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Anti-metabolites

Azacitidine + Enasidenib for Myelodysplastic Syndrome

Phase 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

Subjects must have an IDH2 gene mutation (IDH2-R140 or R172) as determined by local laboratory result

Subjects with a histologically confirmed diagnosis of MDS, including both MDS and refractory anemia with excess blasts in transformation (RAEB-T) (acute myeloid leukemia [AML] with 20-30% blasts and multilineage dysplasia by French-American-British [FAB] criteria) by World Health Organization (WHO), and chronic myelomonocytic leukemia (CMML) are eligible

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years

Awards & highlights

Study Summary

This trial looks at the side effects and effectiveness of azacitidine and enasidenib for treating patients with myelodysplastic syndrome caused by an IDH2 mutation.

Who is the study for?

This trial is for patients with IDH2-mutant myelodysplastic syndrome who have specific gene mutations, normal liver and kidney function, can consent to the study's requirements, and are not pregnant or nursing. Men must use contraception if with a partner of childbearing potential. It includes those new to treatment (Arm A) or who didn't respond well to previous therapies (Arm B).Check my eligibility

What is being tested?

The trial is testing how effective azacitidine combined with enasidenib is in treating myelodysplastic syndrome linked to IDH2 mutations. The goal is to see if these drugs can halt cancer cell growth by inhibiting certain enzymes.See study design

What are the potential side effects?

Potential side effects may include issues related to organ inflammation, digestive system disturbances due to enzyme inhibition, fatigue from anemia management, and possible reactions at the infusion site where medication enters the body.

Eligibility Criteria

Inclusion Criteria

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

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My cancer has an IDH2 gene mutation.

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I have been diagnosed with MDS, RAEB-T, or CMML.

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All my side effects from previous cancer treatments, except hair loss, are mild or gone.

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I did not respond to or relapsed after 6 cycles of treatment with hypomethylating agents.

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I can take care of myself and am up and about more than half of the day.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of adverse events

Overall response rate

Secondary outcome measures

Anti-tumor activity

Drug exposure levels

Event-free survival (EFS)

+2 more

Other outcome measures

Biomarkers analysis

Side effects data

From 2016 Phase 1 & 2 trial • 21 Patients • NCT02273739

71%

Nausea

57%

Fatigue

57%

Diarrhoea

43%

Urinary tract infection

43%

Insomnia

43%

Anaemia

43%

Somnolence

29%

Blood bilirubin increased

29%

Pyrexia

29%

Cough

29%

Leukocytosis

29%

Dry eye

29%

Constipation

29%

Vomiting

29%

Decreased appetite

29%

Hyperbilirubinaemia

14%

Alanine aminotransferase increased

14%

Bacteraemia

14%

Wound complication

14%

Lung infection

14%

Dehydration

14%

Herpes simplex

14%

Hypomagnesaemia

14%

Syncope

14%

Arthralgia

14%

Hyperglycaemia

14%

Oral infection

14%

Contusion

14%

Lymphocyte count decreased

14%

Hypercalcaemia

14%

Hypoxia

14%

Back pain

14%

Thrombocytopenia

14%

Palpitations

14%

Aspiration

14%

Pneumonia aspiration

14%

Apnoea

14%

Respiratory failure

14%

Gait disturbance

14%

Hyponatraemia

14%

Flank pain

14%

Tumour pain

14%

Metabolic encephalopathy

14%

Confusional state

14%

Abdominal discomfort

14%

Pleural effusion

14%

Chills

14%

Productive cough

14%

Nasal congestion

14%

Dysphagia

14%

Angina pectoris

14%

Sinus tachycardia

14%

Abdominal distension

14%

Abdominal pain upper

14%

Flatulence

14%

Salivary hypersecretion

14%

Amylase increased

14%

Aspartate aminotransferase increased

14%

Blood bilirubin unconjugated increased

14%

Blood creatinine increased

14%

International normalised ratio increased

14%

Hypoalbuminaemia

14%

Muscle spasms

14%

Muscle tightness

14%

Muscular weakness

14%

Musculoskeletal pain

14%

Headache

14%

Tremor

14%

Anxiety

14%

Dyspnoea

14%

Oropharyngeal pain

14%

Wheezing

14%

Butterfly rash

14%

Hypertension

14%

Hypotension

14%

Venous thrombosis limb

14%

Conjunctivitis

14%

Oral candidiasis

14%

Pharyngitis

14%

Upper respiratory tract infection

14%

Wound

14%

Hypocalcaemia

14%

Hypokalaemia

14%

Dizziness

14%

Facial paresis

14%

Hemiparesis

14%

Scrotal erythema

14%

Non-cardiac chest pain

100%

80%

60%

40%

20%

Study treatment Arm

Enasidenib 400 mg

Enasidenib 100 mg

Enasidenib 200 mg

Enasidenib 650 mg

Trial Design

2Treatment groups

Experimental Treatment

Group I: Arm II (enasidenib)Experimental Treatment2 Interventions

Patients relapsed and/or refractory to HMA therapy receive enasidenib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Group II: Arm I (enasidenib, azacitidine)Experimental Treatment3 Interventions

Patients who are HMA-naive receive enasidenib PO QD on days 1-28 and 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

2012

Completed Phase 3

~1440

Enasidenib

2020

Completed Phase 2

~560

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor

2,972 Previous Clinical Trials

1,787,258 Total Patients Enrolled

National Cancer Institute (NCI)NIH

13,661 Previous Clinical Trials

40,924,381 Total Patients Enrolled

CelgeneIndustry Sponsor

636 Previous Clinical Trials

128,908 Total Patients Enrolled

Media Library

Azacitidine (Anti-metabolites) Clinical Trial Eligibility Overview. Trial Name: NCT03383575 — Phase 2

Myelodysplastic Syndrome Research Study Groups: Arm I (enasidenib, azacitidine), Arm II (enasidenib)

Myelodysplastic Syndrome Clinical Trial 2023: Azacitidine Highlights & Side Effects. Trial Name: NCT03383575 — Phase 2

Azacitidine (Anti-metabolites) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03383575 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How is Enasidenib usually employed to benefit patients?

"Enasidenib is regularly utilized as a form of induction chemotherapy, but it has also been successful in treating refractory anemias, leukemia, myelocytic conditions and various multilineage dysplasia."

Answered by AI

How many participants have been recruited for this investigation?

"At the time of this inquiry, there are no open opportunities for enrolment into this medical study. The research was posted on January 17th 2018 and last edited on September 22nd 2022. However, there are 2788 studies recruiting patients with anemic refractory excess blasts, as well as 193 trials dedicated to Enasidenib that have active recruitment periods."

Answered by AI

Is enrollment in this investigation still ongoing?

"According to the material posted on clinicaltrials.gov, this medical trial is no longer recruiting participants. It was originally made available on January 17th 2018 and it's latest update came out in September 22nd 2022. However, there are still 2981 active trials which are presently looking for volunteers."

Answered by AI

Are there any other trials that have assessed the efficacy of Enasidenib?

"Currently, 193 studies are ongoing that examine the efficacy of Enasidenib with 33 in Phase 3. Edmonton, Alberta is home to a majority of these clinical trials; however there 6023 sites worldwide running such experiments."

Answered by AI

What potential hazards might patients face when using Enasidenib?

"Despite being in Phase 2 and lacking clinical data for efficacy, the safety of Enasidenib has been given a rating of 2 by Power."

Answered by AI

What objective is this medical experiment attempting to accomplish?

"According to Celgene, the primary metric of efficacy for this trial over a three year period will be incidence of adverse events. Secondary objectives include overall survival (measured with Kaplan-Meier method and log rank tests), event-free survival (Kaplan-Meier method/log rank tests) and anti-tumor activity (graphical summaries/descriptive statistics)."

Answered by AI

Recent research and studies

idh2Journal

Targeted Therapy with the Mutant IDH2 Inhibitor Enasidenib for High-risk <i>idh2</i>-mutant Myelodysplastic Syndrome

DiNardo, Courtney D., Sangeetha Venugopal, Curtis A. Lachowiez, Koichi Takahashi, Sanam Loghavi, Guillermo Montalban-Bravo, Xuemei Wang, et al.. 2022. “Targeted Therapy with the Mutant IDH2 Inhibitor Enasidenib for High-risk idh2-mutant Myelodysplastic Syndrome”. Blood Advances. American Society of Hematology. doi:10.1182/bloodadvances.2022008378.

clinicaltrials.govStudy

Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome

2018. "Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03383575.

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