New pill shows promise for Hard-to-Treat blood cancer
NCT ID NCT05141682
First seen Jun 25, 2026 · Last updated Jul 29, 2026 · Updated 5 times
Summary
This early-phase trial is testing an oral chemotherapy drug called azacitidine (CC-486) in 11 adults with a rare type of leukemia (T-LGLL) that has come back or not responded to prior treatment. The study aims to find the best dose and see how well the drug works at shrinking or controlling the cancer. Researchers will also monitor side effects and how long any benefits last.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- oral azacitidine (CC-486)
- What this could lead to
- If successful, this could offer a new oral treatment option for patients with T-LGLL leukemia that has not responded to standard therapies.
- What could go wrong
- This is a very small early-phase trial (11 participants) testing safety and dosing, so it may not lead to a proven treatment. Side effects of azacitidine are possible, and the cancer may still progress.
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 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
11 people
The number who actually took part.
- Started
-
Jun 2022
- Expected to finish
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Sep 2026
An estimate. End dates often move.
- Lead sponsor
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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
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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 or older * Diagnosis of T-LGLL defined as: CD3+CD8+ cell population \> 650/mm\^3 and the presence of a clonal T-cell receptor (within 1 month of diagnosis). This also includes patients with rare T-LGLL variants include CD4+ T-LGLL, and gamma/delta T-LGLL which can be CD4- and CD8, though patients still must have an LGL cell population \>500 cells/mm3 and the presence of a clonal T-cell receptor within 1 month of diagnosis or relapse. Note: patients with myelodysplastic syndrome (MDS)-like T-LGLL may be included with principal investigator (PI) approval even if CD3+CD8+ cell population is \< 650/mm\^3, though +TCR is required. Natural-killer (NK) large granular lymphocytic leukemia (LGL) is also permitted, provided there is a clonal NK-cell population noted with \> 500 cells/mm\^3 * Failed at least one line of frontline therapy; off treatment for at least 14 days or 5 half-lives, whichever is longer * Require Treatment for T-LGLL (One or more required) * Symptomatic anemia with hemoglobin \< 10 g/dL * Transfusion-dependent anemia * Neutropenia with absolute neutrophil count (ANC) \< 500/mm\^3 * Neutropenia with ANC \< 1500/mm\^3 with recurrent infections * Platelet count \>= 50 x 10\^9/L * Serum creatinine =\< 2 x the upper limit of normal (ULN) * Total bilirubin =\< 1.5 x ULN (patients with Gilbert's syndrome with a bilirubin \> 1.5 x ULN permitted) * Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\< 1.5 x ULN * Eastern cooperative oncology group (ECOG) performance status =\< 2 * Men and women of reproductive potential must agree to follow accepted birth control methods for the duration of the study. Female subject is either post-menopausal or surgically sterilized or willing to use an acceptable method of birth control (i.e., a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide, or abstinence) for the duration of the study. Male subject agrees to use an acceptable method for contraception for the duration of the study * Able to sign informed consent Exclusion Criteria: * Active Infection requiring ongoing anti-microbial treatment. Patients with human immunodeficiency virus (HIV), positive hepatitis B surface antigen or hepatitis C antibody will be excluded * Concurrent immune-suppressive therapy (prednisone or equivalent up to 20 mg permitted to treat T-LGL symptoms, but must be weaned within one month of initiation of trial drug). Patients on stable, chronic prednisone =\< 10 mg for rheumatologic/autoimmune conditions are exempted from this requirement. They may enroll on the study * Active, concurrent malignancy unless deemed related to T-LGLL by PI * Prior use of 5-azacytidine or decitabine * Positive pregnancy test
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210, United States
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University of Virginia
Charlottesville, Virginia, 22908, United States
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