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Can a Gene-Targeted pill turn the tide against a rare blood cancer?

NCT ID NCT03503409

Ongoing Disease control Sponsor: Groupe Francophone des Myelodysplasies Source: ClinicalTrials.gov ↗

First seen Aug 10, 2026 · Last updated Aug 11, 2026 · Updated 1 time

Summary

This trial is testing an oral drug called AG-120 (ivosidenib) in adults with myelodysplastic syndrome (MDS) or a related early form of leukemia that carries a specific IDH1 gene mutation. The drug is designed to block the mutated enzyme that drives abnormal blood cell growth. Researchers are looking to see if the treatment can improve blood cell counts and slow the progression of the disease in patients who have not responded to or have relapsed after standard therapy.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
AG-120 (ivosidenib), an oral drug that targets cancer cells with a specific IDH1 gene mutation
What this could lead to
If successful, this could offer a new targeted treatment option for people with myelodysplastic syndrome who have an IDH1 mutation and have not responded to standard therapy.
What could go wrong
This is a phase II trial, so the drug may not prove effective or safe in a larger population. Side effects are possible, and the benefit may be limited to certain patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ACUTE MYELOID LEUKEMIA MYELODYSPLASTIC SYNDROMES

Contacts and locations

Locations

  • CH Angers

    Angers, 49933, France

  • CH Le Mans/Service d'hématologie Oncologie

    Le Mans, 72000, France

  • CHRU de Limoges

    Limoges, 87046, France

  • CHU Brabois

    Vandœuvre-lès-Nancy, 54511, France

  • CHU Hôtel Dieu/Service d'Hématologie Clinique

    Nantes, 44093, France

  • CHU Montpellier St Eloi

    Montpellier, 34295, France

  • CHU côte de Nacre

    Caen, 14033, France

  • CHU de Grenoble/Clinique Universitaire d'hématologie 6e A

    Grenoble, 38043, France

  • CHU de Haut-Lévèque/Centre François Magendie/Service des maladies du sang

    Pessac, 33604, France

  • CHU de Poitiers/Pôle de cancérologie - secteur tertiaire-

    Poitiers, 86021, France

  • CHU de Tours

    Tours, 37044, France

  • CLINICA Ematologica ANCONA

    Ancona, 7160126, Italy

  • Centre Henri Becquerel

    Rouen, 76038, France

  • Centre Hospitalier de Versailles-Hôpital André Mignot

    Versailles, 78157, France

  • Centre Hospitalier de la Côte Basque

    Bayonne, 64109, France

  • Clinica Ematologica Genova

    Genova, 1016132, Italy

  • Ematologia ALESSANDRIA

    Alessandria, 16115121, Italy

  • Ematologia BOLOGNA

    Bologna, 40138, Italy

  • Ematologia BRESCIA

    Brescia, 125123, Italy

  • Ematologia FIRENZE

    Florence, 350134, Italy

  • Ematologia GENOVA

    Genova, 1016132, Italy

  • Ematologia LECCE

    Lecce, 73100, Italy

  • Ematologia MILANO

    Milan, 3520122, Italy

  • Ematologia ORBASSANO

    Orbassano, 1010043, Italy

  • Ematologia ROMA

    Roma, 1000144, Italy

  • Ematologia ed Immunologia Clinica PADOVA

    Padova, 235128, Italy

  • GHU Caremeau

    Nîmes, 30029, France

  • Hôpital Archet 1/Service d'Hématologie Clinique

    Nice, 06200, France

  • Hôpital E. Muller-GHR Mulhouse Sud-Alsace

    Mulhouse, 68100, France

  • Hôpital Henri Mondor

    Paris, 94010, France

  • Hôpital Necker

    Paris, 75743, France

  • Hôpital Nord Franche-Comté/Service de médecine interne / Hématologie clinique

    Belfort, 90015, France

  • Hôpital Saint Louis - Hématologie Séniors

    Paris, 75010, France

  • Institut Paoli Calmettes/Unité d'Hématologie 3

    Marseille, 13273, France

  • Médecine Interne/IUCT Oncopole

    Toulouse, 31059, France

  • Reggio Calabria

    Reggio Calabria, 2189124, Italy

  • centre hospitalier de Lyon

    Lyon, 69495, France

  • institut de cancérologie Lucien Neuwirth

    Saint-Priest-en-Jarez, 42271, France

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