Myelodysplastic SyndromesPhase 1/2NCT06020833
A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
A Phase 1/2 study testing Roxadustat in combination with retinoic acid in people with Myelodysplastic Syndromes, run by Peking Union Medical College Hospital.
Trial summary
Roxadustat has been approved for low-risk MDS clinical trials, but the trial results are not available. For refractory low-risk MDS, the effective rate of roxadustat treatment is about 20-30%, and roxadustat combined with retinoic acid may have better efficacy in the treatment of refractory low-risk MDS. 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 in combination with retinoic acid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Aug 2023to Dec 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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
- Low-risk MDS unresponsive to first-line therapy
Who cannot join
- myelofibrosis
- Severe heart and kidney function bu'quan
About this study
Roxadustat has been approved for low-risk MDS clinical trials, but the trial results are not available. For refractory low-risk MDS, the effective rate of roxadustat treatment is about 20-30%, and roxadustat combined with retinoic acid may have better efficacy in the treatment of refractory low-risk MDS.
Read the detailed description
Patients with low-risk myelodysplastic syndrome who are refractory to the regular treatments have to live on transfusions which lead to poor quality of life (QoL) and survival. Roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor, which increases both endogenous EPO and iron metabolism, has shown promising results in several phase 3 clinical trials for chronic kidney disease.
Roxadustat comprehensively improves iron metabolism by upregulating DCYTB and DMT1 through the HIF pathway, which can promote the iron absorption and utilization. Detail mechanisms include upregulating transferrin receptors to increase iron uptake, upregulating transferrin to promote iron transport and downregulating ferritin levels to indirectly improve iron absorption and transportation.
Patients with refractory low-risk MDS were selected and given roxadustat 150mg po qod combined with retinoic acid 20mg po bid.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Roxadustat in combination with retinoic acid
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Roxadustat in combination with retinoic acidDrug
Roxadustat 150mg po qod combined with retinoic acid 20mg po bid
| Study group | Role | What they receive |
|---|---|---|
| Roxadustat combined with retinoic acid group | Experimental | Not described Receives: Drug: Roxadustat in combination with retinoic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall response rate (OR)
Complete and partial remission with roxadustat combined with retinoic acid. Partial remission includes freedom from transfusion dependence or reduction in transfusion volume within 8 weeks
Measured: 3, 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2023
- Study startAug 2023 (estimated)
- Primary completionOct 2025 (estimated)
- Study completionDec 2026 (estimated)
Why it stopped: The study has not actually been conducted.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06020833
- Sponsor's protocol ID
- Roxadustat-RA-MDS
- Status last verified
- Aug 2023
- First submitted
- 17 Feb 2022
- Last updated
- 7 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Carraway HE, Saygin C. Therapy for lower-risk MDS. Hematology Am Soc Hematol Educ Program. 2020 Dec 4;2020(1):426-433. doi: 10.1182/hematology.2020000127.
PubMed 33275714
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
- ClinicalTrials.gov ID
- NCT06020833
- Acronym / sponsor ID
- Roxadustat-RA-MDS
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study has not actually been conducted.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Aug 2023 (estimated)
- Primary completion
- Oct 2025 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 1 Sep 2023 (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.
Roxadustat has been approved for low-risk MDS clinical trials, but the trial results are not available. For refractory low-risk MDS, the effective rate of roxadustat treatment is about 20-30%, and roxadustat combined with retinoic acid may have better efficacy in the treatment of refractory low-risk MDS.
Detailed description
Patients with low-risk myelodysplastic syndrome who are refractory to the regular treatments have to live on transfusions which lead to poor quality of life (QoL) and survival. Roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor, which increases both endogenous EPO and iron metabolism, has shown promising results in several phase 3 clinical trials for chronic kidney disease.
Roxadustat comprehensively improves iron metabolism by upregulating DCYTB and DMT1 through the HIF pathway, which can promote the iron absorption and utilization. Detail mechanisms include upregulating transferrin receptors to increase iron uptake, upregulating transferrin to promote iron transport and downregulating ferritin levels to indirectly improve iron absorption and transportation.
Patients with refractory low-risk MDS were selected and given roxadustat 150mg po qod combined with retinoic acid 20mg po bid.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Roxadustat combined with retinoic acid group | Experimental | Drug: Roxadustat in combination with retinoic acid |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Low-risk MDS unresponsive to first-line therapy
Key exclusion criteria (2 listed)
- myelofibrosis
- Severe heart and kidney function bu'quan
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 |
|---|---|---|
| Roxadustat in combination with retinoic acidRoxadustat 150mg po qod combined with retinoic acid 20mg po bid | Drug | Roxadustat combined with retinoic acid group |
Primary outcome
overall response rate (OR)
Time frame: 3, 6 months
Complete and partial remission with roxadustat combined with retinoic acid. Partial remission includes freedom from transfusion dependence or reduction in transfusion volume within 8 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
- Background: PubMed 33275714Carraway HE, Saygin C. Therapy for lower-risk MDS. Hematology Am Soc Hematol Educ Program. 2020 Dec 4;2020(1):426-433. doi: 10.1182/hematology.2020000127.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Roxadustat in combination with retinoic acid in people with Myelodysplastic Syndromes, run by Peking Union Medical College Hospital.
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 1 inclusion and 2 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: withdrawn, as of the sponsor's last update on 7 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall response rate (OR) (time frame: 3, 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06020833, which also lists 1 linked publication.
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 7 Jul 2026.