Phase 1 Leukaemia Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Children and young people aged 0 to 21 years who weigh at least 5 kg, with at least 80% of participants being under 18 years old.
• People diagnosed with a specific type of acute leukemia that has one of three particular genetic changes (called KMT2A-r, NPM1-m, or NUP98-r), confirmed by a central laboratory.
• People whose leukemia has come back (relapsed) or did not respond to standard treatment, including bone marrow transplant, with the relapse confirmed through bone marrow testing or, in some cases, a blood test showing a high number of leukemia cells.
• People whose leukemia returned in the bone marrow (alone or combined with disease outside the bone marrow, including in the nervous system), but not those with disease only outside the bone marrow.
• People who are well enough to carry out basic daily activities, as measured by standard health scoring tools appropriate for their age.
• People whose kidneys are working well enough (kidney filtration rate of at least 60 mL/min).
• People whose liver is working well enough, with certain blood test results within acceptable ranges (unless any abnormality is caused by leukemia itself).
• People whose heart is working well enough, with the heart pumping at an acceptable level and no signs of heart failure in the four weeks before screening.
• People who have recovered from the side effects of previous cancer treatments, and who have waited the required amount of time since their last treatment before joining (the waiting period varies depending on the type of treatment received).
• People who have had a bone marrow or stem cell transplant may be eligible if at least 84 days have passed since the transplant, and there is no active graft-versus-host disease (a complication where donated cells attack the body), with limited exceptions for mild skin-related cases managed with low-dose steroids.
• People who have previously been treated with a different menin inhibitor (a type of targeted drug) may be eligible, unless they had a severe reaction specifically linked to that drug's effects (confirm with trial site).
• People (or their parent/guardian) who are able to provide written consent before any study procedures begin, in line with local laws.
• Female participants who could become pregnant must have a negative pregnancy test before joining.
• Female participants who are breastfeeding must agree to stop breastfeeding for the duration of the study.
• People who are able to become pregnant must agree to use highly effective contraception during treatment and for six months after treatment ends.
• Male participants must agree to use a condom during intercourse and not to father a child or donate sperm during treatment and for four months after treatment ends.
• Participants in the United States or Canada must have already enrolled in a related study called APAL2020SC before joining this trial.

Who may not be able to join:

• People who, in the investigator's opinion, may not be able to follow the study requirements.
• People with Down syndrome.
• People whose leukemia has only returned outside the bone marrow (isolated extramedullary relapse), or only in the central nervous system (isolated CNS relapse), or who have symptomatic CNS3 disease.
• People diagnosed with a specific leukemia type called acute promyelocytic leukemia (APL) or juvenile myelomonocytic leukemia (JMML).
• People who have a condition affecting how their body absorbs food or medication through the digestive system, which could prevent the study drug from being taken by mouth.
• People who are currently taking proton pump inhibitors (a type of medication that reduces stomach acid, such as omeprazole or pantoprazole), as these can affect how the study drug is absorbed.
• People who are currently taking part in another clinical trial using an experimental drug.
• People with any known inherited bone marrow failure condition.
• People who have a known allergy to any of the medications used in this trial.
• People with an active infection that is not under control at the time of joining.
• People with active or uncontrolled HIV, hepatitis B, or hepatitis C infection.
• Female participants who are pregnant or breastfeeding.
• People with a pre-existing condition that puts them at high risk of serious infection, uncontrolled bleeding, or low blood cell counts unrelated to leukemia or its treatment (for example, cystic fibrosis or an immune deficiency condition).
• People who are taking other experimental medications, or have taken them within 30 days before the first dose of the study drug.
• People with significant inborn heart conditions, such as long QT syndrome or certain uncorrected heart defects that pose a serious risk (note: some minor heart defects may not exclude participation — confirm with trial site).
• People with an underlying medical condition that, in the investigator's opinion, would make the study treatment unsafe or make it hard to assess side effects.
• People with a known allergy to fludarabine, cytarabine, or any of their ingredients (these are chemotherapy drugs used in this trial).
• People who have received a live vaccine within the past six months.

Important: Always verify eligibility with the trial site directly before applying.