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IDH1 Inhibitor

Ivosidenib + Venetoclax +/- Azacitidine for Leukemia

Portland, OR
Phase 1 & 2
Recruiting
Led By Courtney DiNardo
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age > 18 years
ECOG performance status of < 2
Must not have
Nursing women, women of childbearing potential (WOCBP) with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception. Appropriate highly effective method(s) of contraception include oral or injectable hormonal birth control, IUD, and double barrier methods (for example a condom in combination with a spermicide)
Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 3 years
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions
No Placebo-Only Group

Summary

This trial is testing the side effects and best dose of two drugs, venetoclax and ivosidenib, when given together with or without a third drug, azacitidine. The drugs may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

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Who is the study for?
This trial is for patients with IDH1-mutated blood cancers like AML, MDS, or MPN who are either treatment-naive and ineligible for standard chemotherapy or have relapsed/refractory disease. Participants must be in stable condition with acceptable organ function and agree to contraception. Those previously treated with ivosidenib/venetoclax, active infections (HBV/HCV/HIV), uncontrolled medical conditions, severe allergies to study drugs, or on certain medications are excluded.Check my eligibility
What is being tested?
The trial examines the effectiveness of combining venetoclax and ivosidenib with or without azacitidine in treating hematologic malignancies. It aims to determine the best dose while assessing side effects when these drugs work together compared to using just venetoclax and ivosidenib.See study design
What are the potential side effects?
Potential side effects include risks associated with blocking enzymes needed by cancer cells which may affect normal cell growth as well. Azacitidine as a chemotherapy drug can cause cell death leading to symptoms such as fatigue, nausea, bruising due to low blood counts, and increased risk of infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am older than 18 years.
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I can do most of my daily activities without help.
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My kidneys work well enough, with a creatinine clearance over 30 ml/min.
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Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am not pregnant, using effective birth control, or not of childbearing potential.
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I do not have severe stomach or metabolic issues affecting medication absorption.
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I have previously been treated with ivosidenib or venetoclax.
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I am not taking strong medication inducers like rifampin or St. John's wort within 3 days of starting the study.
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I am allergic to ivosidenib or venetoclax.
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I am currently being treated for another cancer.
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I do not have active hepatitis B, hepatitis C, or HIV.
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Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Dose-limiting toxicity
Incidence of adverse events
Overall response rate (ORR)
Secondary study objectives
Duration of response
Event-free survival
Overall survival
+1 more
Other study objectives
Peripheral blood and bone marrow aspirate samples
Plasma concentrations and pharmacokinetic parameter

Side effects data

From 2024 Phase 2 trial • 528 Patients • NCT03498521
38%
Anaemia
23%
Nausea
21%
Fatigue
15%
Asthenia
14%
Thrombocytopenia
12%
Neutropenia
12%
Constipation
11%
Pyrexia
10%
Abdominal pain
10%
Platelet count decreased
9%
Diarrhoea
9%
Neutrophil count decreased
9%
Vomiting
8%
Oedema peripheral
8%
Back pain
7%
White blood cell count decreased
7%
Weight decreased
7%
Hyponatraemia
5%
Leukopenia
5%
Hypothyroidism
5%
Decreased appetite
5%
Hypoalbuminaemia
5%
COVID-19
5%
Hypomagnesaemia
5%
Bone pain
5%
Insomnia
4%
Sepsis
3%
Atrial fibrillation
2%
Dyspnoea
2%
Renal failure
1%
Haemoptysis
1%
Gastroenteritis viral
1%
Pleural effusion
1%
Wrist fracture
1%
Hydronephrosis
1%
Wound infection
1%
Urosepsis
1%
Urinary tract infection
1%
Peritonitis
1%
Pneumonia
1%
Cardiac arrest
1%
Anxiety
1%
Delirium
1%
Cardio-respiratory arrest
1%
Respiratory tract infection
1%
Respiratory tract infection fungal
1%
Circulatory collapse
1%
Tumour associated fever
1%
Myalgia
1%
Cardiac failure chronic
1%
Pain
1%
Cerebral haemorrhage
1%
Febrile neutropenia
1%
Anal abscess
1%
Hip fracture
1%
Death
1%
General physical health deterioration
1%
Ascites
1%
Haemorrhoidal haemorrhage
1%
Abdominal sepsis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Category 2
Molecularly-Guided Therapy MGT) Category 1
Chemotherapy Category 1

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (venetoclax, ivosidenib, azacitidine)Experimental Treatment3 Interventions
Patients receive venetoclax PO daily on days 1-14. Patients also receive ivosidenib PO daily on days 15-28 of cycle 1 and days 1-28 of subsequent cycles. Patients may also receive azacitidine IV over 30-60 minutes or SC on days 1-7. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Azacitidine
FDA approved
Ivosidenib
FDA approved
Venetoclax
FDA approved

Find a Location

Closest Location:Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center· Cleveland, OH· 129 miles

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor
3,099 Previous Clinical Trials
1,812,459 Total Patients Enrolled
Courtney DiNardoPrincipal InvestigatorM.D. Anderson Cancer Center
8 Previous Clinical Trials
552 Total Patients Enrolled
Courtney DiNardo, MDPrincipal InvestigatorM.D. Anderson Cancer Center
13 Previous Clinical Trials
552 Total Patients Enrolled

Media Library

Ivosidenib (IDH1 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03471260 — Phase 1 & 2
Acute Myeloid Leukemia Research Study Groups: Treatment (venetoclax, ivosidenib, azacitidine)
Acute Myeloid Leukemia Clinical Trial 2023: Ivosidenib Highlights & Side Effects. Trial Name: NCT03471260 — Phase 1 & 2
Ivosidenib (IDH1 Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03471260 — Phase 1 & 2
~6 spots leftby Sep 2025