NCT07722988

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).

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
201

participants targeted

Target at P25-P50 for phase_3

Timeline
69mo left

Started Apr 2027

Longer than P75 for phase_3

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 20, 2026

Completed
3 days until next milestone

First Posted

Study publicly available on registry

July 23, 2026

Completed
8 months until next milestone

Study Start

First participant enrolled

April 1, 2027

Expected
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2030

2.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2032

Last Updated

July 23, 2026

Status Verified

July 1, 2026

Enrollment Period

3.4 years

First QC Date

July 20, 2026

Last Update Submit

July 20, 2026

Conditions

Myelodysplastic Syndromes

Keywords

RoxadustatFG-4592LR-MDSMDSMyelodysplastic syndromesOralTransfusionHigh transfusion burdenAnemiaRed blood cellTransfusion independenceHemoglobinKyntra BioKyntraFibroGenKYNBESA

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants With Red Blood Cell-transfusion Independence (RBC-TI) Over Any Consecutive 56-day Period

    Week 1 through Week 24

Secondary Outcomes (4)

  • 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)

Study Arms (2)

Roxadustat

EXPERIMENTAL

Participants will receive roxadustat, administered orally 3 times per week (TIW).

Drug: Roxadustat

Placebo

PLACEBO COMPARATOR

Participants will receive placebo matching to roxadustat, administered orally TIW.

Drug: Placebo

Interventions

RoxadustatDRUG

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

Also known as: FG-4592
Roxadustat
PlaceboDRUG

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

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Myelodysplastic SyndromesAnemia

Interventions

roxadustat

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Central Study Contacts

Mairead Carney

CONTACT

415-978-1337mcarney@kyntrabio.com

Javier Moreno

CONTACT

415-978-1466jmoreno@kyntrabio.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 20, 2026

First Posted

July 23, 2026

Study Start (Estimated)

April 1, 2027

Primary Completion (Estimated)

September 1, 2030

Study Completion (Estimated)

December 1, 2032

Last Updated

July 23, 2026

Record last verified: 2026-07

Data Sharing

IPD Sharing
Will not share