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

NCT ID: NCT06020833

Last Updated: 2023-09-01

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-31
• Study Completion Date: 2026-12-31

Brief 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.

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Roxadustat combined with retinoic acid group

Group Type: EXPERIMENTAL

Roxadustat in combination with retinoic acid

Intervention Type: DRUG

Roxadustat 150mg po qod combined with retinoic acid 20mg po bid

Interventions

Roxadustat in combination with retinoic acid

Roxadustat 150mg po qod combined with retinoic acid 20mg po bid

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Low-risk MDS unresponsive to first-line therapy

Exclusion Criteria

• myelofibrosis
• Severe heart and kidney function bu'quan

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Bing Han

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Central Contacts

bing Han, Ph.D

Role: CONTACT

hanbing_li@sina.com.cn

+86 13601059938

References

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.

Reference Type: BACKGROUND

PMID: 33275714 (View on PubMed)

Other Identifiers

Roxadustat-RA-MDS

Identifier Type: -

Identifier Source: org_study_id