About the PyramIDH trial

PyramIDH is the name of a randomised clinical study designed to measure the efficacy of Ivosidenib, an oral inhibitor which specifically targets the gene mutation IDH1, compared with the commonly used hypomethylating agent (HMA) Azacitidine (often referred to as Aza).   

Why this study matters

Following promising results from phase 2 of the study, which showed a 72% overall response rate for Ivosidenib (as a monotherapy, or single treatment) in patients with previously untreated  IDH1-mutant MDS,  the aim of this third phase is to further validate the findings by testing a wider cohort of patients.   

Trial design

The trial managers are looking to recruit approximately 48 MDS patients with the IDH-1 mutation who have not previously been treated with hypomethylating agents.  They will be split randomly into two groups, with the patients in one group to be treated with Ivosidenib and the other group Azacitidine.   

Who can take part?

• You are 18 or older and have been diagnosed with MDS with the genetic mutation IDH-1
• You have not yet been treated with hypomethylating agents
• You live within reasonable travelling distance of one of the trial centres listed below

UK trial sites and contacts

• Churchill Hospital, Oxford – Contact: Dr Connor Sweeney
• King’s College Hospital, London – Contact: Dr Lynn Quek
• University College Hospital, London - Dr Rob Sellar
• Western General Hospital, Edinburgh – Contact: Dr Victoria Campbell
• Torbay Hospital, Torbay – Contact: Dr Zhao Rui
• St James’s Hospital, Leeds – Contact: Dr Catherine Cargo