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NAFLDPhase 1NCT05979389

Bicalutamide Therapy in Young Women With NAFLD and PCOS

A Phase 1 study testing Bicalutamide 50 mg in people with NAFLD and PCOS, run by University of California, San Francisco at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

Nonalcoholic steatohepatitis (NASH), or fat-related liver inflammation and scarring is projected to be the leading cause of cirrhosis in the United States (U.S.) within the next few years. Women are at disproportionate risk for NASH, with approximately 15 million U.S. women affected. Read more about this study

Who is it for?
Ages 18 to 42 years, women only
Conditions the study is for
NAFLD, PCOS
What is being tested?
Bicalutamide 50 mg
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
50 participants
Where
1 siteUnited States
Study dates
14 Feb 2024to Aug 2028
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 42 years
  • Women only
  • No healthy volunteers
  • Adult

Who may be able to join

  • Women aged 18-42 years with hyperandrogenic PCOS
  • NASH identified on liver biopsy or probable NASH on transient elastography- controlled attenuation parameter (TE-CAP) with cutoffs defined as CAP score ≥270 decibel/m and TE score > 7.0 kPA or alanine aminotransferase ≥40 U/L).

Who cannot join

  • Uncontrolled diabetes
  • Alcohol consumption >2 drinks per day for at least 3 consecutive months over the previous 5 years
  • Other chronic liver disease (i.e. hepatitis B virus, hepatitis C virus, autoimmune hepatitis) or cirrhosis from any cause
  • Recent or planned upcoming weight reduction surgery within five years of diagnosis of biopsy-confirmed NASH
  • HIV infection
  • Drugs associated with fatty liver (i.e. amiodarone, methotrexate, systemic glucocorticoids, tamoxifen, anabolic steroids, valproic acid) for more than 4 weeks prior to baseline or during study
Show all 9Show fewer
  • Recent, current, or planned upcoming pregnancy or current perimenopausal status
  • Renal impairment (glomerular filtration rate <45 ml/min/1.73m or potassium levels > 5.0 mmol/L)
  • Androgen receptor antagonist use (i.e. spironolactone or flutamide) for more than 3 months within one year prior to baseline

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

United States1 site, 1 recruiting
  • University of California San Francisco

    San Francisco, California, 94143

    Lily Carbullido, 415-502-3725, MyLiverHealth@ucsf.edu

    Monika Sarkar, M.D.

    Recruiting

Other trials in United States

About this study

Official title: Pilot Trial of Bicalutamide Versus Placebo in Reproductive-Aged Women With Nonalcoholic Fatty Liver Disease (NAFLD) and Polycystic Ovary Syndrome (PCOS)

Nonalcoholic steatohepatitis (NASH), or fat-related liver inflammation and scarring is projected to be the leading cause of cirrhosis in the United States (U.S.) within the next few years. Women are at disproportionate risk for NASH, with approximately 15 million U.S. women affected. There is an urgent need to understand risk factors for NASH and its progression in women, and sex hormones may provide a missing link. This study will study the contribution of androgens to liver injury and progression in PCOS and mechanistic role of dysregulated lipid metabolism and visceral adiposity in this process. Such findings will provide the rationale for future efficacy studies evaluating selective androgen receptor (AR) antagonism for NASH in PCOS, or alternatively, the need to directly target visceral adiposity or lipid-specific pathways as part of a precision medicine approach to halt fibrosis progression in the nearly 5 million young women with PCOS and NAFLD in the U.S., who remain at increased risk for early onset and progressive liver disease.

Read the detailed description

This is a single center, double-blind, placebo-controlled, randomized, (1:1) parallel group pilot clinical trial of bicalutamide in women with either biopsy-proven or believed NAFLD receiving 6 months of bicalutamide or placebo. 50 women are targeted for enrollment. Each participant will be administered a single dose of bicalutamide or placebo once daily for a total of 6 months. In person evaluations will take place at Month 1, 2, 3, 4, 5, and 6. There will be a telephone follow up visit within 1 month of end of treatment. This is a pilot clinical trial that is largely feasibility focused. Study outcomes will include:

  • Change in liver stiffness on Magnetic Resonance Elastography (MRE)
  • Change in hepatic steatosis by Magnetic Resonance Proton Density Fat Fraction (PDFF)
  • Change in visceral adipose tissue (VAT) volume by Magnetic Resonance Imaging (MRI)
  • Change in NASH histology as assessed by the continuous NAFLD activity score (NAS), which measures different components of NASH on liver biopsy.
  • Biochemical endpoints: serum lipids \& HOMA-IR
  • Feasibility outcomes including Rates (and reasons) for the following: a) % women that decline/women contacted for study inclusion (i.e. concern regarding randomization to placebo) b) % women enrolled/women screened (i.e. exclusion criteria too narrow), c) study dropout (i.e. medication side effects, too frequent study visits, and/or phlebotomy)
Conditions
NAFLD, PCOS
Being tested
Bicalutamide 50 mg, Placebo
Medical subject headings
Non-alcoholic Fatty Liver Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Bicalutamide 50 mgDrug

    Also called Casodex

    Bicalutamide capsules will be prepared from U.S. Pharmacopeia grade powder at a dose of 50 mg

  • PlaceboDrug

    Matching placebo capsules of the same color, mass, and appearance to the bicalutamide capsules will be filled using microcrystalline cellulose powder.

Study groups in NCT05979389
Study groupRoleWhat they receive
BicalutamideExperimental50 mg capsule administered orally once daily for 6 months
Receives: Drug: Bicalutamide 50 mg
PlaceboPlacebo comparatorMatching placebo capsule administered orally once daily for 6 months
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in liver stiffness on Magnetic Resonance Elastography (MRE)

    The investigators will assess for change in the MRE quantified liver stiffness in kilopascals (kPA)

    Measured: Baseline and 6 months

Secondary outcomes (4)
  1. Change in hepatic steatosis by Magnetic Resonance Proton Density Fat Fraction (PDFF)

    The investigators will assess for percent change in fat fraction by MRI-PDFF

    Measured: Baseline and 6 months

  2. Change in visceral adipose tissue (VAT) volume by Magnetic Resonance Imaging (MRI)

    The investigators will assess for percent change in VAT as quantified by MRI

    Measured: Baseline and 6 months

  3. Change HOMA-IR (Homeostatic model assessment (HOMA) for insulin resistance (IR))

    The investigators will assess change in continuous measures of HOMA-IR as insulin resistance is known to contribute to NASH progression

    Measured: Baseline and 6 months

  4. Change in the NAFLD Activity Score (NAS) on a scale from 0 (low activity) to 8 (high activity)

    The investigators will assess for change in this histologic scoring system of NASH as a continuous measure among women willing to undergo end of treatment biopsy (not required).

    Measured: Baseline and 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Aug 2023
  2. Study start14 Feb 2024
  3. Primary completionAug 2028 (estimated)
  4. Study completionAug 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Other
Allocation
Randomised
Intervention model
Parallel groups
Model description
The following treatment regimens will be used: * Experimental treatment - bicalutamide, 50 mg once daily * Placebo or Comparator - one capsule, once daily
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05979389
Sponsor's protocol ID
23-39274
Other IDs
R01DK134633-01A1
Status last verified
May 2026
First submitted
28 Jul 2023
Last updated
29 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Maldonado SS, Grab J, Wang CW, Huddleston H, Cedars M, Sarkar M. Polycystic ovary syndrome is associated with nonalcoholic steatohepatitis in women of reproductive age. Hepatol Commun. 2022 Oct;6(10):2634-2639. doi: 10.1002/hep4.2039. Epub 2022 Jul 21.

    PubMed 35861548
  • Sarkar MA, Suzuki A, Abdelmalek MF, Yates KP, Wilson LA, Bass NM, Gill R, Cedars M, Terrault N; NASH Clinical Research Network. Testosterone is Associated With Nonalcoholic Steatohepatitis and Fibrosis in Premenopausal Women With NAFLD. Clin Gastroenterol Hepatol. 2021 Jun;19(6):1267-1274.e1. doi: 10.1016/j.cgh.2020.09.045. Epub 2020 Oct 1.

    PubMed 33010412
  • Sanchez-Garrido MA, Tena-Sempere M. Metabolic dysfunction in polycystic ovary syndrome: Pathogenic role of androgen excess and potential therapeutic strategies. Mol Metab. 2020 May;35:100937. doi: 10.1016/j.molmet.2020.01.001. Epub 2020 Feb 5.

    PubMed 32244180
  • O'Reilly MW, Kempegowda P, Walsh M, Taylor AE, Manolopoulos KN, Allwood JW, Semple RK, Hebenstreit D, Dunn WB, Tomlinson JW, Arlt W. AKR1C3-Mediated Adipose Androgen Generation Drives Lipotoxicity in Women With Polycystic Ovary Syndrome. J Clin Endocrinol Metab. 2017 Sep 1;102(9):3327-3339. doi: 10.1210/jc.2017-00947.

    PubMed 28645211
  • Dieudonne MN, Pecquery R, Boumediene A, Leneveu MC, Giudicelli Y. Androgen receptors in human preadipocytes and adipocytes: regional specificities and regulation by sex steroids. Am J Physiol. 1998 Jun;274(6):C1645-52. doi: 10.1152/ajpcell.1998.274.6.C1645.

    PubMed 9611130
  • Gambineri A, Patton L, Vaccina A, Cacciari M, Morselli-Labate AM, Cavazza C, Pagotto U, Pasquali R. Treatment with flutamide, metformin, and their combination added to a hypocaloric diet in overweight-obese women with polycystic ovary syndrome: a randomized, 12-month, placebo-controlled study. J Clin Endocrinol Metab. 2006 Oct;91(10):3970-80. doi: 10.1210/jc.2005-2250. Epub 2006 Jul 25.

    PubMed 16868063
  • Cusi K. Role of obesity and lipotoxicity in the development of nonalcoholic steatohepatitis: pathophysiology and clinical implications. Gastroenterology. 2012 Apr;142(4):711-725.e6. doi: 10.1053/j.gastro.2012.02.003. Epub 2012 Feb 8.

    PubMed 22326434
  • Macut D, Tziomalos K, Bozic-Antic I, Bjekic-Macut J, Katsikis I, Papadakis E, Andric Z, Panidis D. Non-alcoholic fatty liver disease is associated with insulin resistance and lipid accumulation product in women with polycystic ovary syndrome. Hum Reprod. 2016 Jun;31(6):1347-53. doi: 10.1093/humrep/dew076. Epub 2016 Apr 12.

    PubMed 27076501
  • Kumarendran B, O'Reilly MW, Manolopoulos KN, Toulis KA, Gokhale KM, Sitch AJ, Wijeyaratne CN, Coomarasamy A, Arlt W, Nirantharakumar K. Polycystic ovary syndrome, androgen excess, and the risk of nonalcoholic fatty liver disease in women: A longitudinal study based on a United Kingdom primary care database. PLoS Med. 2018 Mar 28;15(3):e1002542. doi: 10.1371/journal.pmed.1002542. eCollection 2018 Mar.

    PubMed 29590099
  • Ismail FF, Meah N, Trindade de Carvalho L, Bhoyrul B, Wall D, Sinclair R. Safety of oral bicalutamide in female pattern hair loss: A retrospective review of 316 patients. J Am Acad Dermatol. 2020 Nov;83(5):1478-1479. doi: 10.1016/j.jaad.2020.03.034. Epub 2020 Mar 22. No abstract available.

    PubMed 32213304
  • Fernandez-Nieto D, Saceda-Corralo D, Rodrigues-Barata R, Hermosa-Gelbard A, Moreno-Arrones O, Jimenez-Cauhe J, Ortega-Quijano D, Vano-Galvan S. Oral bicalutamide for female pattern hair loss: A pilot study. Dermatol Ther. 2019 Nov;32(6):e13096. doi: 10.1111/dth.13096. Epub 2019 Oct 10. No abstract available.

    PubMed 31579984

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Bicalutamide 50 mg in people with NAFLD and PCOS, run by University of California, San Francisco at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 29 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Lily Carbullido ((415)-502-3725, myliverhealth@ucsf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in liver stiffness on Magnetic Resonance Elastography (MRE) (time frame: Baseline and 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05979389, which also lists 11 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 May 2026.

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