Actively Recruiting
A Phase 3 Randomized Study of Ivosidenib or Azacitidine Alone in Adults With IDH1 Mutated Myelodysplastic Syndromes Who Are New to Hypomethylating Agent Treatment
Led by Institut de Recherches Internationales Servier · Updated on 2026-05-11
48
Participants Needed
62
Research Sites
56 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
Researchers are studying treatments for adults with myelodysplastic syndromes (MDS) who have a specific mutation in the IDH1 protein and have never been treated with hypomethylating agents. The trial compares two treatments: ivosidenib (IVO) alone and azacitidine (AZA) alone. This Phase 3, randomized, open-label study aims to understand how these treatments work for patients with different risk levels of MDS, focusing on how many achieve complete or partial responses within four months. Participants will be randomly assigned to receive either ivosidenib orally each day during 28-day cycles or azacitidine by injection for the first 7 days of each 28-day cycle. Treatment continues until disease relapse, unacceptable side effects, pregnancy, stem cell transplant, or other specified reasons. Study visits occur weekly during the first cycle and then on day 1 of each subsequent cycle. After treatment ends, participants have a safety follow-up visit and are monitored for overall survival. During the study, participants undergo various assessments including bone marrow aspirates, physical exams, heart tests like ECHO and ECG, blood and urine tests, and questionnaires about quality of life. Researchers measure the number of participants achieving complete or partial responses by 4 months as the primary outcome. Secondary outcomes include overall response rates, survival times, transformation to leukemia, transfusion independence, quality of life changes, and drug levels in the blood. Safety is monitored through adverse event reporting, and the study lasts about four years to gather long-term data.
CONDITIONS
Brief 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
Eligibility Criteria
You may qualify if you...
- Diagnosed with IDH1 R132 mutated myelodysplastic syndromes (MDS) per WHO criteria
- Moderate high, high, or very high-risk MDS per IPSS-M score, any blood counts, blast counts 0-19%
- Low and moderate low-risk MDS must have cytopenias (platelets <100, ANC <1000/mm3, or hemoglobin <10g/dL) and blast counts 5-19%
- Eligible for hypomethylating agent therapy
- Age 18 years or older
- Locally or centrally confirmed IDH1 R132 C/G/H/L/S mutation
You will not qualify if you...
- Prior treatment for MDS with hypomethylating agents, chemotherapy, investigational agents, bcl-2 inhibitor regimens, hematopoietic stem cell transplant, or IDH1 inhibitors
- Blast count greater than 20% by bone marrow biopsy or aspirate
AI-Screening
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Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease relapse or discontinuation
Participants receive either Ivosidenib orally once daily or Azacitidine by injection for 1 week of each 4-week cycle until disease relapse, unacceptable toxicity, or other specified reasons.
Monthly visits for treatment cycles
Duration - 30 to 35 days after treatment discontinuation
Participants are followed for safety and long-term outcomes after treatment ends.
1 safety follow-up visit
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
Research Team
I
Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies Department
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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