A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
Primary Purpose
Myelodysplastic Syndromes
Status
Not yet recruiting
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Roxadustat in combination with retinoic acid
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndromes
Eligibility Criteria
Inclusion Criteria: Low-risk MDS unresponsive to first-line therapy Exclusion Criteria: myelofibrosis Severe heart and kidney function bu'quan
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Roxadustat combined with retinoic acid group
Arm Description
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Full Information
NCT ID
NCT06020833
First Posted
February 17, 2022
Last Updated
August 26, 2023
Sponsor
Peking Union Medical College Hospital
1. Study Identification
Unique Protocol Identification Number
NCT06020833
Brief Title
A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
Official Title
A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
August 2023 (Anticipated)
Primary Completion Date
October 2025 (Anticipated)
Study Completion Date
December 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking Union Medical College Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Roxadustat combined with retinoic acid group
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Roxadustat in combination with retinoic acid
Intervention Description
Roxadustat 150mg po qod combined with retinoic acid 20mg po bid
Primary Outcome Measure Information:
Title
overall response rate (OR)
Description
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
Time Frame
3, 6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Low-risk MDS unresponsive to first-line therapy
Exclusion Criteria:
myelofibrosis
Severe heart and kidney function bu'quan
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
bing Han, Ph.D
Phone
+86 13601059938
Email
hanbing_li@sina.com.cn
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
33275714
Citation
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.
Results Reference
background
Learn more about this trial
A Single-arm Trial of Roxadustat Combined With Retinoic Acid in the Treatment of Refractory Low-risk MDS
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