Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT03263091
Previous Study | Return to List | Next Study

Efficacy and Safety of FG-4592 for Treatment of Anemia in Patients With Lower Risk MDS 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03263091
Recruitment Status : Recruiting
First Posted : August 28, 2017
Last Update Posted : December 12, 2019
Sponsor:
Collaborator:
AstraZeneca
Information provided by (Responsible Party):
FibroGen

Tracking Information
First Submitted Date  ICMJE August 22, 2017
First Posted Date  ICMJE August 28, 2017
Last Update Posted Date December 12, 2019
Actual Study Start Date  ICMJE September 7, 2017
Estimated Primary Completion Date April 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 23, 2017)
Efficacy of roxadustat (FG-4592) to achieve transfusion independence ≥ 56 consecutive days [ Time Frame: 28 weeks ]
Efficacy of roxadustat (FG-4592) in achieving hemoglobin correction and maintenance and reducing the number of red blood cell packs transfused in 28 weeks in comparison to baseline
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 25, 2017)
  • Evaluate the incidence of treatment emergent adverse events of roxadustat [ Time Frame: 52 weeks ]
    Adverse events, serious adverse events, vital signs, electrocardiograms, blood pressure, heart rate, and physical exams
  • Evaluate the impact of roxadustat on RBC transfusion requirements [ Time Frame: 52 weeks ]
    Impact of roxadustat (FG-4592) in achieving hemoglobin correction and maintenance and reducing the number of red blood cell packs transfused throughout the course of the study in comparison to baseline
  • Effect of roxadustat on quality of life parameters, as measured by PROMIS and EQ-5D-5L assessment [ Time Frame: 52 weeks ]
    Change in raw score from baseline in Physical Function (PF) and Fatigue score as measured by Patient-Reported Outcome Measurement Information System (PROMIS). And change in raw score from baseline in the EuroQol Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) assessment.
  • Evaluate transfusion independence ≥ 56 consecutive days [ Time Frame: 52 weeks ]
    Evaluate transfusion independence by measuring the number of red blood cell packs transfused throughout the course of the study in comparison to baseline
Original Secondary Outcome Measures  ICMJE
 (submitted: August 23, 2017)
  • Evaluate the safety of roxadustat [ Time Frame: 52 weeks ]
    Adverse events, serious adverse events, vital signs, electrocardiograms, blood pressure, heart rate, and physical exams
  • Evaluate the impact of roxadustat on RBC transfusion requirements [ Time Frame: 52 weeks ]
    Impact of roxadustat (FG-4592) in achieving hemoglobin correction and maintenance and reducing the number of red blood cell packs transfused throughout the course of the study in comparison to baseline
  • Effect of roxadustat on quality of life parameters, as measured by PROMIS and EQ-5D-5L assessment [ Time Frame: 52 weeks ]
    Mean change from baseline in Physical Function (PF) and Fatigue score as measured by Patient-Reported Outcome Measurement Information System (PROMIS). And mean change from baseline in the EuroQol Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) assessment.
  • Evaluate transfusion independence ≥ 56 consecutive days [ Time Frame: 52 weeks ]
    Evaluate transfusion independence by measuring the number of red blood cell packs transfused throughout the course of the study in comparison to baseline
Current Other Pre-specified Outcome Measures
 (submitted: August 23, 2017)
Duration of transfusion independence [ Time Frame: 52 weeks ]
Evaluate transfusion independence by measuring the number of red blood cell packs transfused throughout the course of the study in comparison to baseline
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of FG-4592 for Treatment of Anemia in Patients With Lower Risk MDS With Low Red Blood Cell Transfusion Burden
Official Title  ICMJE 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)
Brief Summary The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in patients with Lower Risk Myelodysplastic Syndrome and Low Red Blood Cell Transfusion Burden.
Detailed Description This study includes an Open-Label and a Double-Blind component. In each, there is an up to 28 days screen period followed by a treatment period of 52 weeks and a 4 week end of treatment assessment.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts)
Intervention  ICMJE
  • Drug: FG-4592
    Oral
    Other Names:
    • Roxadustat
    • ASP1517
    • AZD9941
  • Drug: Placebo
    Oral
Study Arms  ICMJE
  • Experimental: FG-4592 (Open Label, Double-blind, Three times a week)
    Weight-based starting doses; dose adjustments to hemoglobin levels are allowed during the study.
    Intervention: Drug: FG-4592
  • Placebo Comparator: Placebo (Double-blind, Three times a week)
    Weight-based starting doses; dose adjustments to hemoglobin levels are allowed during the study.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 23, 2017)
184
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2021
Estimated Primary Completion Date April 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Diagnosis of primary MDS classified as very low, low or intermediate risk with <5% blasts. There is no minimum time from diagnosis except to allow for proper IPSS-R classification to be made, and to show transfusion dependence.
  • RBC transfusion of either 2-4 pRBC units over the 8 weeks prior to randomization or 1 pRBC in two consecutive periods of 8 weeks within the 16 weeks prior to randomization
  • No restriction on prior use of ESAs, except no ESA use within 8 weeks prior to registration/randomization
  • Pre-transfusion hemoglobin of <= 10 g/dL,
  • ECOG of 0-2 at screen
  • History of cured malignancy with no evidence of recurrence for a least 3 years are eligible

Key Exclusion Criteria:

  • Diagnosis of secondary MDS
  • Significant myelofibrosis (>2+fibrosis)
  • MDS associated with 5q(del) abnormality
  • Screen serum erythropoietin level > 400 mIU/mL,
  • Clinically significant anemia due to non-MDS etiologies
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Charles Bradley, PhD 1-415-978-1672 082MDSstudy@fibrogen.com
Contact: Gopal Saha, MD 082MDSstudy@fibrogen.com
Listed Location Countries  ICMJE Australia,   Belgium,   Germany,   Israel,   Italy,   Korea, Republic of,   Russian Federation,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03263091
Other Study ID Numbers  ICMJE FGCL-4592-082
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Responsible Party FibroGen
Study Sponsor  ICMJE FibroGen
Collaborators  ICMJE AstraZeneca
Investigators  ICMJE
Study Chair: K. Peony Yu, MD FibroGen
PRS Account FibroGen
Verification Date December 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP