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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03263091
Ethics application status
Date submitted
22/08/2017
Date registered
28/08/2017

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
Anemia 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
OL and OL High-EPO Components: Number of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence (TI) =8 Weeks (=56 Consecutive Days) Since First Dose in the First 28 Weeks of Treatment
Timepoint [1] 0 0
28 weeks
Primary outcome [2] 0 0
DB Component: Number of Participants Who Achieved RBC TI =56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment
Timepoint [2] 0 0
28 weeks
Secondary outcome [1] 0 0
OL and OL High-EPO Components: Number of Participants Who Achieved TI =50% Reduction From Baseline in Number of Packs of Red Blood Cells (pRBC) Transfusions Over 8 Weeks
Timepoint [1] 0 0
Baseline up to Week 8
Secondary outcome [2] 0 0
DB Component: Number of Participants Who Achieved TI =56 Consecutive Days Since First Dose in 52 Weeks of Treatment
Timepoint [2] 0 0
52 weeks
Secondary outcome [3] 0 0
DB Component: Number of Participants Who Achieved TI =56 Consecutive Days Anytime During the Study
Timepoint [3] 0 0
Baseline up to Week 56
Secondary outcome [4] 0 0
DB Component: Number of Participants Who Achieved =50% Reduction From Baseline in Number of pRBC Transfusions Over 8 Weeks
Timepoint [4] 0 0
Baseline up to Week 8
Secondary outcome [5] 0 0
DB Component: Cumulative Number of Participant Exposure Weeks (PEW) of TI Over the First 28 Weeks of Treatment
Timepoint [5] 0 0
28 weeks
Secondary outcome [6] 0 0
DB Component: Change From Baseline in Number of pRBC Packs Transfused Over the First 28 Weeks of Treatment
Timepoint [6] 0 0
Baseline, Week 28
Secondary outcome [7] 0 0
DB Component: Number of Participants Who Achieved TI =20 Consecutive Weeks During the Study
Timepoint [7] 0 0
Baseline up to Week 56
Secondary outcome [8] 0 0
DB Component: Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System-Short Form (PROMIS-SF) v2.0 Physical Function (PF) 10b Score at Week 9
Timepoint [8] 0 0
Baseline, Week 9
Secondary outcome [9] 0 0
DB Component: Mean Change From Baseline in the PROMIS-SF v1.0 Fatigue 13a Score at Week 9
Timepoint [9] 0 0
Baseline, Week 9
Secondary outcome [10] 0 0
DB Component: Mean Change From Baseline in the European Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) Visual Analogue Scale Score at Week 9
Timepoint [10] 0 0
Baseline, Week 9

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
Stopped early
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
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
South Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
United States of America
State/province [11] 0 0
Virginia
Country [12] 0 0
Belgium
State/province [12] 0 0
Antwerpen
Country [13] 0 0
Belgium
State/province [13] 0 0
Brussels Capital Region
Country [14] 0 0
Belgium
State/province [14] 0 0
Limburg
Country [15] 0 0
Belgium
State/province [15] 0 0
West-Vlaanderen
Country [16] 0 0
Belgium
State/province [16] 0 0
Brussels
Country [17] 0 0
Belgium
State/province [17] 0 0
Yvoir
Country [18] 0 0
Canada
State/province [18] 0 0
British Columbia
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario
Country [20] 0 0
Denmark
State/province [20] 0 0
Odense
Country [21] 0 0
France
State/province [21] 0 0
Alpes-Maritimes
Country [22] 0 0
France
State/province [22] 0 0
Isère
Country [23] 0 0
France
State/province [23] 0 0
Paris
Country [24] 0 0
France
State/province [24] 0 0
Tours
Country [25] 0 0
Germany
State/province [25] 0 0
Baden-Wurttemberg
Country [26] 0 0
Germany
State/province [26] 0 0
Bayern
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Germany
State/province [27] 0 0
Nordrhein-Westfalen
Country [28] 0 0
Germany
State/province [28] 0 0
Sachsen
Country [29] 0 0
Germany
State/province [29] 0 0
Düsseldorf
Country [30] 0 0
India
State/province [30] 0 0
Tamil Nadu
Country [31] 0 0
India
State/province [31] 0 0
West Bengal
Country [32] 0 0
Israel
State/province [32] 0 0
HaMerkaz
Country [33] 0 0
Israel
State/province [33] 0 0
HaZafon
Country [34] 0 0
Israel
State/province [34] 0 0
Haifa
Country [35] 0 0
Israel
State/province [35] 0 0
Ramat Gan
Country [36] 0 0
Israel
State/province [36] 0 0
Tel Aviv
Country [37] 0 0
Israel
State/province [37] 0 0
Tel HaShomer
Country [38] 0 0
Italy
State/province [38] 0 0
Lombardia
Country [39] 0 0
Italy
State/province [39] 0 0
Ravenna
Country [40] 0 0
Italy
State/province [40] 0 0
Alessandria
Country [41] 0 0
Italy
State/province [41] 0 0
Bologna
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Italy
State/province [42] 0 0
Firenze
Country [43] 0 0
Italy
State/province [43] 0 0
Genova
Country [44] 0 0
Italy
State/province [44] 0 0
Lecce
Country [45] 0 0
Italy
State/province [45] 0 0
Milano
Country [46] 0 0
Italy
State/province [46] 0 0
Reggio Calabria
Country [47] 0 0
Italy
State/province [47] 0 0
Rimini
Country [48] 0 0
Italy
State/province [48] 0 0
Roma
Country [49] 0 0
Italy
State/province [49] 0 0
Terni
Country [50] 0 0
Italy
State/province [50] 0 0
Torino
Country [51] 0 0
Italy
State/province [51] 0 0
Varese
Country [52] 0 0
Korea, Republic of
State/province [52] 0 0
Incheon Gwang'yeogsi
Country [53] 0 0
Korea, Republic of
State/province [53] 0 0
Incheon Gwangyeogsi
Country [54] 0 0
Korea, Republic of
State/province [54] 0 0
Jeonranamdo
Country [55] 0 0
Korea, Republic of
State/province [55] 0 0
Seoul Teugbyeolsi
Country [56] 0 0
Korea, Republic of
State/province [56] 0 0
Seoul
Country [57] 0 0
Poland
State/province [57] 0 0
Bialystok
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Poland
State/province [58] 0 0
Bydgoszcz
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Poland
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Krakow
Country [60] 0 0
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Pila
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Poland
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Skorzewo
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Poland
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Slupsk
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Poland
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Warszawa
Country [64] 0 0
Russian Federation
State/province [64] 0 0
Kaluga
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Russian Federation
State/province [65] 0 0
Moscow
Country [66] 0 0
Russian Federation
State/province [66] 0 0
Omsk
Country [67] 0 0
Russian Federation
State/province [67] 0 0
Saint Petersburg
Country [68] 0 0
Russian Federation
State/province [68] 0 0
St. Petersburg
Country [69] 0 0
Spain
State/province [69] 0 0
Barcelona
Country [70] 0 0
Spain
State/province [70] 0 0
Catalunya
Country [71] 0 0
Spain
State/province [71] 0 0
Navarra
Country [72] 0 0
Spain
State/province [72] 0 0
Madrid
Country [73] 0 0
Spain
State/province [73] 0 0
Salamanca
Country [74] 0 0
Spain
State/province [74] 0 0
Sevilla
Country [75] 0 0
Spain
State/province [75] 0 0
Valencia
Country [76] 0 0
Turkey
State/province [76] 0 0
Ankara
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Turkey
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Dikimevi
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Turkey
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Izmir
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Turkey
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Kayseri
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Turkey
State/province [80] 0 0
Tekirdag
Country [81] 0 0
Turkey
State/province [81] 0 0
Yenisehir
Country [82] 0 0
United Kingdom
State/province [82] 0 0
Lincolnshire
Country [83] 0 0
United Kingdom
State/province [83] 0 0
Oxford
Country [84] 0 0
United Kingdom
State/province [84] 0 0
Harrow
Country [85] 0 0
United Kingdom
State/province [85] 0 0
London
Country [86] 0 0
United Kingdom
State/province [86] 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
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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.