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IDH1 Inhibitor

Ivosidenib for Blood Disorders

Phase 2
Recruiting
Led By Kelly Bolton, M.D.
Research Sponsored by Washington University School of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
IDH1 gene mutation (R132) confirmed by droplet digital PCR (ddPCR) testing, at a frequency > 2%. This will be performed locally and confirmed at Washington University.
Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of ≥1 blood count indexes below the following thresholds: Hgb <10 g/dL, ANC <1.8 × 10^9/L, Platelets <100 × 10^9/L
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through completion of treatment (estimated to be 17 months)
Awards & highlights

Study Summary

This trial is testing a new drug to see if it's effective and safe for people with clonal cytopenia of undetermined significance, a blood disorder.

Who is the study for?
Adults with clonal cytopenia of undetermined significance (CCUS) and specific IDH1 gene mutations who have had unexplained low blood counts for at least six months. Participants must be in stable health, not pregnant or breastfeeding, without active cancer or heart issues, and able to consent.Check my eligibility
What is being tested?
The trial is testing Ivosidenib's safety and effectiveness in improving blood count abnormalities in patients with CCUS carrying IDH1 mutations. It's an open-label study where all participants receive the drug, conducted remotely across multiple centers.See study design
What are the potential side effects?
Potential side effects of Ivosidenib may include but are not limited to digestive disturbances, liver enzyme elevations, fatigue, joint pain or swelling, skin problems like rash or itchiness, and changes in heart rhythm.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My cancer has a specific IDH1 gene mutation.
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I have had low blood counts for over 6 months without a known cause.
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My cancer has a specific IDH1 gene mutation.
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I am 18 years old or older.
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I can take care of myself and perform daily activities.
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My liver and kidney functions are within the required limits.
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I am 18 years old or older.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through completion of treatment (estimated to be 17 months)
This trial's timeline: 3 weeks for screening, Varies for treatment, and through completion of treatment (estimated to be 17 months) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Rate of improvement in hematologic parameters
Secondary outcome measures
Change in mutant IDH1 variant allele fraction
Disease free survival
Number of adverse events as measured by CTCAE v 5.0

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%
Hypokalaemia
5%
Rash
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

Trial Design

1Treatment groups
Experimental Treatment
Group I: IvosidenibExperimental Treatment1 Intervention
-Ivosidenib is an oral drug which will be administered on an outpatient basis at a dose of 500 mg daily for up to 17 months (approximately 18 28-day cycles), with each cycle being 28 days.
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?

Servier Hellas Pharmaceuticals Ltd.Industry Sponsor
1 Previous Clinical Trials
2,285 Total Patients Enrolled
Washington University School of MedicineLead Sponsor
1,935 Previous Clinical Trials
2,299,858 Total Patients Enrolled
Gateway for Cancer ResearchOTHER
45 Previous Clinical Trials
2,555 Total Patients Enrolled

Media Library

Ivosidenib (IDH1 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05030441 — Phase 2
Clonal Cytopenia Research Study Groups: Ivosidenib
Clonal Cytopenia Clinical Trial 2023: Ivosidenib Highlights & Side Effects. Trial Name: NCT05030441 — Phase 2
Ivosidenib (IDH1 Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05030441 — Phase 2

Frequently Asked Questions

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

Are enrollment opportunities still available for this trial?

"Affirmative. Data hosted on clinicaltrials.gov reveals that this medical experiment was originally posted in April of 2022 and is actively seeking volunteers for participation. 15 subjects will be recruited from 6 distinct healthcare facilities."

Answered by AI

Are there any documented accounts of Ivosidenib being used in research projects?

"As of now, Ivosidenib is undergoing 21 separate medical trials. Two are at the Phase 3 level and much of the research is happening in Frankfurt, Connecticut with a total 932 trial sites across the globe."

Answered by AI

To what extent can this study accommodate participants?

"To complete the research project, 15 volunteers meeting the criteria need to be recruited. The Servier Hellas Pharmaceuticals Ltd is responsible for managing this trial and has chosen two centres in Missouri and New york as their base of operations: Washington University School of Medicine in Saint Louis and Memorial Sloan Kettering Cancer Center in Manhattan."

Answered by AI

Are there a plethora of facilities implementing this trial within North America?

"This clinical trial is being conducted out of Washington University School of Medicine in Saint Louis, Missouri, Memorial Sloan Kettering in New york, New York and Mayo Clinic in Rochester Minnesota. Additionally, there are 4 additional sites participating."

Answered by AI

Has Ivosidenib been officially recognized by the FDA?

"Our team believes that ivosidenib warrants a safety score of 2 due to the lack of evidence regarding its efficacy in clinical trials. However, there is some data available supporting ivosidenib's safety."

Answered by AI

Who else is applying?

What state do they live in?
Illinois
How old are they?
65+
What portion of applicants met pre-screening criteria?
Did not meet criteria
Recent research and studies
clinicaltrials.govStudy
Ivosidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH1
2022. "Ivosidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH1". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05030441.
All > Thrombocytopenia
~6 spots leftby Aug 2025
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