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Checkpoint Inhibitor

Nivolumab + Ipilimumab for Acute Myeloid Leukemia or Myelodysplastic Syndrome

Phase 1

Waitlist Available

Led By Gheath Al-Atrash

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

Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study until 3 months after the last treatment

Patients must have received preparative regimens to include either busulfan- or melphalan-based regimens

Must not have

Patients with a known history of any of the following autoimmune diseases are excluded: Patients with a history of inflammatory bowel disease (including Crohn's disease and ulcerative colitis), Patients with a history of rheumatoid arthritis, systemic progressive sclerosis (scleroderma), systemic lupus erythematosus, autoimmune vasculitis (e.g., Wegener's granulomatosis), Patients with solid organ allografts (such as renal transplant) are excluded, Ongoing immunosuppressive therapy for the treatment of GVHD. Patients receiving GVHD prophylaxis will be allowed on this study, Patients with symptomatic central nervous system (CNS) leukemia at the time of evaluation or patients with poorly controlled CNS leukemia, Active and uncontrolled disease/(active uncontrolled infection, uncontrolled hypertension despite adequate medical therapy, active and uncontrolled congestive heart failure New York Heart Association [NYHA] class III/IV, clinically significant and uncontrolled arrhythmia) as judged by the treating physician, Patients with known human immunodeficiency virus seropositivity will be excluded, Known to be positive for hepatitis B by surface antigen expression. Known to have active hepatitis C infection (positive by polymerase chain reaction or on antiviral therapy for hepatitis C within the last 6 months), Any other medical, psychological, or social condition that may interfere with study participation or compliance, or compromise patient safety in the opinion of the investigator, Patients unwilling or unable to comply with the protocol, Pregnant or breastfeeding

Patients with acute GVHD > grade 2 at any time during the post-transplant course

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 year

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trialtests immunotherapy with monoclonal antibodies to treat high risk acute myeloid leukemia or myelodysplastic syndrome that has not responded to treatment.

Who is the study for?

This trial is for patients with high-risk, treatment-resistant or relapsed acute myeloid leukemia or myelodysplastic syndrome after a donor stem cell transplant. Participants must have stable vital organ functions, agree to contraception if of childbearing potential, and not have severe graft-versus-host disease, uncontrolled illnesses, known allergies to the study drugs, certain autoimmune diseases, active infections like HIV or hepatitis C.

What is being tested?

The trial is testing the safety and optimal doses of two immunotherapy drugs—nivolumab and ipilimumab—administered post-stem cell transplant. These monoclonal antibodies are designed to boost the immune system's ability to fight cancer by targeting tumor cells' growth and spread mechanisms.

What are the potential side effects?

Potential side effects include immune-related reactions that can affect organs (like inflammation), infusion-related symptoms (such as fever or chills), fatigue, skin rash, hormonal gland issues (like thyroid dysfunction), digestive problems (colitis), liver enzyme elevation which may indicate liver inflammation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a male and will use birth control during and for 3 months after the study.

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I have been treated with busulfan or melphalan before my transplant.

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My white blood cell count has been stable for 3 days.

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I am not able to have children, am postmenopausal, or have a negative pregnancy test.

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My AML or MDS has returned or didn't respond after a stem cell transplant.

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I can take care of myself but might not be able to do heavy physical work.

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My kidney function is within the required range.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had severe graft-versus-host disease after a transplant.

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I am allergic to nivolumab, ipilimumab, or their ingredients.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Optimal dose of nivolumab in combination with ipilimumab

Secondary study objectives

Disease-free survival (DFS)

Duration of response (DOR)

Overall response rate (ORR)

+1 more

Other study objectives

Immune cell phenotype

Immune checkpoint molecule expression

Immune phenotype in patients who experience aGVHD

+4 more

Side effects data

From 2024 Phase 3 trial • 529 Patients • NCT02017717

80%

Fatigue

70%

Diarrhoea

70%

Headache

40%

Vomiting

40%

Aspartate aminotransferase increased

40%

Rash maculo-papular

40%

Alanine aminotransferase increased

40%

Lipase increased

30%

Partial seizures

30%

Hemiparesis

30%

Gait disturbance

30%

Fall

30%

Cough

30%

Dry skin

30%

Amylase increased

30%

Nausea

30%

Confusional state

20%

Malignant neoplasm progression

20%

Pyrexia

20%

Candida infection

20%

Mucosal infection

20%

Decreased appetite

20%

Back pain

20%

Dysphonia

20%

Hypotension

20%

Colitis

20%

Hyperthyroidism

20%

Oedema peripheral

20%

Muscular weakness

20%

Hypothyroidism

10%

Tinnitus

10%

Cushingoid

10%

Diabetic ketoacidosis

10%

Procedural haemorrhage

10%

Blood bilirubin increased

10%

Bradycardia

10%

Sinus tachycardia

10%

Hyperglycaemia

10%

Hypocalcaemia

10%

Neck pain

10%

Brain oedema

10%

Hydrocephalus

10%

Lethargy

10%

Seizure

10%

Hypertension

10%

Palpitations

10%

Cheilitis

10%

Presyncope

10%

Face oedema

10%

Oedema

10%

Conjunctivitis

10%

Enterocolitis infectious

10%

Oral candidiasis

10%

Pneumonia

10%

Sinusitis

10%

Staphylococcal infection

10%

Blood alkaline phosphatase increased

10%

Spinal pain

10%

Tremor

10%

Dizziness

10%

Dysarthria

10%

Urinary retention

10%

Dyspnoea exertional

10%

Nasal congestion

10%

Pneumonitis

10%

Dermatitis

10%

Erythema

10%

Rash

10%

Klebsiella infection

10%

Hypomagnesaemia

10%

Syncope

10%

Haemorrhage intracranial

10%

Pancreatitis

10%

Cholecystitis

10%

Upper respiratory tract infection

10%

Acute kidney injury

10%

Dermatitis bullous

10%

Lymphopenia

10%

Optic nerve disorder

10%

Visual impairment

10%

Dehydration

10%

Hypokalaemia

10%

Scoliosis

10%

Cognitive disorder

10%

Memory impairment

10%

Hallucination

10%

Insomnia

10%

Irritability

10%

Urinary incontinence

10%

Dyspnoea

10%

Dermatitis acneiform

10%

Pelvic venous thrombosis

10%

Sepsis

100%

80%

60%

40%

20%

Study treatment Arm

Cohort 1: Arm N1+I3

Cohort 2: Arm B

Part A Cohort 1c: Arm N3+RT+TMZ

Part A Cohort 1d: Arm N3+RT

Part B Cohort 1c: Arm N3+RT+TMZ

Part B Cohort 1d: Arm N3+RT

Cohort 1: Arm N3

Cohort 1b: Arm N3+I1

Cohort 2: Arm N3

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.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Arm C (nivolumab and ipilimumab)Experimental Treatment2 Interventions

Beginning at least 6 weeks post-stem cell transplant, patients receive nivolumab IV over 60 minutes on days 1, 14, and 28, and ipilimumab IV over 90 minutes on day 1. Treatment repeats every 6 weeks for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Group II: Arm B (ipilimumab)Experimental Treatment1 Intervention

Beginning at least 6 weeks post-stem cell transplant, patients receive ipilimumab IV over 90 minutes on day 1. Treatment repeats every 21 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Group III: Arm A (nivolumab)Experimental Treatment1 Intervention

Beginning at least 6 weeks post-stem cell transplant, patients receive nivolumab IV over 60 minutes on days 1 and 15. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ipilimumab

FDA approved

Nivolumab

FDA approved

0 / 10Eligibility criteria met