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

Sponsor
Peking Union Medical College Hospital (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT06020833
Collaborator
(none)
25
Enrollment
1
Arm
40
Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Roxadustat in combination with retinoic acid
 Phase 1/Phase 2

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

Study Type:
Interventional
Anticipated Enrollment :
25 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
Anticipated Study Start Date :
Aug 1, 2023
Anticipated Primary Completion Date :
Oct 1, 2025
Anticipated Study Completion Date :
Dec 1, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: Roxadustat combined with retinoic acid group

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

Outcome Measures

Primary Outcome Measures

  1. overall response rate (OR) [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

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
  • Low-risk MDS unresponsive to first-line therapy
Exclusion Criteria:

  • myelofibrosis

  • Severe heart and kidney function bu'quan

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Peking Union Medical College Hospital

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

Responsible Party:
Bing Han, Professor, Peking Union Medical College Hospital
ClinicalTrials.gov Identifier:
NCT06020833
Other Study ID Numbers:
  • Roxadustat-RA-MDS
First Posted:
Sep 1, 2023
Last Update Posted:
Sep 1, 2023
Last Verified:
Aug 1, 2023
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Myelodysplastic Syndromes
Tretinoin

Study Results

No Results Posted as of Sep 1, 2023