A study of the short-term effects of oral Roxadustat in volunteers over the age of 40.

An open-label volunteer study of the impact of short-term oral Roxadustat exposure on gene expression in whole blood in healthy adults aged 40 years and over.

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12625000491460

Enrollment

Registered

2025-05-21

Start date

2025-06-27

Completion date

2025-07-31

Last updated

2026-06-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

Roxadustat is a first-in-class oral prolyl hydroxylase inhibitor, marketed since 2019 exclusively for the treatment of anaemia in chronic kidney disease. Roxadustat increases hypoxia inducible factor (HIF) signalling, the primary mediator of the body’s normal adaptive response to low oxygen levels. HIF is a transcription factor that influences the expression of over 400 genes relating to energy production when oxygen is scarce (glycolysis), improved tissue oxygen delivery (erythropoiesis and angiogenesis), cell survival under stress and enhanced innate immunity. All of these biological mechanisms play a key role in favourable outcomes after the tissue injury of major surgery and the associated stress response. The HYPE Pilot Study (RGS0000006528, SMHS HREC, ACTRN12624000188538) is a separately registered double-blind phase 2 RCT that is currently recruiting at Royal Perth Hospital to explore the safety and potential benefit of three preoperative doses of Roxadustat 100mg in patients undergoing major noncardiac surgery. It remains unclear to what extent such brief Roxadustat exposure changes gene expression in the key pathways outlined above. The HYPE Volunteer Study (RGS0000007340, Central HREC) will complement the HYPE Pilot Study by improving understanding in this area i.e. will potentially substantiate any clinical effects observed in the HYPE Pilot Study and may support a subsequent phase 3 trial.

Interventions

Three doses of Roxadustat 100mg oral tablets taken on alternate days across a 5-day period i.e. 1x100mg on Day 1; 1x100mg on Day 3; 1x100mg on Day 5. Adherence will be monitored by completion of a Treatment Diary.

Study design

Allocation

Non-randomised trial

Intervention model

Single group

Primary purpose

Prevention

Masking

Open (masking not used)

Eligibility

Sex/Gender

All

Age

40 Years to No maximum

Healthy volunteers

Yes

Inclusion criteria

1. Age 40 years or over

Exclusion criteria

1. Inability to provide informed consent 2. Peanut or Soya allergy 3. Pregnancy or breast feeding 4. Epilepsy 5. Severe liver disease (Childs Pugh C) 6. Current upper respiratory tract infection 6. Current bacterial infection

Outcome results