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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
Sponsor:
Collaborators:
AstraZeneca
Astellas Pharma Inc
Information provided by (Responsible Party):
FibroGen

Study Description
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Brief Summary:
The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in participants with lower risk MDS and low red blood cell transfusion burden.

Condition or disease Intervention/treatment Phase
Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts) Drug: Roxadustat Drug: Placebo Phase 3

Detailed Description:
This study includes an Open-Label Lead in, a Double-Blind component, and an Open-Label High Erythropoietin component. There is a screening period of up to 42 days followed by a treatment period of 52 weeks and a 4-week end of treatment assessment.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 184 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Roxadustat

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.

 Drug: Roxadustat
Oral tablets
Other Names:
  • FG-4592
  • ASP1517
  • AZD9941
Placebo Comparator: Placebo
Double-blind: Participants will receive placebo matching to roxadustat for a duration of 52 weeks.
 Drug: Placebo
Oral tablets


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of Participants who Achieve Transfusion Independence (TI) ≥56 Consecutive Days in the First 28 Weeks of Treatment [ Time Frame: 28 weeks ]

Secondary Outcome Measures :
  1. Percentage of Participants who Achieve TI ≥56 Consecutive Days Anytime During the Study [ Time Frame: Weeks 28 and 52 ]
  2. Percentage of Participants who Achieve ≥50% Reduction From Baseline in Number of RBC Transfusion Over Any 8 Weeks [ Time Frame: Weeks 28 and 52 ]
  3. Cumulative Number of Participant-Exposure-Week of TI [ Time Frame: Weeks 28 and 52 ]
  4. Number of Packs of Red Blood Cells (pRBC) Packs Transfused Compared to Baseline [ Time Frame: Weeks 28 and 52 ]
  5. Percentage of Participants who Achieved TI for > 20 Weeks (140 Days) [ Time Frame: Weeks 28 and 52 ]
  6. 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 ]
  7. Mean Change From Baseline in PROMIS Fatigue Score [ Time Frame: Baseline, Weeks 9, 17, 28, 52 and 56 ]
  8. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Sponsors and Collaborators
FibroGen
AstraZeneca
Astellas Pharma Inc
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: FibroGen
ClinicalTrials.gov Identifier: NCT03263091    
Other Study ID Numbers: FGCL-4592-082
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by FibroGen:
Myelodysplastic Syndromes
Anemia
Hemoglobin (Hb)
Low Risk Myelodysplastic Syndrome
Low Risk MDS
Additional relevant MeSH terms:
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Preleukemia
Anemia
Myelodysplastic Syndromes
Syndrome
Disease
 Pathologic Processes
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Neoplasms