Alport syndrome (AS, ATS), Alport syndrome-like hereditary nephritis, Alport syndrome-like hereditary nephritis (ASLHN, ASLN), Alport's Syndrome

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.