Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial enrolled 16 people in total across four groups, each receiving a different dose level of the study treatment (3 people in Dose Level 1, 4 in Dose Level 2, 6 in Dose Level 3, and 3 in Dose Level 4). The trial was studying a treatment for a blood cancer condition, and its main goal was to find out how many participants experienced a "dose-limiting toxicity" (a serious side effect severe enough to set an upper limit on the dose) during the first round of treatment. It also looked at how participants' disease responded after one cycle of treatment. The reported data shows that, for the primary measure, out of the participants assessed at each dose level, no dose-limiting toxicities were recorded in Dose Levels 1, 2, or 4 (0 out of 3 participants each), while 1 out of 5 assessed participants experienced one in Dose Level 3. For the secondary measure looking at disease response after one cycle, the reported data shows that across the four dose groups: complete remission (disease appearing to clear with blood count recovery) was recorded in 0, 1, 1, and 0 participants respectively; complete remission without full blood count recovery was recorded in 1, 0, 0, and 1 participants; and partial remission was recorded in 1, 1, 1, and 0 participants. Some response category breakdowns (such as stable disease and progressive disease) were not reported in the submitted data. For the measure of remaining detectable disease at the end of cycle one, the reported data shows most participants across dose levels still had detectable disease remaining, with smaller numbers (0, 1, 1, and 1 across the four groups) recorded as having undetectable levels. It is worth noting that this was a very small, early-phase trial, and the reported data describes only what was observed and measured in these 16 participants — it was not designed to draw broad conclusions. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 1 Leukaemia Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People aged 12 months to 21 years old at the time of joining the study.
- People who have a certain type of leukemia (ALL) with a high level of cancer cells in the bone marrow (25% or more), with or without cancer spread to other areas.
- People whose leukemia has not responded well to treatment, shown by a specific bone marrow test finding 0.1% or more cancer cells, done at an approved testing laboratory.
- People newly diagnosed with B-cell or T-cell ALL whose disease did not respond to consolidation (an early phase of intensive) therapy.
- People with B-cell ALL experiencing their first relapse (cancer returning) whose disease has not responded to treatment.
- People with B-cell ALL experiencing a second or later relapse, either at the time of relapse or if the disease has not responded to treatment.
- People with T-cell ALL experiencing a first or later relapse, either at the time of relapse or if the disease has not responded to treatment.
- People with lymphoblastic lymphoma or peripheral T-cell lymphoma that has come back or has not responded to treatment, with the diagnosis confirmed by tissue testing, and with measurable signs of disease present at the time of joining.
- People who are well enough to carry out some daily activities, as measured by standard medical scoring tools appropriate for their age.
- People who have recovered from the side effects of any previous cancer treatment.
- People whose kidneys, liver, heart, lungs, and bone marrow are working at an adequate level, based on test results.
- People whose blood triglyceride (a type of fat) and cholesterol levels are at or below 300 mg/dL.
- Female patients of childbearing potential who have a negative pregnancy test before joining.
- People who agree to use effective contraception during the study.
- People with blood sugar levels within the normal range for their age.
Who may not be able to join:
- People with isolated CNS (brain/spinal fluid) disease and very low bone marrow cancer cell levels (below 0.1%) — these people are not eligible under the leukemia criteria.
- People currently taking steroid medications (corticosteroids) whose dose has not been stable or decreasing for at least 7 days before joining.
- People currently taking another experimental drug not approved by regulatory authorities in the USA, Canada, or Australia.
- People currently receiving or expected to receive other cancer treatments, radiation therapy, or immunotherapy during the study (with limited exceptions such as hydroxyurea or intrathecal chemotherapy given up to one week before starting the study).
- People taking medications to prevent organ rejection or graft-versus-host disease (such as cyclosporine or tacrolimus) unless enough time has passed since the last dose.
- People currently taking blood-thinning medications, including aspirin or low molecular weight heparin, unless enough time has passed since the last dose.
- People currently taking ACE inhibitors (a type of blood pressure medication) unless enough time has passed since the last dose.
- People currently taking calcium channel blockers (another type of blood pressure or heart medication) unless enough time has passed since the last dose.
- People taking certain anti-seizure medications that affect how the liver processes drugs (such as phenytoin, phenobarbitol, or carbamazepine), unless enough time has passed since the last dose.
- People taking certain antifungal medications (such as fluconazole or voriconazole) unless enough time has passed since the last dose.
- People who have had a stem cell transplant and have active graft-versus-host disease, or where fewer than 84 days have passed since the transplant.
- People who have had certain types of radiation therapy without the required waiting period having passed.
- People with a positive blood culture within 48 hours of joining, a fever with signs of infection within 48 hours, a positive fungal culture within 30 days, or an active infection requiring intravenous treatment.
- People with Burkitt's leukemia or Burkitt's lymphoma.
- People with Down syndrome or Fanconi Anaemia.
- People with known involvement of the optic nerve or retina (the structures related to vision).
- People with a serious illness, psychiatric condition, or personal situation that could affect their safety or ability to follow the study requirements.
- People whose platelet counts are below 20,000/mm³ or who are known not to respond to blood or platelet transfusions.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Susan Rheingold, MD, Children's Hospital of Philadelphia
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
4 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Number of Patients That Experienced DLT During Cycle 1 of Therapy
Can't join this trial?
Data last synced from ClinicalTrials.gov: 22 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.