A Phase 3 interventional study of Ivosidenib and Azacitidine in Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) and Myelodysplastic Syndromes (MDS), sponsored by Institut de Recherches Internationales Servier. Recruiting at 62 sites in 10 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-03.
Sponsored by Institut de Recherches Internationales Servier · Phase 3, Interventional, and Treatment
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.
1,997 studies on the registry are indexed under Myelodysplastic Syndromes; 324 are open to participants now.
This study's planned enrollment of 48 is above the median of 40 across 1,611 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Institut de Recherches Internationales Servier is the lead sponsor of 67 studies on the registry; 4 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 9 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Ivosidenib
Drug: Azacitidine
Two 250 mg tablets, totaling 500 mg, administered orally once daily until disease relapse or progression, unacceptable toxicity, confirmed pregnancy, undergoing HSCT, death, withdrawal of consent, lost to follow-up, or Sponsor ending the study, whichever occurs first.
Azacitidine 75mg/m\^2/day administered by subcutaneous (SC) or intravenous (IV) injection for 1 week (7 days) of each 4-week (28 day) treatment cycle until disease relapse or progression, unacceptable toxicity, confirmed pregnancy, undergoing HSCT, death, withdrawal of consent, lost to follow-up, or Sponsor ending the study, whichever occurs first.
Number of participants achieving CR and PR by 4 months
Complete remission (CR) or Partial remission (PR) as per International Working Group (IWG) 2006 criteria
Time frame: Through 4 months after starting treatment
Overall Response (OR) rate per IWG 2023 criteria
Defined as CR (or CR equivalent) + PR + CRL + CRh + hematological improvement (HI)
Time frame: Through the end of the study (approximately 4 years)
Event-free survival (EFS)
Defined as the date of randomization to the date of first documented confirmed relapse /progression /death, whichever occurs first
Time frame: Through the end of the study (approximately 4 years)
Overall Survival (OS)
Defined as the time from randomization to the date of death due to any cause. Participants who are alive at the analysis cutoff date will be censored at the date they were last known to be alive.
Time frame: Through the end of the study (approximately 4 years)
Duration of CR and PR
Among participants who achieved CR+PR per IWG 2006 criteria
Time frame: Through the end of the study (approximately 4 years)
Time to CR and PR
Defined as time from the date of the randomization to the date of CR+PR, among participants who achieve CR+PR based on IWG 2006 Response Criteria
Time frame: Through the end of the study (approximately 4 years)
Acute myeloid leukemia (AML) transformation rate
Time frame: Through the end of the study (approximately 4 years)
Time to transfusion independence (TTTI)
Defined as time from date of randomization to date transfusion independence (TI) is first observed (Day 1 of a ≥ 56 days period without a transfusion), among participants who are baseline transfusion dependent and have achieved post-baseline TI. In the event a participant had more than one ≥ 56-day period, which met TI criteria, the earliest period will be used in analysis.
Time frame: Through the end of the study (approximately 4 years)
Duration of transfusion independence (DOTI)
Among participants who have achieved post-baseline TI, DOTI will be calculated as the time from the date TI is first observed (Day 1 of a ≥ 56-day period without a transfusion) until the day before the participants had a subsequent transfusion.
Time frame: Through the end of the study (approximately 4 years)
Transfusion independence rate
Time frame: Through the end of the study (approximately 4 years)
Change from baseline in Quality of life (QOL) based on the QUALMS score
Quality of Life in Myelodysplasia Scale (QUALMS) scores range from 0 to 100, with a higher score representing a better QOL.
Time frame: Through the Event Free Survival Follow up (approximately 4 years)
Change from baseline in health economic outcomes measures based on EQ-5D-5L score
Health economic outcomes measures as assessed by the 5-level EuroQol five dimensions questionnaire (EQ-5D-5L) scores range from 5 to 25 with a higher number representing a worse health status.
Time frame: Through the Event Free Survival Follow up (approximately 4 years)
Number of participants who proceed to hematopoietic stem cell transplantation (HSCT)
Time frame: Through the end of the study (approximately 4 years)
Ivosidenib plasma concentrations
For participants receiving ivosidenib monotherapy
Time frame: Through Cycle 22 (each cycle is 28 days)
2-HG plasma concentrations
For participants receiving ivosidenib monotherapy
Time frame: Through Cycle 22 (each cycle is 28 days)
Number of participants achieving CR and PR by 6 months as per IWG 2006 criteria
Time frame: Through 6 months after starting treatment
Number of participants achieving CR and PR by 6 months as per IWG 2023 criteria
Time frame: Through 6 months after starting treatment
Number of participants achieving CR and PR by 4 months as per IWG 2023 criteria
Time frame: Through 4 months after starting treatment
Number of adverse events (AEs) and serious adverse events (SAEs)
Time frame: Through the Safety Follow-up Visit (30-35 days after discontinuation of treatment)
Not enrolling at this site — choose another above.
Plan to share: Yes — Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in patients: sponsored by Servier with a first patient enrolled as of 1 January 2004 onwards for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
Time frame: After Marketing Authorization in EEA or US if the study is used for the approval.
Access criteria: Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.
Data sharing details ↗ · clinicaltrials.servier.com
No publications or documents are linked to this record.
Started Dec 2024 (actual). Primary completion is estimated for Nov 2027, 34 months after the start.
Of 45 phase 3 interventional studies under Myelodysplastic Syndromes, half reached primary completion within 50 months of starting (the median), counting those still running; a quarter within 38 and three quarters within 95. This study's planned 34 months is below that.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drugs tested here: Ivosidenib · Azacitidine
Institut de Recherches Internationales Servier