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

NCT07722988 — Phase 3, Not yet recruiting, 201 participants, Kyntra Bio

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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

Registry	NCT07722988
Status	Not yet recruiting
Phase	Phase 3
Study type	Interventional; randomized, parallel, masking: quadruple, treatment
Enrollment	201 (Estimated)
Sponsor	Kyntra Bio (Industry)
Conditions	Myelodysplastic Syndromes
Interventions	Roxadustat, Placebo (Drug)
Dates	start 2027-04-01 (estimated), primary completion 2030-09-01 (estimated), completion 2032-12-01 (estimated), first posted 2026-07-23, last updated 2026-07-23

-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).

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+Arms and interventions

Arm	Type	Interventions	Description
Roxadustat	Experimental	Drug: Roxadustat	Participants will receive roxadustat, administered orally 3 times per week (TIW).
Placebo	Placebo comparator	Drug: Placebo	Participants will receive placebo matching to roxadustat, administered orally TIW.

Roxadustat (Drug), also known as FG-4592: Roxadustat will be administered per schedule specified in the arm description.
Placebo (Drug): Placebo matching to roxadustat will be administered per schedule specified in the arm description.

+Outcome measures

Primary outcomes

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

Secondary outcomes

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)]

+Eligibility

Sex: All, ages: 18 Years to any

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 exclusion criteria may apply.