Ivosidenib maintenance after allogeneic stem cell transplant for IDH1‑mutant AML or high‑risk MDS

A Prospective, Single-arm, Open-label, Multicenter Phase II Trial Evaluating the Efficacy and Safety of Ivosidenib Maintenance Therapy After Allogeneic Stem Cell Transplantation (alloSCT) in Patients With Acute Myeloid Leukemia (AML) and Myelodysplastic Neoplasia (MD) Carrying an IDH1 Mutation

This will test whether taking ivosidenib after a recent allogeneic stem cell transplant helps people with AML or high‑risk MDS who have an IDH1 mutation stay in remission longer.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 76 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Technische Universität Dresden (other)
• Locations: 12 sites (Aachen and 11 other locations)
• Trial ID: NCT06717958 on ClinicalTrials.gov

What this trial studies

This phase 2 trial gives oral ivosidenib 250 mg daily as maintenance to patients with IDH1‑mutated acute myeloid leukemia (AML) or high‑risk myelodysplastic neoplasia (MDS) who had an allogeneic stem cell transplant within the past 100 days. Participants are monitored regularly for relapse, adverse events, and graft‑versus‑host disease, and those with severe active GVHD requiring >1 mg/kg steroids are excluded. The primary endpoint is event‑free survival, with secondary endpoints including safety and tolerability. The study is led by Technische Universität Dresden with sites in Aachen, Dresden, and Essen, Germany.

Who should consider this trial

Good fit: Adults with AML or high‑risk MDS who have a confirmed IDH1 mutation and received an allogeneic stem cell transplant within the previous 100 days and who do not have severe active GVHD requiring high‑dose steroids.

Not a fit: Patients without an IDH1 mutation, those with severe (grade III–IV) active graft‑versus‑host disease requiring >1 mg/kg steroids, or those beyond the ~100‑day post‑transplant window are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, ivosidenib maintenance could reduce relapse risk and extend relapse‑free survival after transplant for patients with IDH1‑mutated AML or high‑risk MDS.

How similar studies have performed: Ivosidenib is an approved targeted therapy that has shown activity in IDH1‑mutant AML, but its use as routine post‑transplant maintenance is relatively novel and not yet proven.

Eligibility criteria

Inclusion criterion:

* AML or MDS with IDH1 mutation

Exclusion criteria:

* active GvHD (graft vs host disease) grade III-IV according to Harris criteria requiring steroids \>1mg/ kg prednisolone equivalent

Where this trial is running

Aachen and 11 other locations

• Universitätsklinikum Aachen — Aachen, Germany (NOT_YET_RECRUITING)
• Technische Universitaet Dresden, Medizinische Klinik und Poliklinik I — Dresden, Germany (NOT_YET_RECRUITING)
• Universitätsklinikum Essen — Essen, Germany (NOT_YET_RECRUITING)
• Martin-Luther-Universitaet Halle-Wittenberg — Halle, Germany (RECRUITING)
• University Medical Center Hamburg-Eppendorf — Hamburg, Germany (NOT_YET_RECRUITING)
• Universitätsklinikum Jena — Jena, Germany (NOT_YET_RECRUITING)
• Universitätsklinikum Schleswig-Holstein — Kiel, Germany (RECRUITING)
• Universität Leipzig — Leipzig, Germany (NOT_YET_RECRUITING)
• Klinikum der Technischen Universitaet Muenchen (TUM Klinikum) — München, Germany (NOT_YET_RECRUITING)
• Universität Münster — Münster, Germany (NOT_YET_RECRUITING)
• Rostock University Medical Center — Rostock, Germany (NOT_YET_RECRUITING)
• Universitaetsklinikum Ulm — Ulm, Germany (NOT_YET_RECRUITING)

Study contacts

• Study coordinator: Jan Moritz Middeke, Dr. med.

Conditions: AML, MDS