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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03263091




Registration number
NCT03263091
Ethics application status
Date submitted
22/08/2017
Date registered
28/08/2017
Date last updated
12/12/2019

Titles & IDs
Public title
Efficacy and Safety of FG-4592 for Treatment of Anemia in Patients With Lower Risk MDS With Low Red Blood Cell Transfusion Burden
Scientific 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)
Secondary ID [1] 0 0
FGCL-4592-082
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts) 0 0
Condition category
Condition code
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - FG-4592
Treatment: Drugs - Placebo

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.

Placebo Comparator: Placebo (Double-blind, Three times a week) - Weight-based starting doses; dose adjustments to hemoglobin levels are allowed during the study.


Treatment: Drugs: FG-4592
Oral

Treatment: Drugs: Placebo
Oral

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Efficacy of roxadustat (FG-4592) to achieve transfusion independence = 56 consecutive days - 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
Timepoint [1] 0 0
28 weeks
Secondary outcome [1] 0 0
Evaluate the incidence of treatment emergent adverse events of roxadustat - Adverse events, serious adverse events, vital signs, electrocardiograms, blood pressure, heart rate, and physical exams
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
Evaluate the impact of roxadustat on RBC transfusion requirements - 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
Timepoint [2] 0 0
52 weeks
Secondary outcome [3] 0 0
Effect of roxadustat on quality of life parameters, as measured by PROMIS and EQ-5D-5L assessment - 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.
Timepoint [3] 0 0
52 weeks
Secondary outcome [4] 0 0
Evaluate transfusion independence = 56 consecutive days - Evaluate transfusion independence by measuring the number of red blood cell packs transfused throughout the course of the study in comparison to baseline
Timepoint [4] 0 0
52 weeks

Eligibility
Key inclusion criteria
Key

- 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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,Saint AlbansTAS
Recruitment hospital [1] 0 0
Investigational Site - Darlinghurst
Recruitment hospital [2] 0 0
Investigational Site - Liverpool
Recruitment hospital [3] 0 0
Investigational Site - South Brisbane
Recruitment hospital [4] 0 0
Investigational Site - Victoria Park
Recruitment hospital [5] 0 0
Investigational Site - Hobart
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
2170 - Liverpool
Recruitment postcode(s) [3] 0 0
4101 - South Brisbane
Recruitment postcode(s) [4] 0 0
3021 - Victoria Park
Recruitment postcode(s) [5] 0 0
7000 - Hobart
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
Belgium
State/province [8] 0 0
Antwerpen
Country [9] 0 0
Belgium
State/province [9] 0 0
Brussels Capital Region
Country [10] 0 0
Belgium
State/province [10] 0 0
Limburg
Country [11] 0 0
Belgium
State/province [11] 0 0
West-Vlaanderen
Country [12] 0 0
Germany
State/province [12] 0 0
Baden-Wurttemberg
Country [13] 0 0
Germany
State/province [13] 0 0
Bayern
Country [14] 0 0
Germany
State/province [14] 0 0
Nordrhein-Westfalen
Country [15] 0 0
Germany
State/province [15] 0 0
Sachsen
Country [16] 0 0
Germany
State/province [16] 0 0
Hamburg
Country [17] 0 0
Israel
State/province [17] 0 0
HaMerkaz
Country [18] 0 0
Israel
State/province [18] 0 0
HaZafon
Country [19] 0 0
Israel
State/province [19] 0 0
Haifa
Country [20] 0 0
Israel
State/province [20] 0 0
Tel Aviv
Country [21] 0 0
Israel
State/province [21] 0 0
Tel HaShomer
Country [22] 0 0
Italy
State/province [22] 0 0
Ravenna
Country [23] 0 0
Italy
State/province [23] 0 0
Alessandria
Country [24] 0 0
Italy
State/province [24] 0 0
Bologna
Country [25] 0 0
Italy
State/province [25] 0 0
Firenze
Country [26] 0 0
Italy
State/province [26] 0 0
Genova
Country [27] 0 0
Italy
State/province [27] 0 0
Rimini
Country [28] 0 0
Italy
State/province [28] 0 0
Terni
Country [29] 0 0
Italy
State/province [29] 0 0
Torino
Country [30] 0 0
Italy
State/province [30] 0 0
Varese
Country [31] 0 0
Korea, Republic of
State/province [31] 0 0
Incheon Gwang'yeogsi
Country [32] 0 0
Korea, Republic of
State/province [32] 0 0
Incheon Gwangyeogsi
Country [33] 0 0
Korea, Republic of
State/province [33] 0 0
Jeonranamdo
Country [34] 0 0
Korea, Republic of
State/province [34] 0 0
Seoul Teugbyeolsi
Country [35] 0 0
Russian Federation
State/province [35] 0 0
Kaluga
Country [36] 0 0
Russian Federation
State/province [36] 0 0
Moscow
Country [37] 0 0
Russian Federation
State/province [37] 0 0
Omsk
Country [38] 0 0
Russian Federation
State/province [38] 0 0
Saint Petersburg
Country [39] 0 0
Spain
State/province [39] 0 0
Barcelona
Country [40] 0 0
Spain
State/province [40] 0 0
Catalunya
Country [41] 0 0
Spain
State/province [41] 0 0
Navarra
Country [42] 0 0
Spain
State/province [42] 0 0
Madrid
Country [43] 0 0
Spain
State/province [43] 0 0
Salamanca
Country [44] 0 0
Spain
State/province [44] 0 0
Sevilla
Country [45] 0 0
United Kingdom
State/province [45] 0 0
Lincolnshire
Country [46] 0 0
United Kingdom
State/province [46] 0 0
Harrow
Country [47] 0 0
United Kingdom
State/province [47] 0 0
London
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
FibroGen
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
AstraZeneca
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03263091
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
K. Peony Yu, MD
Address 0 0
FibroGen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Charles Bradley, PhD
Address 0 0
Country 0 0
Phone 0 0
1-415-978-1672
Fax 0 0
Email 0 0
082MDSstudy@fibrogen.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03263091