Ignite Creation Date:
2025-12-25 @ 2:50 AM
Ignite Modification Date:
2025-12-26 @ 1:32 AM
Study NCT ID:
NCT06020833
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-09-01
First Post:
2022-02-17
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
Sponsor:
Peking Union Medical College Hospital
Organization:
Study Overview
Official Title:
A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: