Myelodysplastic SyndromesPhase 3NCT07722988
A Study to Investigate the Efficacy and Safety of Roxadustat (FG-4592) for Treating Anemia in Participants With Myelodysplastic Syndromes (MDS)
A Phase 3 study testing Roxadustat in people with Myelodysplastic Syndromes, run by Kyntra Bio.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Roxadustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 201 participants
- Where
- No sites listed yet
- Study dates
- Apr 2027to Dec 2032
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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
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).
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Roxadustat, Placebo
- 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
- Medical subject headings
- Myelodysplastic Syndromes, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RoxadustatDrug
Also called FG-4592
Roxadustat will be administered per schedule specified in the arm description.
PlaceboDrug
Placebo matching to roxadustat will be administered per schedule specified in the arm description.
| Study group | Role | What they receive |
|---|---|---|
| Roxadustat | Experimental | Participants will receive roxadustat, administered orally 3 times per week (TIW). Receives: Drug: Roxadustat |
| Placebo | Placebo comparator | Participants will receive placebo matching to roxadustat, administered orally TIW. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Red Blood Cell-transfusion Independence (RBC-TI) Over Any Consecutive 56-day Period
Measured: Week 1 through Week 24
Secondary outcomes (4)
Percentage of Participants With RBC-TI Over Any Consecutive 84-day Period
Measured: 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
Measured: 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
Measured: Week 1 through Week 48 or EOT (up to 5 years)
Percentage of Participants With RBC-TI Over Any Consecutive 168-day Period
Measured: Week 1 through Week 48 or EOT (up to 5 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2026
- Study startApr 2027 (estimated)
- Primary completionSep 2030 (estimated)
- Study completionDec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 201 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kyntra Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07722988
- Sponsor's protocol ID
- FGCL-4592-115
- Status last verified
- Jul 2026
- First submitted
- 20 Jul 2026
- Last updated
- 23 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07722988
- Acronym / sponsor ID
- FGCL-4592-115
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Roxadustat, FG-4592, LR-MDS, MDS, Myelodysplastic syndromes, Oral, Transfusion, High transfusion burden
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 201 participants (estimated)
- Lead sponsor
- Kyntra BioIndustry (pharma, biotech, devices)
- Study start
- Apr 2027 (estimated)
- Primary completion
- Sep 2030 (estimated)
- Study completion
- Dec 2032 (estimated)
- First posted
- 23 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Roxadustat | Experimental | Drug: RoxadustatParticipants will receive roxadustat, administered orally 3 times per week (TIW). |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive placebo matching to roxadustat, administered orally TIW. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RoxadustatAlso: FG-4592Roxadustat will be administered per schedule specified in the arm description. | Drug | Roxadustat |
| PlaceboPlacebo matching to roxadustat will be administered per schedule specified in the arm description. | Drug | Placebo |
Primary outcome
Percentage of Participants With Red Blood Cell-transfusion Independence (RBC-TI) Over Any Consecutive 56-day Period
Time frame: Week 1 through Week 24
4 secondary outcomes
Percentage of Participants With RBC-TI Over Any Consecutive 84-day Period
Time frame: 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
Time frame: 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
Time frame: Week 1 through Week 48 or EOT (up to 5 years)
Percentage of Participants With RBC-TI Over Any Consecutive 168-day Period
Time frame: Week 1 through Week 48 or EOT (up to 5 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Roxadustat in people with Myelodysplastic Syndromes, run by Kyntra Bio.
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 5 inclusion and 5 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: opening soon, as of the sponsor's last update on 23 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 Mairead Carney (415-978-1337, mcarney@kyntrabio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Red Blood Cell-transfusion Independence (RBC-TI) Over Any Consecutive 56-day Period (time frame: Week 1 through Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Jul 2026.