Phase 1 Study of Oral N-Acetylmannosamine in Primary Podocytopathies
Oral ManNAc demonstrated short-term safety and increased plasma SA levels in podocytopathy subjects. Early efficacy signals suggest that proteinuria reduction may correlate with glomerular hyposialylation, identifying a potential treatment biomarker. A phase 2 trial (NCT06664814) is underway to assess long-term outcomes.
Authors
- Petcharat Leoyklang
- Yi Arial Zeng (ORCID: https://orcid.org/0000-0003-1898-8099)
- May Christine V. Malicdan (ORCID: https://orcid.org/0000-0003-1697-6959)
- Kenneth J. Wilkins (ORCID: https://orcid.org/0000-0003-0531-7165)
- Marjan Huizing (ORCID: https://orcid.org/0000-0002-9375-9539)
- Francis Rossignol (ORCID: https://orcid.org/0000-0002-8896-1468)
- Anirban Ganguli
- William A. Gahl
- Thomas A. Cassini
- William D. Figg
- Jodi L. Blake
- Kevin J. O’Brien
- Jeffrey B. Kopp
- Jonathan Bolaños
- Andrea Ashton
Institutions
- National Institutes of Health (US)
- National Human Genome Research Institute (US)
- National Institute of Diabetes and Digestive and Kidney Diseases (US)
- National Institutes of Health Clinical Center (US)
Publication Details
- Journal
- Kidney International Reports
- Published
- 2025-12-31
- DOI
- https://doi.org/10.1016/j.ekir.2025.103758
- Primary Topic
- Renal Diseases and Glomerulopathies
- Type
- article
- Field-Weighted Citation Impact
- 0.00