An Open-Label Pilot Study of Adrenocorticotrophic Hormone in the Treatment of IgA Nephropathy at High Risk of Progression

Ladan Zand, Pietro Canetta, Richard Lafayette, Nabeel Aslam, Novak Jan, Sanjeev Sethi, Fernando C. Fervenza

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3 Scopus citations


Introduction: IgA nephropathy (IgAN) is the most common glomerulonephritis with high risk of progression to end-stage renal disease in patients with proteinuria >1 g/24 hours. There are no known effective treatments in patients with IgAN. Methods: We conducted a prospective open-label pilot study in patients with IgAN using adrenocorticotrophic hormone (ACTH) (Acthar Gel, Mallinckrodt Pharmaceuticals, Bedminster, NJ) at a dosage of 80 units subcutaneously twice weekly for a total of 6 months and followed patients for a total of 12 months. Patients had to have urinary protein >1 g/24 hours despite adequate renin-angiotensin-aldosterone system (RAAS) blockade and estimated glomerular filtration rate (eGFR) >30 ml/min at enrollment. Results: A total of 19 patients were recruited and followed for 1 year. At baseline, the mean age was 34.9 ± 10.5 years with 11 men and 8 women, and 14 Caucasian and 5 Asian individuals. At 12 months, there was a statistically significant decline in 24-hour urinary protein from 2.6 to 1.3 g (P = 0.007) and significant increase in serum albumin (3.79 to 3.93, P = 0.02). There was no significant change in eGFR (65.5 to 61.1 ml/min, P = 0.1). There were 0 complete remissions and 8 partial remissions (42%). There were a total of 6 infections: 2 were viral and 4 required antibiotic therapy (2 sinusitis, 1 pneumonia, 1 otitis media). The most common adverse events included acne, hot flashes, soreness, and anxiety. Conclusion: In summary, patients with IgAN with >1 g/24-hour urinary protein and eGFR >30 ml/min had a significant reduction in 24-hour urinary protein with stable eGFR at 12-month follow-up after being treated with 6 months of ACTH.

Original languageEnglish (US)
Pages (from-to)58-65
Number of pages8
JournalKidney International Reports
Issue number1
StatePublished - Jan 2020
Externally publishedYes

Bibliographical note

Funding Information:
JN reports his current funding from the National Institutes of Health and sponsored research agreements with Retrophin and Alexion, is a co-inventor on US patent applications US08/230,473 and US14/318,082 (assigned to University of Alabama at Birmingham Research Foundation), and is a co-founder of Reliant Glycosciences, LLC. RL has received advisory fees from Mallinckrodt, Omeros, Calliditas, Retrophin, and Otsuka, Inc, and has received research grant support from Mallinckrodt Medical Inc, Apellis, Omeros, Calliditas, and Otsuka. FCF received an unrestricted research grant from Mallinckrodt Medical Inc. All the other authors declared no competing interests.

Funding Information:
The authors gratefully acknowledge technical assistance with ELISA by Stacy Hall. This was an investigator-initiated study and was supported by an unrestricted research grant from Mallinckrodt Pharmaceuticals , Bedminster, NJ.

Publisher Copyright:
© 2019 International Society of Nephrology


  • ACTH
  • IgA nephropathy
  • proteinuria

PubMed: MeSH publication types

  • Journal Article


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