Ivosidenib for Clonal Cytopenia of Undetermined Significance

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Cleveland Clinic - Case Comprehensive Cancer Center, Cleveland, OH
Clonal Cytopenia of Undetermined Significance+3 More
Ivosidenib - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether ivosidenib is safe and effective in treating patients with CCUS.

See full description

Eligible Conditions

  • Clonal Cytopenia of Undetermined Significance

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Clonal Cytopenia of Undetermined Significance

Study Objectives

This trial is evaluating whether Ivosidenib will improve 1 primary outcome and 3 secondary outcomes in patients with Clonal Cytopenia of Undetermined Significance. Measurement will happen over the course of Through 30 days after completion of treatment (estimated to be 18 months).

Month 18
Disease free survival
Number of adverse events as measured by CTCAE v 5.0
Rate of improvement in hematologic parameters
Month 17
Change in mutant IDH1 variant allele fraction

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Clonal Cytopenia of Undetermined Significance

Trial Design

1 Treatment Group

Ivosidenib
1 of 1
Experimental Treatment

This trial requires 15 total participants across 1 different treatment group

This trial involves a single treatment. Ivosidenib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Ivosidenib
Drug
-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.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: through completion of treatment (estimated to be 17 months)
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly through completion of treatment (estimated to be 17 months) for reporting.

Closest Location

Cleveland Clinic - Case Comprehensive Cancer Center - Cleveland, OH

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Hgb <10 g/dL
ANC <1.8 × 10^9/L
Platelets <100 × 10^9/L
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.
At least 18 years of age.
ECOG performance status 0-2
AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
Serum total bilirubin < 1.5 x IULN (an upper limit of bilirubin 5mg/dL is acceptable if it can be attributed to Gilbert's syndrome or erythropoiesis)
Serum creatinine < 2 x IULN or creatinine clearance > 50 mL/min by Cockcroft-Gault glomerular filtration rate estimation
The effects of ivosidenib on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (defined in Section 5.5) prior to study entry, for the duration of study participation, and for 90 days after the last dose of ivosidenib. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of the study, and for 90 days after the last dose of ivosidenib.

Patient Q&A Section

Can leukopenia be cured?

"Leukopenia can be managed by close monitoring. A leukocyte nadir of less than 5000 cells/microL and a complete granulocyte recovery within 1 year are indicative of a positive outcome." - Anonymous Online Contributor

Unverified Answer

What are the signs of leukopenia?

"The presence of a nonregenerative neutrophil count must be considered in the differential diagnosis of patients with signs and symptoms of viral infection. Patients who have leukopenia may develop a life-threatening complication of HIV infection and need an urgent referral from a virologist to the hospital with a specialized service for HIV-positive patients." - Anonymous Online Contributor

Unverified Answer

How many people get leukopenia a year in the United States?

"This survey establishes that more than one in five white men and women will develop a low white blood cell count in their lifetimes. The prevalence of low WBC counts and incidence of low WBC counts appears to be higher in men than women. Given these findings, the American Society of Hematology recommends the routine measurement of WBC counts on all patients who undergo peripheral blood collection." - Anonymous Online Contributor

Unverified Answer

What are common treatments for leukopenia?

"Leucopenia is a common and relatively poorly understood toxicity of chemotherapy. A single treatment for acute leukopenia has been shown to improve symptoms of neutropenia and neutrophil count and reduce the need for hospitalization and medical intervention. If a single cycle is not adequate, an attempt may be made to repeat the treatment. This is not always feasible. When such repeat treatment fails, a more intensive approach, using all possible resources, is often used. There are many possible options for treating leukopenia, but treatment is usually based on treatment priorities." - Anonymous Online Contributor

Unverified Answer

What causes leukopenia?

"If the cause of leukopenia in a patient is clear and the etiology is treated, then the prognosis is good. The cause of leukopenia in most adult patients is unclear and should be investigated." - Anonymous Online Contributor

Unverified Answer

What is leukopenia?

"It is crucial for patients to monitor their nutritional status after chemotherapy and also ensure a good compliance with recommendations. Leukopenia can be easily identified by a complete blood count using a flow cytometer." - Anonymous Online Contributor

Unverified Answer

Does leukopenia run in families?

"These data show that leukopenia run in the family, supporting the conclusion that at least a part of leukopenia in leukaemic patients is genetically determined." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in ivosidenib for therapeutic use?

"Ivosidenib is a selective, competitive, multi-targeted tyrosine kinase inhibitor being investigated for investigational use in MLL-rearranged acute myeloid leukemia in adults and relapsed/refractory MLL-translocated leukemias. It was approved by the FDA for this indication in April 2019. In September 2019, an open label, dose-escalation phase 2 clinical trial of ivosidenib in relapsed/refractory MLL-rearranged acute myeloid leukemia was successfully completed." - Anonymous Online Contributor

Unverified Answer

What is ivosidenib?

"Ivosidenib, marketed by Sanofi-Genzyme as an ALK inhibitor for people with ALK-positive lung cancer and metastatic colorectal cancer, was developed by researchers at Hoffmann-La Roche and Sanofi in collaboration with Daiichi Sankyo, following phase I and II clinical studies. Sanofi is responsible for commercial marketing. Based on positive preliminary results submitted to the FDA in December 2017, Phase III trials in people with ALK-positive solid tumors began in early 2018. For more information on ivosidenib, visit the Sanofi website." - Anonymous Online Contributor

Unverified Answer

Is ivosidenib typically used in combination with any other treatments?

"The median time to treatment initiation was 3.0 months in patients receiving ivosidenib plus chemotherapy and 2.9 months in patients receiving ivosidenib alone. The addition of ivosidenib to chemotherapy decreased the frequency of myelosuppression but did not improve outcomes." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving ivosidenib?

"Ivosidenib is the first clinical trial targeting myelodysplastic and myeloproliferative syndromes with the potential for significant impact on patients with myelodysplastic or myeloproliferative syndromes. Other clinical trials of ivosidenib currently in phase I/II are in various settings and for various indications." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of leukopenia?

"While leukemia may cause leukopenia, other medical conditions such as infection and blood disorders can also increase white blood cell levels. Treatment or diseases affecting the bones such as osteopetrosis or Paget's disease can cause leukopenia and may present similar clinical considerations as those that have been attributed to acute leukemia. It is important to evaluate all possible medical or radiation causes of leukopenia and rule them out. If these causes, leukopenia and related symptoms still persist after a medical work-up is completed, leukopenia must be considered a chemotherapy related immune-mediated adverse effect." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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