Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03263091|
Recruitment Status : Active, not recruiting
First Posted : August 28, 2017
Last Update Posted : September 21, 2022
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|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 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Key Inclusion Criteria:
- Diagnosis of primary MDS classified by the International Prognostic Scoring System - Revised (IPSS-R) as very low, low or intermediate risk with <5% bone marrow blasts. There is no minimum time from diagnosis to registration/randomization except to allow for proper IPSS-R classification to be made (within 16 weeks prior to randomization), and to show transfusion dependence for participants in both portions of the study.
- RBC transfusion of either 2-4 pRBC units during the 8 weeks prior to registration/randomization or 1 pRBC in two consecutive periods of 8 weeks within the 16 weeks prior to registration/randomization. Open-Label Lead-in participants only, the requirement to demonstrate transfusion dependence can also be met by a Principal Investigator starting this particular participant on pRBC transfusion during the screening period.
- No restriction on prior use of recombinant erythropoietins or analogues (erythropoiesis-stimulating agents [ESAs]), except no ESA use within 8 weeks prior to Day 1 registration/randomization.
- Hemoglobin (Hb) ≤10.0 grams/deciliter (g/dL) during screening
- Eastern Cooperative Oncology Group (ECOG) of 0-2 at screening
Key Exclusion Criteria:
- Diagnosis of secondary MDS associated with prior chemotherapy, extensive radiation therapy (>25% of bone marrow reserve), and or/other significant chemical or radiation exposure
- Significant myelofibrosis (>2+ fibrosis)
- MDS associated with 5q(del) cytogenetic abnormality
- Screen serum erythropoietin level > 400 milli-international units (mIU)/milliliter (mL) • Clinically significant anemia, as determined by the investigator, due to non-MDS etiologies such as iron deficiency, vitamin B12 or folate deficiency, autoimmune or hereditary hemolysis or anemia or hemorrhage or hereditary anemia such as sickle cell anemia or thalassemia.
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
|Other Study ID Numbers:||
|First Posted:||August 28, 2017 Key Record Dates|
|Last Update Posted:||September 21, 2022|
|Last Verified:||September 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Low Risk Myelodysplastic Syndrome
Low Risk MDS
Bone Marrow Diseases