PyramIDH Clinical Trial

About the PyramIDH Trial

What is the PyramIDH Trial?

Patients with isocitrate dehydrogenase 1 (IDH1)-mutated newly diagnosed MDS have increased risk of transformation to AML, worse event-free survival (EFS), and reduced OS when compared with patients with wild-type IDH1. Treatment options for such patients are limited. The Phase III PyramIDH clinical trial, sponsored by Servier, is investigating ivosidenib, an oral, targeted inhibitor of mutant IDH1 enzymes, to assess its efficacy and safety as a potential new treatment for this patient population.

Important Attributes

Partnership with Tempus TIME sites in the U.S.
All participants will receive either ivosidenib or azacitidine for their MDS – No placebo group
Pre-screening (blood sampling) to determine mIDH1 status is available centrally through the trial if the local result is unknown
Travel support is offered at most of participating sites

Study Objectives

Assess the efficacy of ivosidenib based on participants’ response per IWG 2006 and IWG 2023 criteria
Evaluate the safety and tolerability profile of ivosidenib in patients with IDH1-mutated HMA naïve MDS
Analyze the impact of ivosidenib on health-related quality of life (HRQoL) and health economic outcomes.
Characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of ivosidenib.

Key Eligibility Criteria

HMA naive IDH1-mutated MDS as per WHO criteria:
- For moderate high-, to very high-risk MDS per IPSS-M, have a score with blast counts 0-19%
- For low- and moderate low-risk MDS per IPSS-M scores, have cytopenias, blast counts between 5-19% and are eligible for HMA therapy
Locally or centrally confirmed IDH1 mutation
Have ECOG PS of 0 to 3
Not had prior therapies aimed at modifying MDS disease, including IDH1 inhibitor
Not have >20% blasts on screening BMA

Clinical Trial Sites in U.S.

Site Locations:	Principal Investigator
Massachusetts General Hospital	Dr. A. Brunner
OSU Comprehensive Cancer Center	Dr. U. Borate
MD Anderson Cancer Center	Dr. G. Garcia-Manero
UT Southwestern Medical Center	Dr. Y. Madanat
University of Chicago Medicine	Dr. T. Odenike
Memorial Sloan Kettering Cancer Center New York – Main Hospital	Dr. T. Haque
UNC Lineberger Comprehensive Cancer Center	Dr. Williams
Willamette Valley Cancer Institute and Research Center – Eugene	Dr. L. Fletcher
Colorado Blood Cancer Institute	Dr. M. Rotta

To refer a patient to a participating site, please contact the nearest site to you or Servier at scientificinformation@servier.com.

US-MED-01076 03/05/26