Actively Recruiting

Phase 3
Age: 18Years +
All Genders
NCT06465953

Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation

Led by Institut de Recherches Internationales Servier · Updated on 2026-05-11

48

Participants Needed

62

Research Sites

208 weeks

Total Duration

On this page

Sponsors

I

Institut de Recherches Internationales Servier

Lead Sponsor

S

Servier Bio-Innovation LLC

Collaborating Sponsor

AI-Summary

What this Trial Is About

This study will enroll participants with myelodysplastic syndromes (MDS) with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have not received treatment with a hypomethylating agent previously. Participants will be randomized to receive either ivosidenib (IVO) alone or azacitidine (AZA) alone. IVO will be administered daily throughout the 28-day treatment cycle and AZA will be administered for the first 7 days of each 28-day cycle. Study visits will be conducted every week during Cycle 1 (Days 1, 8, 15, and 22), and Day 1 of each cycle thereafter. After the last dose of treatment, participants will attend an safety follow-up visit and participants will be followed to assess overall survival. Study visits may include a bone marrow aspirate, physical exam, echocardiogram (ECHO), electrocardiogram (ECG), blood and urine analysis, and questionnaires.

CONDITIONS

Official Title

Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of hypomethylating agent naive IDH1 R132 mutated MDS as defined by WHO criteria (5th edition)
  • Moderate high, high, or very high-risk MDS per IPSS-M score with blast counts 0-19%
  • For low and moderate low-risk MDS per IPSS-M score: have cytopenias related to MDS (platelets <100/microliter, or ANC <1000/mm3, or hemoglobin <10g/dL), blast count between 5-19%, and be eligible for HMA therapy
  • Locally or centrally confirmed IDH1 R132 C/G/H/L/S mutation
Not Eligible

You will not qualify if you...

  • Prior treatment for MDS including hypomethylating agents, cytotoxic chemotherapy, investigational agents, bcl-2 inhibitor regimens, hematopoietic stem cell transplant, or IDH1 inhibitors (growth factors, luspatercept, lenalidomide, and imetelstat allowed for low-risk MDS patients)
  • More than 20% blasts by morphology or immunohistochemistry on screening bone marrow aspirate or biopsy

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 62 locations

1

Presbyterian / St. Luke'S Medical Center

Denver, Colorado, United States, 80218

Not Yet Recruiting

2

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

Chicago, Illinois, United States, 60637

Actively Recruiting

3

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Actively Recruiting

4

MSKCC

New York, New York, United States, 10065

Actively Recruiting

5

Unc Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, United States, 27514

Actively Recruiting

6

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, United States, 43210

Actively Recruiting

7

Oncology Associates of Oregon

Eugene, Oregon, United States, 97401

Actively Recruiting

8

University of Texas UT Southwestern Comprehensive Cancer Center

Dallas, Texas, United States, 75235

Not Yet Recruiting

9

MD Anderson Cancer Centre

Houston, Texas, United States, 77030

Actively Recruiting

10

Royal Adelaide Hospital

Adelaide, Australia, 5000

Not Yet Recruiting

11

Monash Health

Clayton, Australia, 3168

Actively Recruiting

12

Northern Health

Epping, Australia, 3076

Actively Recruiting

13

Liverpool Hospital

Liverpool, Australia, 2170

Not Yet Recruiting

14

Sir Charles Gairdner Hospital

Nedlands, Australia, 6009

Actively Recruiting

15

Calvary Mater Newcastle

Waratah, Australia, 2298

Actively Recruiting

16

Liga Paranaense de Combate ao Câncer - Hospital Erasto Gaertner

Curitiba, Brazil, 81520-060

Actively Recruiting

17

Centro de Pesquisa Clínica - Hospital Nove de Julho

São Paulo, Brazil, 01308-070

Actively Recruiting

18

Real E Benemérita Associação Portuguesa de São Paulo

São Paulo, Brazil, 01321-001

Actively Recruiting

19

Hospital das Clínicas da Faculdade de Medicina da USP

São Paulo, Brazil, 05403-010

Not Yet Recruiting

20

Sociedade Beneficente Israelita Brasileira Hospital Albert Einstein

São Paulo, Brazil, 05652-900

Actively Recruiting

21

Casa de Saúde Santa Marcelina

São Paulo, Brazil, 08270-120

Not Yet Recruiting

22

Centro de Pesquisas Clínicas da Fundação Doutor Amaral Carvalho

São Paulo, Brazil, 17210-190

Not Yet Recruiting

23

Instituto Nacional do Câncer

São Paulo, Brazil, 20231-050

Not Yet Recruiting

24

Chu Nantes-Hotel Dieu

Nantes, France, 44093

Actively Recruiting

25

Chu de Nice - Hôpital L'Archet 1

Nice, France, 062000

Actively Recruiting

26

Hopital Saint Louis

Paris, France, 75010

Actively Recruiting

27

Chu Bordeaux, Hopital Du Haut Leveque

Pessac, France, 33600

Actively Recruiting

28

Institut Universitaire Du Cancer Toulouse-Oncopole

Toulouse, France, 31059

Actively Recruiting

29

Chu Brabois

Vandœuvre-lès-Nancy, France, 54500

Actively Recruiting

30

Universitatsklinikum Dresden Carl Gustav Carus

Dresden, Germany, 01307

Actively Recruiting

31

Marien Hospital Duesseldorf

Düsseldorf, Germany, 40477

Actively Recruiting

32

Universitaetsmedizin Goettingen (Umg)

Göttingen, Germany, 37075

Actively Recruiting

33

Universitaetsklinikum Leipzig

Leipzig, Germany, 04103

Actively Recruiting

34

Tum Klinikum Rechts Der Isar

Munich, Germany, 81675

Actively Recruiting

35

Azienda Ospedaliero Universitaria Delle Marche

Ancona, Italy, 60126

Actively Recruiting

36

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

Bologna, Italy, 40138

Actively Recruiting

37

Azienda Ospedaliero-Universitaria Careggi

Florence, Italy, 50139

Actively Recruiting

38

Humanitas Research Hospital (Istituto Clinico Humanitas)

Milan, Italy, 20089

Actively Recruiting

39

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

Pavia, Italy, 27100

Actively Recruiting

40

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

Roma, Italy, 00133

Actively Recruiting

41

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

Torino, Italy, 10126

Actively Recruiting

42

University of Fukui Hospital

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

Actively Recruiting

43

Kyushu University Hospital

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

Actively Recruiting

44

Japanese Red Cross Society Himeji Hospital

Himeji-city, Hyogo, Japan, 6708540

Actively Recruiting

45

Tokai University Hospital

Isehara-city, Kanagawa, Japan, 2591193

Actively Recruiting

46

Japanese Red Cross Musashino Hospital

Musashino-city, Tokyo, Japan, 1808610

Actively Recruiting

47

Kitasato University Hospital

Sagamihara, Japan, 252-0375

Not Yet Recruiting

48

Umc Amsterdam - Vumc

Amsterdam, Netherlands, 1081HV

Actively Recruiting

49

Umc Groningen

Groningen, Netherlands, 9713GZ

Actively Recruiting

50

Institut Catala D' Oncologia

Badalona, Spain, 8916

Actively Recruiting

51

H. Valle de Hebron

Barcelona, Spain, 8035

Actively Recruiting

52

Clinica Universitaria de Navarra (Madrid)

Madrid, Spain, 28027

Actively Recruiting

53

Clinica Universitaria de Navarra (Pamplona)

Pamplona, Spain, 31008

Actively Recruiting

54

Hospital Clinico Universitario de Salamanca

Salamanca, Spain, 37007

Actively Recruiting

55

Hospital Universitario Virgen de La Macarena

Seville, Spain, 41009

Actively Recruiting

56

H. Universitario La Fe

Valencia, Spain, 46026

Actively Recruiting

57

Western General Hospital

Edinburgh, United Kingdom, EH4 2XU

Actively Recruiting

58

St James' University Hospital

Leeds, United Kingdom, LS9 7TF

Withdrawn

59

University College London Hospital

London, United Kingdom, NW1 2PG

Actively Recruiting

60

Kings College Hospital

London, United Kingdom

Actively Recruiting

61

Churchill Hospital

Oxford, United Kingdom, OX3 7LE

Actively Recruiting

62

Torbay Hospital

Torquay, United Kingdom, TQ2 7AA

Actively Recruiting

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Research Team

I

Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies Department

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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