New combo therapy targets genetic weakness in Hard-to-Treat leukemia
NCT ID NCT02677922
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study tests two experimental drugs (AG-120 and AG-221) that target specific genetic mutations (IDH1 or IDH2) found in some acute myeloid leukemia (AML) patients. Each drug is combined with a standard chemotherapy (azacitidine) and compared to azacitidine alone. The trial enrolls 130 newly diagnosed AML patients who cannot tolerate intensive chemotherapy. The goal is to find the safest and most effective dose and see if the combinations improve response rates.
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) or AG-221 (enasidenib) plus azacitidine
- What this could lead to
- If successful, this could provide a more effective, less intensive treatment option for older or frail patients with a specific genetic subtype of AML.
- What could go wrong
- This is an early-phase trial (1b/2) with a small number of participants, so results may not confirm benefit. Side effects from the drug combinations could be serious.
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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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Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02115, United States
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Local Institution - 102
Boston, Massachusetts, 02114, United States
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Local Institution - 103
Chicago, Illinois, 60637, United States
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Local Institution - 105
Duarte, California, 91010-301, United States
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Local Institution - 106
New York, New York, 10065, United States
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Local Institution - 107
New Haven, Connecticut, 06510, United States
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Local Institution - 108
Chicago, Illinois, 60611, United States
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Local Institution - 110
Dallas, Texas, 75390-85520, United States
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Local Institution - 125
Toronto, Ontario, M5G 2M9, Canada
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Local Institution - 175
Melbourne, 3141, Australia
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Local Institution - 177
Perth, 6000, Australia
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Local Institution - 178
Adelaide, South Australia, SA 5000, Australia
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Local Institution - 200
Paris, 75475, France
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Local Institution - 201
Villejuif, 94805, France
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Local Institution - 202
Pierre-Bénite, 69495, France
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Local Institution - 203
Toulouse, 31059, France
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Local Institution - 204
Pessac, 33604, France
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Local Institution - 205
Lille, 59037, France
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Local Institution - 206
Marseille, 13273, France
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Local Institution - 225
Ulm, 89081, Germany
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Local Institution - 227
Berlin, 12200, Germany
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Local Institution - 230
Dresden, 01307, Germany
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Local Institution - 250
Pesaro, 31122, Italy
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Local Institution - 251
Orbassano, 10043, Italy
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Local Institution - 252
Florence, 50134, Italy
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Local Institution - 253
Bologna, Emilia-Romagna, 40138, Italy
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Local Institution - 254
Padova, 35128, Italy
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Local Institution - 255
Roma, 00189, Italy
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Local Institution - 256
Genova, 16132, Italy
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Local Institution - 277
Utrecht, 3584 CX, Netherlands
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Local Institution - 327
Lisbon, 1169-050, Portugal
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Local Institution - 350
Seoul, 138-736, South Korea
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Local Institution - 351
Seoul, 135-710, South Korea
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Local Institution - 375
Barcelona, 08907, Spain
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Local Institution - 376
Cáceres, 10005, Spain
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Local Institution - 377
Valencia, 46009, Spain
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Local Institution - 378
Madrid, 28007, Spain
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Local Institution - 379
Barcelona, 08036, Spain
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Local Institution - 380
Málaga, 29010, Spain
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Local Institution - 381
Madrid, 28033, Spain
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Local Institution - 401
Zurich, 0, Switzerland
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Local Institution - 403
Basel, 4031, Switzerland
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Local Institution - 427
Headington, OX3 9DU, United Kingdom
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Local Institution - 428
London, SE5 9RS, United Kingdom
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Local Institution - 429
Birmingham, B15 2TH, United Kingdom
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Local Institution - 430
Sutton (Surrey), SM2 5PT, United Kingdom
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Local Institution - 902
Boston, Massachusetts, 02115, United States
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Local Institution - UNK-121
Yvoir, 5530, Belgium
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Local Institution - UNK-52
Gothenburg, 413 45, Sweden
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a fourth drug tip the scales against an aggressive leukemia?
- Double-Barreled attack: Gene-Edited stem cells and CAR T-Cells take on tough leukemia
- New drug combination targets rare childhood leukemia that resists standard treatment
- New combo aims to boost survival in older AML patients
- New combo therapy for AML enters early human testing
- New hope for older AML patients: triple drug combo aims to control disease and prevent relapse