A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS

Myelodysplastic Syndromes Clinical Trial

Official title:

A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06020833
• Other study ID #: Roxadustat-RA-MDS
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: August 2023
• Est. completion date: December 2026

Study information

• Verified date: August 2023
• Source: Peking Union Medical College Hospital
• Contact: bing Han, Ph.D
• Phone: +86 13601059938
• Email: hanbing_li[@]sina.com.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 25
• Est. completion date: December 2026
• Est. primary completion date: October 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Low-risk MDS unresponsive to first-line therapy

Exclusion Criteria:

• myelofibrosis

• Severe heart and kidney function bu'quan

Related Conditions & MeSH terms

• Myelodysplastic Syndromes

Intervention

Drug:
• Roxadustat in combination with retinoic acid
Roxadustat 150mg po qod combined with retinoic acid 20mg po bid

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Peking Union Medical College Hospital

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	3, 6 months