# A randomized, double-blind, multi-center comparative effectiveness study of spironolactone versus doxycycline hyclate for the treatment of acne in women

> **NIH NIH U01** · UNIVERSITY OF PENNSYLVANIA · 2022 · $1,029,338

## Abstract

Project Summary/Abstract
 Acne is one of the most common diseases worldwide, affecting 85% of adolescents and often persisting
into adulthood. Acne is responsible for a greater global burden of disease than psoriasis, cellulitis, and
melanoma. Although mild acne can usually be managed with topical medications, moderate to severe acne
often requires treatment with systemic medications such as oral antibiotics, spironolactone, and isotretinoin.
While these medications are a standard part of care, there are very few randomized clinical trials showing
efficacy and none that show comparative effectiveness between these commonly used treatment options. The
Institute of Medicine has identified this area as one of the top priorities for comparative effectiveness research.
In addition, little is known about the effects of these different treatments on the microbiome.
 Previous work by our group has shown oral antibiotics are the most common systemic medication used in
the treatment of acne and patients frequently use them for more than one year. In fact, dermatologists
prescribe more antibiotics per capita than any other specialty. However, prolonged antibiotic use may be
associated with a variety of adverse outcomes including bacterial antibiotic resistance, pharyngitis, collagen
vascular illnesses, inflammatory bowel disease, and breast and colon cancer. As a result, there is growing
international interest and attention specifically from the Centers for Disease Control regarding improving
antibiotic stewardship in the outpatient setting and multiple clinical guidelines for acne have recommended
reducing the use of antibiotics through the use of non-antimicrobial therapies and by limiting the duration of
therapy.
 For women with moderate to severe acne, spironolactone may represent an effective, well-tolerated
therapeutic alternative to oral antibiotics. Originally developed as a potassium-sparing diuretic, for many years
it has also been used off-label for acne due to its potential impact on hormonal pathogenesis of this disease.
However, despite expert opinion supporting the use of spironolactone in the treatment of acne, its use remains
relatively uncommon and clinical evidence demonstrating the effectiveness of spironolactone is limited to
small, often retrospective studies. Trials comparing the effectiveness of spironolactone to that of other
medications such as oral antibiotics are lacking.
 In Specific Aim 1, we will conduct a double-blind randomized controlled non-inferiority comparative
effectiveness study of spironolactone versus doxycycline for women with acne. Since oral tetracycline-class
antibiotics like doxycycline are currently the most common systemic medication prescribed for acne, the results
of this trial will have substantial implications for the treatment of acne. In Specific Aim 2, we will evaluate the
impact of spironolactone versus doxycycline treatment on the microbiome, which will provide valuable insights
regarding the relativ...

## Key facts

- **NIH application ID:** 10470869
- **Project number:** 5U01AR079752-02
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** David Joel Margolis
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $1,029,338
- **Award type:** 5
- **Project period:** 2021-08-17 → 2027-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10470869

## Citation

> US National Institutes of Health, RePORTER application 10470869, A randomized, double-blind, multi-center comparative effectiveness study of spironolactone versus doxycycline hyclate for the treatment of acne in women (5U01AR079752-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10470869. Licensed CC0.

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