Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden
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|ClinicalTrials.gov Identifier: NCT03263091|
Recruitment Status : Active, not recruiting
First Posted : August 28, 2017
Last Update Posted : September 21, 2022
|Condition or disease||Intervention/treatment||Phase|
|Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts)||Drug: Roxadustat Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||184 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3 Randomized Double-Blind Placebo-Controlled Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Patients With Lower Risk Myelodysplastic Syndrome (MDS) With Low Red Blood Cell (RBC) Transfusion Burden (LTB)|
|Actual Study Start Date :||September 7, 2017|
|Estimated Primary Completion Date :||March 31, 2023|
|Estimated Study Completion Date :||September 30, 2023|
Open-label, lead-in: Participants will receive sequential escalating roxadustat doses (1.5 milligrams/kilograms [mg/kg], 2.0 mg/kg and 2.5 mg/kg), three times a week (TIW) based upon their actual weight at the randomization visit to identify the starting dose for double-blind period.
Double-blind: Participants will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks.
Open-label: Participants with high serum erythropoietin levels (>400 milli-international units [mIU]/milliliter [mL] mIU/mL) will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks.
Placebo Comparator: Placebo
Double-blind: Participants will receive placebo matching to roxadustat for a duration of 52 weeks.
- Percentage of Participants who Achieve Transfusion Independence (TI) ≥56 Consecutive Days in the First 28 Weeks of Treatment [ Time Frame: 28 weeks ]
- Percentage of Participants who Achieve TI ≥56 Consecutive Days Anytime During the Study [ Time Frame: Weeks 28 and 52 ]
- Percentage of Participants who Achieve ≥50% Reduction From Baseline in Number of RBC Transfusion Over Any 8 Weeks [ Time Frame: Weeks 28 and 52 ]
- Cumulative Number of Participant-Exposure-Week of TI [ Time Frame: Weeks 28 and 52 ]
- Number of Packs of Red Blood Cells (pRBC) Packs Transfused Compared to Baseline [ Time Frame: Weeks 28 and 52 ]
- Percentage of Participants who Achieved TI for > 20 Weeks (140 Days) [ Time Frame: Weeks 28 and 52 ]
- Mean Change From Baseline in Physical Function as Measured by Patient Reported Outcomes Measurement Information System (PROMIS) [ Time Frame: Baseline, Weeks 9, 17, 28, 52 and 56 ]
- Mean Change From Baseline in PROMIS Fatigue Score [ Time Frame: Baseline, Weeks 9, 17, 28, 52 and 56 ]
- Mean Change From Baseline in EuroQol Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) Assessment Score [ Time Frame: Baseline, Weeks 9, 17, 28, 52 and 56 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03263091