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New hope for rare MDS: targeted drug ivosidenib takes on standard therapy

NCT ID NCT06465953

Disease control Sponsor: Institut de Recherches Internationales Servier Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Sep 04, 2026 · Updated 5 times

Summary

This study is for people with a rare form of myelodysplastic syndromes (MDS) that has a specific genetic change called IDH1 mutation. Participants have not had prior treatment with certain drugs. They will be randomly assigned to receive either ivosidenib (a daily pill) or azacitidine (injections for 7 days each month). The goal is to see which drug helps control the disease better, looking at remission rates and overall survival.

What this could mean

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

Active substance
Ivosidenib (a targeted drug) and azacitidine (a chemotherapy-like drug)
What this could lead to
If it works, this could point toward a more effective treatment for a specific type of MDS, potentially improving remission rates.
What could go wrong
This is a small, early-phase 3 trial with only 48 participants. The drug may not work better than the standard treatment, and side effects are possible.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 48 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2024

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of HMA naive IDH1 R132 mutated MDS defined according to WHO criteria (5th edition): * Moderate high, high and very high-risk MDS per IPSS-M score will be eligible regardless of blood counts and with blast counts 0-19%. * Low and moderate low-risk MDS per IPSS-M score must: * Have cytopenias related to MDS, defined as: \<100 platelets/microliter, or absolute neutrophil count (ANC) \<1000/mm3, or hemoglobin \<10g/dL AND * Have a blast count between 5-19% AND * Be eligible for HMA therapy (very low risk participants are to be excluded) * Locally or centrally confirmed IDH1 R132 C/G/H/L/S mutation Exclusion Criteria: * Received prior anticancer/disease modifying treatment for MDS (including HMA's, cytotoxic chemotherapy, investigational agents, bcl-2 inhibitor based-regimens, hematopoietic stem cell transplant (HSCT), IDH1 inhibitors). For LR-MDS patients, prior treatment with growth factors, luspatercept, lenalidomide, and imetelstat are allowed. * \>20% blasts by morphology or immunohistochemistry on screening bone marrow aspirate/biopsy

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Conditions

The condition(s) this trial relates to.

myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

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

Hypomethylating agent (HMA) naive myelodysplastic syndromes (MDS) Myelodysplastic syndromes (MDS)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    61 sites in 10 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino - Presidio Molinette

    RECRUITING

    Torino, 10126, Italy

  • Azienda Ospedaliero Universitaria Delle Marche

    RECRUITING

    Ancona, 60126, Italy

  • Azienda Ospedaliero-Universitaria Careggi

    RECRUITING

    Florence, 50139, Italy

  • Calvary Mater Newcastle

    RECRUITING

    Waratah, 2298, Australia

  • Casa de Saúde Santa Marcelina

    RECRUITING

    São Paulo, 08270-120, Brazil

  • Centro de Pesquisa Clínica - Hospital Nove de Julho

    RECRUITING

    São Paulo, 01308-070, Brazil

  • Centro de Pesquisas Clínicas da Fundação Doutor Amaral Carvalho

    RECRUITING

    São Paulo, 17210-190, Brazil

  • Chu Bordeaux, Hopital Du Haut Leveque

    RECRUITING

    Pessac, 33600, France

  • Chu Brabois

    RECRUITING

    Vandœuvre-lès-Nancy, 54500, France

  • Chu Nantes-Hotel Dieu

    RECRUITING

    Nantes, 44093, France

  • Chu de Nice - Hôpital L'Archet 1

    RECRUITING

    Nice, 062000, France

  • Churchill Hospital

    RECRUITING

    Oxford, OX3 7LE, United Kingdom

  • Clinica Universitaria de Navarra (Madrid)

    RECRUITING

    Madrid, 28027, Spain

  • Clinica Universitaria de Navarra (Pamplona)

    RECRUITING

    Pamplona, 31008, Spain

  • Dipartimento Di Biomedicina E Prevenzione - Universita Degli Studi Di Roma "Tor Vergata"

    RECRUITING

    Roma, 00133, Italy

  • Fondazione I.R.C.C.S. Policlinico San Matteo

    RECRUITING

    Pavia, 27100, Italy

  • H. Universitario La Fe

    RECRUITING

    Valencia, 46026, Spain

  • H. Valle de Hebron

    RECRUITING

    Barcelona, 8035, Spain

  • Hopital Saint Louis

    RECRUITING

    Paris, 75010, France

  • Hospital Clinico Universitario de Salamanca

    RECRUITING

    Salamanca, 37007, Spain

  • Hospital Universitario Virgen de La Macarena

    RECRUITING

    Seville, 41009, Spain

  • Hospital das Clínicas da Faculdade de Medicina da USP

    RECRUITING

    São Paulo, 05403-010, Brazil

  • Humanitas Research Hospital (Istituto Clinico Humanitas)

    RECRUITING

    Milan, 20089, Italy

  • Institut Catala D' Oncologia

    RECRUITING

    Badalona, 8916, Spain

  • Institut Universitaire Du Cancer Toulouse-Oncopole

    RECRUITING

    Toulouse, 31059, France

  • Instituto Nacional do Câncer

    NOT_YET_RECRUITING

    São Paulo, 20231-050, Brazil

  • Istituto Di Ematologia "Lorenzo E Ariosto Seragnoli" - Policlinico Di S. Orsola

    RECRUITING

    Bologna, 40138, Italy

  • Japanese Red Cross Musashino Hospital

    RECRUITING

    Musashino-city, Tokyo, 1808610, Japan

  • Japanese Red Cross Society Himeji Hospital

    RECRUITING

    Himeji-city, Hyogo, 6708540, Japan

  • Kings College Hospital

    RECRUITING

    London, United Kingdom

  • Kitasato University Hospital

    NOT_YET_RECRUITING

    Sagamihara, 252-0375, Japan

  • Kyushu University Hospital

    RECRUITING

    Higashi-ku, Fukuoka-city, Fukuoka, 8128582, Japan

  • Liga Paranaense de Combate ao Câncer - Hospital Erasto Gaertner

    RECRUITING

    Curitiba, 81520-060, Brazil

  • Liverpool Hospital

    NOT_YET_RECRUITING

    Liverpool, 2170, Australia

  • MD Anderson Cancer Centre

    RECRUITING

    Houston, Texas, 77030, United States

  • MSKCC

    RECRUITING

    New York, New York, 10065, United States

  • Marien Hospital Duesseldorf

    RECRUITING

    Düsseldorf, 40477, Germany

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Monash Health

    RECRUITING

    Clayton, 3168, Australia

  • Northern Health

    RECRUITING

    Epping, 3076, Australia

  • Ohio State University Comprehensive Cancer Center

    RECRUITING

    Columbus, Ohio, 43210, United States

  • Oncology Associates of Oregon

    RECRUITING

    Eugene, Oregon, 97401, United States

  • Presbyterian / St. Luke'S Medical Center

    RECRUITING

    Denver, Colorado, 80218, United States

  • Real E Benemérita Associação Portuguesa de São Paulo

    RECRUITING

    São Paulo, 01321-001, Brazil

  • Royal Adelaide Hospital

    NOT_YET_RECRUITING

    Adelaide, 5000, Australia

  • Sir Charles Gairdner Hospital

    RECRUITING

    Nedlands, 6009, Australia

  • Sociedade Beneficente Israelita Brasileira Hospital Albert Einstein

    RECRUITING

    São Paulo, 05652-900, Brazil

  • St James' University Hospital

    WITHDRAWN

    Leeds, LS9 7TF, United Kingdom

  • Tokai University Hospital

    RECRUITING

    Isehara-city, Kanagawa, 2591193, Japan

  • Torbay Hospital

    RECRUITING

    Torquay, TQ2 7AA, United Kingdom

  • Tum Klinikum Rechts Der Isar

    RECRUITING

    Munich, 81675, Germany

  • Umc Amsterdam - Vumc

    RECRUITING

    Amsterdam, 1081HV, Netherlands

  • Umc Groningen

    RECRUITING

    Groningen, 9713GZ, Netherlands

  • Unc Lineberger Comprehensive Cancer Center

    RECRUITING

    Chapel Hill, North Carolina, 27514, United States

  • Universitaetsklinikum Leipzig

    RECRUITING

    Leipzig, 04103, Germany

  • Universitaetsmedizin Goettingen (Umg)

    RECRUITING

    Göttingen, 37075, Germany

  • Universitatsklinikum Dresden Carl Gustav Carus

    RECRUITING

    Dresden, 01307, Germany

  • University College London Hospital

    RECRUITING

    London, NW1 2PG, United Kingdom

  • University of Chicago, Duchossois Center for Advanced Medicine (DCAM)

    RECRUITING

    Chicago, Illinois, 60637, United States

  • University of Fukui Hospital

    RECRUITING

    Yoshida-gun, Eiheiji-cho 670-8540 Himeji, 910-1193, Japan

  • University of Texas UT Southwestern Comprehensive Cancer Center

    RECRUITING

    Dallas, Texas, 75235, United States

  • Western General Hospital

    RECRUITING

    Edinburgh, EH4 2XU, United Kingdom

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