Clincosm LogoSign in Get a demo

Efficacy and Safety of FG-4592 for Treatment of Anemia in Patients With Lower Risk MDS With Low Red Blood Cell Transfusion Burden

Sponsor
FibroGen (Industry)
Overall Status
Recruiting
CT.gov ID
NCT03263091
Collaborator
AstraZeneca (Industry), Astellas Pharma Inc (Industry)
204
Enrollment
73
Locations
3
Arms
57.8
Anticipated Duration (Months)
2.8
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 3

Detailed Description

This study includes an Open-Label Lead in, a Double-Blind component and an Open-Label High Erythropoietin component. In each, there is an up to 42 days screen period followed by a treatment period of 52 weeks and a 4 week end of treatment assessment.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
204 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 :
Sep 7, 2017
Anticipated Primary Completion Date :
Jan 1, 2022
Anticipated Study Completion Date :
Jul 1, 2022

Arms and Interventions

Arm Intervention/Treatment
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.

Drug: FG-4592
Oral
Other Names:
  • Roxadustat
  • ASP1517
  • AZD9941

    		•
    Placebo Comparator: Placebo (Double-blind, Three times a week)

    Weight-based starting doses; dose adjustments to hemoglobin levels are allowed during the study.

    Drug: Placebo
    Oral
    Experimental: Open-Label High Erythropoietin

    Weight-based starting doses; dose adjustments to hemoglobin levels are allowed during the study.

    Drug: FG-4592
    Oral
    Other Names:
  • Roxadustat
  • ASP1517
  • AZD9941

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Efficacy of roxadustat (FG-4592) to achieve transfusion independence ≥ 56 consecutive days [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

    Secondary Outcome Measures

    1. Evaluate the incidence of treatment emergent adverse events of roxadustat [52 weeks]

      Adverse events, serious adverse events, vital signs, electrocardiograms, blood pressure, heart rate, and physical exams

    2. Evaluate the impact of roxadustat on RBC transfusion requirements [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

    3. Effect of roxadustat on quality of life parameters, as measured by PROMIS and EQ-5D-5L assessment [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.

    4. Evaluate transfusion independence ≥ 56 consecutive days [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

    Other Outcome Measures

    1. Duration of transfusion independence [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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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 (Double Blind); for Open- Label High Erythropoietin patients serum erythropoietin level must be >400 mIU/mL

    • Clinically significant anemia due to non-MDS etiologies

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Investigational Site Los Angeles California United States 91326
    2 Investigational Site Pasadena California United States 91105
    3 Investigational Site Torrance California United States 90505
    4 Investigational Site Valencia California United States 91355
    5 Investigational Site Ventura California United States 93003
    6 Investigational Site Westlake Village California United States 91361
    7 Investigational Site Whittier California United States 90603
    8 Investigational Site Pembroke Pines Florida United States 33028
    9 Investiational Site Weston Florida United States 33331
    10 Investgational site Atlanta Georgia United States 30322
    11 Investigational Site Saint Louis Missouri United States 63110
    12 Investigational Site Canton Ohio United States 44718
    13 Investigational Site Cleveland Ohio United States 44111
    14 Investigational Site Cleveland Ohio United States 44195
    15 Investigational Site Mayfield Heights Ohio United States 44124
    16 Investigational Site Philadelphia Pennsylvania United States 19106
    17 Investigational Site Houston Texas United States 77030
    18 Investigational Site Darlinghurst New South Wales Australia 2010
    19 Investigational Site Liverpool New South Wales Australia 2170
    20 Investigational Site South Brisbane Queensland Australia 4101
    21 Investigational Site Victoria Park Saint Albans Australia 3021
    22 Investigational Site Hobart Tasmania Australia 7000
    23 Investigational Site Wilrijk Antwerpen Belgium 2610
    24 Investigational Site Bruxelles Brussels Capital Region Belgium 1200
    25 Investigational Site Hasselt Limburg Belgium 3500
    26 Investigational Site Brugge West-Vlaanderen Belgium 8000
    27 Investigational Site Freiburg Baden-Wurttemberg Germany 79106
    28 Investigational Site Munchen Bayern Germany 81675
    29 Investigational Site Münster Nordrhein-Westfalen Germany 48149
    30 Investigational Site Dresden Sachsen Germany 01307
    31 Investigational Site Leipzig Sachsen Germany 04103
    32 Investigational Site Hamburg Germany 20246
    33 Investigational Site Kfar Saba HaMerkaz Israel 4428164
    34 Investigational Site Zerifin HaMerkaz Israel 70300
    35 Investigational Site Nahariya HaZafon Israel 22100
    36 Investigational Site Haifa Israel 3436212
    37 Investigational Site Tel Aviv Israel 49372
    38 Investgational site Tel HaShomer Israel
    39 Investigational Site Meldola Ravenna Italy 47014
    40 Investigational Site Alessandria Italy 15121
    41 Investigational Site Bologna Italy 40138
    42 Investigational Site Firenze Italy 50134
    43 Investigational Site Genova Italy 16132
    44 Investigational Site Ravenna Italy
    45 Investigational Site Rimini Italy
    46 Investigational Site Terni Italy 5100
    47 Investigational Site Torino Italy
    48 Investigational Site Varese Italy 21100
    49 Investigational Site Incheon Incheon Gwang'yeogsi Korea, Republic of 21565
    50 Investigational Site Incheon Incheon Gwangyeogsi Korea, Republic of 21565
    51 Investigational Site Hwasun Jeonranamdo Korea, Republic of 58128
    52 Investigational Site Seoul Seoul Teugbyeolsi Korea, Republic of 06351
    53 Investigational Site Kaluga Russian Federation 248007
    54 Investigational Site Moscow Russian Federation 111123
    55 Investigational Site Moscow Russian Federation 123182
    56 Investigational Site Moscow Russian Federation 129110
    57 Investigational Site Omsk Russian Federation 644013
    58 Investigational Site Saint Petersburg Russian Federation 191024
    59 Investigational Site Saint Petersburg Russian Federation 197089
    60 Investigational Site Saint Petersburg Russian Federation 197341
    61 Investigational Site Sabadell Barcelona Spain 08208
    62 Investigational Site Barcelona Catalunya Spain 08003
    63 Investigational Site Pamplona Navarra Spain 31008
    64 Investigational Site Barcelona Spain 08041
    65 Investigational Site Madrid Spain 28034
    66 Investigational Site Madrid Spain 28050
    67 Investigational Site Salamanca Spain 37007
    68 Investigational Site Sevilla Spain 41009
    69 Investigational Site Sevilla Spain 41013
    70 Investigational Site Boston Lincolnshire United Kingdom PE21 9QS
    71 Investigational Site Harrow United Kingdom HA1 3UJ
    72 Investigational Site London United Kingdom SE5 9RS
    73 Investigational Site Manchester United Kingdom M20 4BX

    Sponsors and Collaborators

    • FibroGen
    • AstraZeneca
    • Astellas Pharma Inc

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    FibroGen
    ClinicalTrials.gov Identifier:
    NCT03263091
    Other Study ID Numbers:
    • FGCL-4592-082
    First Posted:
    Aug 28, 2017
    Last Update Posted:
    Feb 9, 2021
    Last Verified:
    Feb 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    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:
    Anemia
    Myelodysplastic Syndromes

    Study Results

    No Results Posted as of Feb 9, 2021