Can a targeted pill turn the tide against a bone marrow disorder?
NCT ID NCT03744390
First seen Aug 10, 2026 · Last updated Aug 11, 2026 · Updated 1 time
Summary
This trial is testing an oral drug called enasidenib (AG-221) in people with myelodysplastic syndrome (MDS) who have a specific genetic change called an IDH2 mutation. The drug is designed to block the mutated enzyme and help restore normal blood cell production. Participants receive continuous 28-day cycles of the drug, and researchers will measure how well it improves blood counts and slows disease progression. The study includes adults with higher-risk MDS or certain types of acute myeloid leukemia that have not responded to prior treatment.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Enasidenib (AG-221), an oral drug that targets the IDH2 mutation
- What this could lead to
- If effective, enasidenib could offer a new treatment option for people with myelodysplastic syndrome who have not responded to standard therapies, potentially improving blood counts and delaying progression to leukemia.
- What could go wrong
- This is a phase II trial with a modest number of participants, so results may not be definitive. The drug may cause side effects, and not all patients may respond.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CH Le Mans/Service d'hématologie Oncologie
Le Mans, 72000, France
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CH d'Angers/Service des Maladies du sang
Angers, 49933, France
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CH de la Cote Basque
Bayonne, 64109, France
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CH lyon
Lyon, 69495, France
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CHU Côte de Nacre/Service d'Hématologie Clinique
Caen, 14033, France
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CHU Montpellier St Eloi
Montpellier, Montpellier, 34295, France
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CHU Nantes - Hôtel Dieu/Service d'Hématologie Clinique
Nantes, 44093, France
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CHU de Bordeaux
Bordeaux, 33604, France
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CHU de Grenoble
Grenoble, 38043, France
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CHU de Nimes
Nîmes, 30029, France
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CHU de Tours
Tours, 37044, France
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Centre Henri Becquerel/Département d'Hématologie
Rouen, 76 038, France
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GHR Mulhouse Sud-Alsace
Mulhouse, 68100, France
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Hôpital André Mignot
Versailles, LE Chesnay, 78157, France
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Hôpital Archet 1/Service d'Hématologie Clinique
Nice, 06202, France
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Hôpital Henri Mondor
Créteil, 94010, France
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Hôpital Saint Louis - Service d'hématologie séniors
Paris, 75010, France
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Hôpital saint Antoine
Paris, 75571, France
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Institut Paoli Calmettes/Unité d'Hématologie 3
Marseille, 13009, France
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Institut de cancérologie Lucien Neuwirth Saint priest en Jarez
Saint-Priest-en-Jarez, 42271, France
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Médecine Interne/IUCT Oncopole
Toulouse, 31059, France
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centre hospitalier Victor Dupouy
Argenteuil, 95107, France
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