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IDH1 Inhibitor

Ivosidenib for Advanced Solid Cancers, Lymphoma & Histiocytosis

Phase 2
Waitlist Available
Led By Elizabeth D Alva
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
For patients with solid tumors without known bone marrow involvement: Peripheral absolute neutrophil count (ANC) >= 1000/mm^3 (within 7 days prior to enrollment)
Patients must be >= than 12 months and =< 21 years of age at the time of study enrollment
Must not have
Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant
Patients who have an uncontrolled infection
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial will test ivosidenib to see if it can help patients with advanced solid tumors, lymphoma, or histiocytic disorders that have IDH1 mutations.

Who is the study for?
This trial is for young patients aged between 12 months and 21 years with advanced solid tumors, lymphoma, or histiocytic disorders that have a specific genetic change (IDH1 mutation). They must be able to swallow tablets, have not used IDH1 inhibitors before, and meet certain health criteria like adequate blood counts and organ function. Pregnant individuals or those on certain medications are excluded.
What is being tested?
The trial is testing Ivosidenib's effectiveness in treating cancers with IDH1 mutations. It's a phase II study focusing on whether this drug can stop cancer cells from growing by targeting the IDH pathway—a critical cell signaling route involved in tumor development.
What are the potential side effects?
While the exact side effects of Ivosidenib aren't listed here, similar drugs often cause issues like diarrhea, fatigue, joint pain or swelling, skin problems, nausea. There may also be more serious risks such as liver problems or an abnormal heart rhythm.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My white blood cell count is healthy for cancer treatment.
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I am between 12 and 21 years old.
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I can swallow pills without any issues.
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My body surface area is at least 0.78 square meters.
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I have received a stem cell infusion, with or without total body irradiation.
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I have received external beam radiation therapy.
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I have not undergone radiopharmaceutical therapy.
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I was assigned a specific treatment in the APEC1621SC study due to a certain mutation.
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I have never taken AG-120 (ivosidenib) or any IDH1 inhibitor.
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My kidney function, measured by creatinine clearance or GFR, is good.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am taking medication to prevent graft-versus-host disease after a bone marrow transplant.
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I do not have any infections that are currently uncontrolled.
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I am not taking drugs that strongly affect liver enzyme CYP3A4.
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I have had progressive multifocal leukoencephalopathy in the past.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 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
Objective response rate (ORR; complete response + partial response) in pediatric patients treated with ivosidenib
Secondary study objectives
Percentage of patients experiencing grade 3 or higher adverse events
Preliminary estimates of the pharmacokinetics (PK) of ivosidenib in children and adolescents with relapsed or refractory cancer
Progression free survival (PFS)
Other study objectives
Biomarker analysis
Profiling changes in tumor genomics

Side effects data

From 2021 Phase 3 trial • 187 Patients • NCT02989857
28%
Nausea
28%
Diarrhoea
23%
Fatigue
21%
Oedema peripheral
16%
Abdominal pain
16%
Anaemia
14%
Decreased appetite
14%
Weight decreased
12%
Vomiting
12%
Asthenia
12%
Cough
12%
Ascites
12%
Constipation
12%
Arthralgia
9%
Hypertension
9%
Abdominal pain upper
9%
Dizziness
9%
Muscle spasms
9%
Muscular weakness
9%
Dyspnoea
9%
Blood alkaline phosphatase increased
7%
Upper respiratory tract infection
7%
Hypoalbuminaemia
7%
Pruritus
7%
Hypophosphataemia
7%
Aspartate aminotransferase increased
7%
Alanine aminotransferase increased
7%
Insomnia
7%
Abdominal discomfort
5%
Rash
5%
Hypokalaemia
5%
Back pain
5%
White blood cell count decreased
5%
Hyperglycaemia
5%
Hyperkalaemia
5%
Pyrexia
5%
Headache
5%
Abdominal distension
5%
Blood bilirubin increased
5%
Confusional state
5%
Platelet count decreased
2%
Electrocardiogram QT prolonged
2%
Chills
2%
Cholangitis
2%
Gastrointestinal haemorrhage
2%
Intestinal pseudo-obstruction
2%
Biliary obstruction
2%
Bacteraemia
2%
Clostridium difficile colitis
2%
Escherichia bacteraemia
2%
Hip fracture
2%
Hypercalcaemia
2%
Encephalopathy
2%
Acute kidney injury
2%
Hypotension
2%
Gastrooesophageal reflux disease
2%
Hypomagnesaemia
2%
Blood creatinine increased
2%
Dyspepsia
2%
Urinary tract infection
2%
Rash maculo-papular
2%
Dry mouth
2%
Multiple sclerosis relapse
2%
Spinal cord compression
2%
Syncope
2%
Hyponatraemia
2%
Hepatic cirrhosis
2%
Oesophageal varices haemorrhage
2%
Upper gastrointestinal haemorrhage
100%
80%
60%
40%
20%
0%
Study treatment Arm
After Cross Over to AG-120
AG-120
Placebo

Awards & Highlights

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 (ivosidenib)Experimental Treatment1 Intervention
Patients receive ivosidenib PO QD. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ivosidenib
2019
Completed Phase 1
~20

Find a Location

Who is running the clinical trial?

Children's Oncology GroupNETWORK
460 Previous Clinical Trials
239,988 Total Patients Enrolled
30 Trials studying Wilms Tumor
19,585 Patients Enrolled for Wilms Tumor
National Cancer Institute (NCI)Lead Sponsor
13,906 Previous Clinical Trials
41,011,979 Total Patients Enrolled
54 Trials studying Wilms Tumor
21,271 Patients Enrolled for Wilms Tumor
Elizabeth D AlvaPrincipal InvestigatorChildren's Oncology Group

Media Library

Ivosidenib (IDH1 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04195555 — Phase 2
Wilms Tumor Research Study Groups: Treatment (ivosidenib)
Wilms Tumor Clinical Trial 2023: Ivosidenib Highlights & Side Effects. Trial Name: NCT04195555 — Phase 2
Ivosidenib (IDH1 Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04195555 — Phase 2
~3 spots leftby Mar 2025
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