Efficacy & Safety of FG-4592 in Anaemia with Lower Risk MDS & RBC LTB
Research type
Research Study
Full 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)
IRAS ID
229602
Contact name
Holly Spencer
Contact email
Sponsor organisation
FibroGen, Inc.
Eudract number
2017-001773-17
Duration of Study in the UK
1 years, 7 months, 2 days
Research summary
The purpose of this study is to find out if the experimental drug roxadustat (also known as FG-4592) is safe and effective for potentially treating anaemia (not enough red blood cells) in people diagnosed with lower risk myelodysplastic syndrome (MDS) (a rare disorder in which the participants bone marrow does not make enough healthy blood cells).
There are several drugs called Erythropoiesis Stimulating Agents (ESAs) that are sometimes prescribed by doctors to treat anaemia in MDS participants which are considered to be an “off-label use” (not approved by the Medicines & Healthcare Products Regulatory Agency for treatment of anaemia in MDS participants). ESAs stimulate red blood cell production. Roxadustat is different from these ESAs in that it is thought to mimic the body’s normal response to high altitude (such as going up a mountain) by increasing the production of red blood cells.
Participants will be asked to participate because they have MDS and the study doctor believes their level of haemoglobin (a protein in red blood cells that carries oxygen) may be low enough to be considered for participation in this study to evaluate a possible drug which could help their anaemia.
Approximately 184 MDS study participants will participate at about 80 study sites worldwide. Up to 24 MDS study participants will participate in one group and up to 160 MDS study participants will participate in this group, the Double-Blind Group (Double-Blind means that both participant and the study doctor will not know whether they are taking roxadustat or placebo).
Participants will be randomly assigned to take roxadustat or placebo at a ratio of 3 to 2 (approximately 96 participants will take roxadustat and 64 participants will take placebo). Participants will have a 60% chance of receiving roxadustat and a 40% chance of receiving placebo.
Participants will receive roxadustat or placebo for a maximum of 52 weeks.
This study is being sponsored and financed by a company called FibroGen, Inc.
REC name
London - Chelsea Research Ethics Committee
REC reference
17/LO/2075
Date of REC Opinion
20 Feb 2018
REC opinion
Further Information Favourable Opinion