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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03263091




Registration number
NCT03263091
Ethics application status
Date submitted
22/08/2017
Date registered
28/08/2017
Date last updated
1/03/2024

Titles & IDs
Public title
Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome 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
Haematological diseases
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

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

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.

Placebo Comparator: Placebo - Double-blind: Participants will receive placebo matching to roxadustat for a duration of 52 weeks.


Treatment: Drugs: Roxadustat
Oral tablets

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

Outcomes
Primary outcome [1] 0 0
Percentage of Participants who Achieve Transfusion Independence (TI) =56 Consecutive Days in the First 28 Weeks of Treatment
Timepoint [1] 0 0
28 weeks
Secondary outcome [1] 0 0
Percentage of Participants who Achieve TI =56 Consecutive Days Anytime During the Study
Timepoint [1] 0 0
Weeks 28 and 52
Secondary outcome [2] 0 0
Percentage of Participants who Achieve =50% Reduction From Baseline in Number of RBC Transfusion Over Any 8 Weeks
Timepoint [2] 0 0
Weeks 28 and 52
Secondary outcome [3] 0 0
Cumulative Number of Participant-Exposure-Week of TI
Timepoint [3] 0 0
Weeks 28 and 52
Secondary outcome [4] 0 0
Number of Packs of Red Blood Cells (pRBC) Packs Transfused Compared to Baseline
Timepoint [4] 0 0
Weeks 28 and 52
Secondary outcome [5] 0 0
Percentage of Participants who Achieved TI for > 20 Weeks (140 Days)
Timepoint [5] 0 0
Weeks 28 and 52
Secondary outcome [6] 0 0
Mean Change From Baseline in Physical Function as Measured by Patient Reported Outcomes Measurement Information System (PROMIS)
Timepoint [6] 0 0
Baseline, Weeks 9, 17, 28, 52 and 56
Secondary outcome [7] 0 0
Mean Change From Baseline in PROMIS Fatigue Score
Timepoint [7] 0 0
Baseline, Weeks 9, 17, 28, 52 and 56
Secondary outcome [8] 0 0
Mean Change From Baseline in EuroQol Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) Assessment Score
Timepoint [8] 0 0
Baseline, Weeks 9, 17, 28, 52 and 56

Eligibility
Key inclusion criteria
Key

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

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
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/09/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
22/06/2023
Sample size
Target
Accrual to date
Final
184
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
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United States of America
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Colorado
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Florida
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Georgia
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Missouri
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North Carolina
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Ohio
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Pennsylvania
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South Carolina
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Texas
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Virginia
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Belgium
State/province [12] 0 0
Antwerpen
Country [13] 0 0
Belgium
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Brussels Capital Region
Country [14] 0 0
Belgium
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Limburg
Country [15] 0 0
Belgium
State/province [15] 0 0
West-Vlaanderen
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Belgium
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Brussels
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Yvoir
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Canada
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British Columbia
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Canada
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Ontario
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Denmark
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Odense
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Alpes-Maritimes
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Isère
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Paris
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Tours
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Baden-Wurttemberg
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Bayern
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Sachsen
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Tamil Nadu
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West Bengal
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HaMerkaz
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HaZafon
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Haifa
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Rimini
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Incheon Gwang'yeogsi
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Seoul Teugbyeolsi
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Seoul
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Bialystok
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Krakow
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Pila
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Skorzewo
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Slupsk
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Warszawa
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Kaluga
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Moscow
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Omsk
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Valencia
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Tekirdag
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Yenisehir
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United Kingdom
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Lincolnshire
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Oxford
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Harrow
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London
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United Kingdom
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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
Other collaborator category [2] 0 0
Commercial sector/Industry
Name [2] 0 0
Astellas Pharma Inc
Address [2] 0 0
Country [2] 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 participants with lower risk MDS and low red blood cell transfusion
burden.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03263091
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/ct2/show/NCT03263091