A Study to Investigate the Efficacy and Safety of Roxadustat (FG-4592) for Treating Anemia in Participants With Myelodysplastic Syndromes (MDS)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) in Participants With High Red Blood Cell Transfusion Burden

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07722988

Enrollment

201

Registered

2026-07-23

Start date

2027-04-01

Completion date

2032-12-01

Last updated

2026-07-23

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

Conditions

Myelodysplastic Syndromes

Keywords

Roxadustat, FG-4592, LR-MDS, MDS, Myelodysplastic syndromes, Oral, Transfusion, High transfusion burden, Anemia, Red blood cell, Transfusion independence, Hemoglobin, Kyntra Bio, Kyntra, FibroGen, KYNB, ESA

Brief summary

The study is designed to determine the efficacy and safety of roxadustat for the treatment of anemia due to very low, low, or intermediate risk MDS in participants with high transfusion burden (HTB).

Interventions

DRUGRoxadustat

Roxadustat will be administered per schedule specified in the arm description.

DRUGPlacebo

Placebo matching to roxadustat will be administered per schedule specified in the arm description.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

Key Inclusion Criteria: * Diagnosis of MDS according to World Health Organization (WHO) criteria confirmed by bone marrow aspirate and biopsy within 12 weeks before randomization. * Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate risk MDS * HTB: Participants requiring ≥ 4 units of packed red blood cell (pRBC) in two consecutive 8-week periods prior to randomization * Refractory to, intolerant to, or ineligible for prior erythropoiesis-stimulating agents (ESAs). * Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2 Key

Exclusion criteria

* Pregnant or breastfeeding females * Participant has any significant medical illness or is considered vulnerable by local regulations * Prior allogeneic or autologous stem cell transplant * Major surgery within 8 weeks prior to randomization. Note: Other protocol-defined inclusion and

Design outcomes

Primary

Measure	Time frame
Percentage of Participants With Red Blood Cell-transfusion Independence (RBC-TI) Over Any Consecutive 56-day Period	Week 1 through Week 24

Secondary

Measure	Time frame
Percentage of Participants With RBC-TI Over Any Consecutive 84-day Period	Week 1 through Week 48 or end of treatment (EOT; up to 5 years)
Percentage of Participants With RBC-TI Over Any Consecutive 112-day Period	Week 1 through Week 48 or EOT (up to 5 years)
Percentage of Participants With ≥50% Reduction in Rate of Total Red Blood Cell (RBC) Units Transfused During a 16-week Period	Week 1 through Week 48 or EOT (up to 5 years)
Percentage of Participants With RBC-TI Over Any Consecutive 168-day Period	Week 1 through Week 48 or EOT (up to 5 years)

Contacts

CONTACTMairead Carney

mcarney@kyntrabio.com415-978-1337

CONTACTJavier Moreno

jmoreno@kyntrabio.com415-978-1466

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 24, 2026