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Can a targeted drug revive blood cell production in a Pre-Leukemic condition?

NCT ID NCT05030441

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time

Summary

This trial tests whether ivosidenib, a drug that targets IDH1 mutations, can improve blood counts in people with clonal cytopenia of undetermined significance (CCUS) who have these mutations. CCUS is a condition with low blood counts and an increased risk of developing blood cancers. The study will measure improvements in red blood cells, platelets, and neutrophils after treatment. It also explores whether the drug reduces the proportion of cells carrying the IDH1 mutation.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ivosidenib (Tibsovo), an oral drug that targets IDH1 mutations
What this could lead to
If successful, this could offer a targeted treatment to improve blood counts in people with CCUS and IDH1 mutations, potentially slowing or preventing progression to more serious blood disorders.
What could go wrong
This is a small pilot study, so results may not be conclusive. The drug may not improve blood counts for everyone, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

20 people

The number who actually took part.

Started

Apr 2022

Expected to finish

Jan 2030

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 * 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 * Adequate organ function as defined below: * 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. * Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable). Exclusion Criteria: * Indication of hematologic disease by bone marrow biopsy within 6 months of study entry. \*Evidence of disease progression from time of bone marrow biopsy to enrollment based on investigator review of symptoms and complete blood counts * Active malignancy (defined as \> 1 cm disease on most recent CT scan in the past 6 months). * Currently receiving therapy for solid tumor malignancy. * Currently receiving any other investigational agents. * Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally. * A history of allergic reactions attributed to compounds of similar chemical or biologic composition to ivosidenib or other agents used in the study. * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia. * Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 72 hours of study entry. * Heartrate corrected QT interval (QTc) \> 450 msec or with other factors that increase the risk of QT prolongation or arrhythmic events (e.g. heart failure, hypokalemia, family history of long QT interval syndrome). * Known medical history of progressive multifocal leukoencephalopathy (PML). * Currently taking medications known to be CYP3A4 strong inducers and sensitive substrates.

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As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Cleveland Clinic - Case Comprehensive Cancer Center

    Cleveland, Ohio, 44195, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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