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Experimental drug targets rare blood condition in tiny pilot study

NCT ID NCT05102370

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tested a drug called enasidenib in 4 people with a rare blood condition (CCUS) and a specific gene change (IDH2). The goal was to see if the drug could safely improve low blood cell counts. The study is complete, but results are not yet reported.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

4 people

The number who actually took part.

Started

Oct 2021

Finished

Jan 2026

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: * Age ≥18 years at the time of signing the informed consent form. * Willing and able to adhere to the study visit schedule and other protocol requirements. * 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 * Results of bone marrow biopsy within 1 month of study entry (screening bone marrow biopsy) must not indicate hematologic disease. * IDH2 gene mutation (R140 or R172), performed locally, at a frequency \>2%. * ECOG performance status of 0-2. * Adequate organ function, defined as: * Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤3x upper limit of normal (ULN), unless the subject has Gilbert's syndrome. * Serum total bilirubin \<1.5x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis. In these cases, approval from the study Principal Investigator is required. * Creatinine clearance greater than 50 mL/min based on the Cockroft-Gault glomerular filtration rate estimation. * Patients being enrolled on study on the basis of anemia, will only be eligible if folate, B12, serum iron, serum ferritin, total iron binding capacity, haptoglobin and peripheral smear within normal limits * Women of childbearing potential may participate provided they have a negative serum pregnancy test at screening and a negative serum or urine pregnancy test within 72 h of starting treatment. * Women of childbearing potential (WOCBP) and males with partners who are WOCBP must agree to abstain from sexual intercourse or to use 1 highly effective form of contraception during the study and for at least 4 months following the last dose of enasidenib. Males with partners who are WOCBP must agree to use a barrier method. Exclusion Criteria: * Active malignancy defined as \>1-cm disease on most recent CT scan in the past 6 months or recent history of cancer (i.e. within the past 5 years) with \>50% chance of cancer recurrence in the next 5 years. * Current or prior history of hematologic malignancy. * Therapy (including maintenance therapy) for solid-tumor malignancy within the last 6 months. * Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally. * Active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment). * Positive direct Coombs test * Evidence of hypersplenism on physical exam * Pregnant or lactating (women).

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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 (Data Collection Only)

    Cleveland, Ohio, 44195, United States

  • Mayo Clinic (Data Collection Only)

    Rochester, Minnesota, 55905, United States

  • Memorial Sloan Kettering Bergen (Limited protocol activities)

    Montvale, New Jersey, 07645, United States

  • Memorial Sloan Kettering Cancer Center (All Protocol Activities)

    New York, New York, 10065, United States

  • Memorial Sloan Kettering Monmouth (All protocol activities)

    Middletown, New Jersey, 07748, United States

  • Memorial Sloan Kettering Nassau (Limited protocol activities)

    Rockville Centre, New York, 11553, United States

  • Memorial Sloan Kettering Suffolk-Commack (Limited protocol activities)

    Commack, New York, 11725, United States

  • Memorial Sloan Kettering Westchester (All Protocol Activities)

    Harrison, New York, 10604, United States

  • Memorial Sloan Kettering at Basking Ridge (All Protocol Activities)

    Basking Ridge, New Jersey, 07920, United States

  • Ohio State University (Data Collection Only)

    Columbus, Ohio, 43210, United States

  • Oregon Health & Science University (Data Collection Only)

    Portland, Oregon, 97239, United States

  • Washington University (Data Collection and Specimen Analysis)

    St Louis, Missouri, 63110, United States

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