Low Dose Daunorubicin in Pediatric Relapsed/Refractory Acute Leukemia
United States1 participantsStarted 2020-05-08
Plain-language summary
In this pilot study, eligible pediatric patients will be treated with 5 consecutive days of low dose daunorubicin. All patients who receive low dose daunorubicin will be evaluated daily for potential toxicity during those 5 days. Once the patient has received 5 doses of daunorubicin, subsequent therapy will be at the discretion of the primary oncology team.
Who can participate
Age range
1 Year – 21 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Patients with pathologically confirmed ALL or AML, whose disease is refractory to two induction therapeutic attempts, or who are in 2nd or greater relapse, or who are in 1st relapse or refractory to a single therapeutic attempt but are unable to receive intensive therapy at the time of consent.
* All prior upfront therapies including bone marrow transplant are acceptable. Pulse steroids (of 5 days duration or less in the prior month) administered as part of a routine maintenance therapy are acceptable.
* Age 1 to 21 years of age, inclusive
* Established central catheter IV access
Exclusion Criteria:
* Females who are known to be pregnant or lactating
* Any Grade 3 or higher Cardiac Disorder per CTCAE version 5
* Patients with echocardiographic evidence of cardiomyopathy (shortening fraction \<27% or ejection fraction \<50%)
* Uncontrolled sepsis
* Absolute Blast Count \>50 x10(3)/mcL at enrollment or on day 1 of study
* Direct hyperbilirubinemia \>5mg/dL
* Grade 3 or higher anaphylaxis to daunorubicin
* Non-English speaking
* Patients, who in the opinion of the PI, are unable to tolerate any study-specific procedures
* Patients who have received cyclosporine, tacrolimus or other agents to prevent or treat graft-vs-host disease post bone marrow transplant in the last 14 days
* Concurrent investigational drugs or other chemotherapeutic agents (excluding hydroxyurea), immunotherapies or biosimilars during the 5 days of daunorubicin.
* Prior cumulative do…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Low Dose Daunorubicin Feasbility as Assessed by Absolute Blast Count
Timeframe: 24 months
2
Incidence of Low Dose Daunorubicin Feasbility as Assessed by Extramedullary Leukemia Progression
Timeframe: 24 months
3
Incidence of Low Dose Daunorubicin Feasbility as Assessed by Patient Symptoms
Timeframe: 24 months
4
T-cell Based Immune Responses Against Chemoresistant Leukemia Stem Cells (LSC) Are Stimulated at Lower Doses of Daunorubicin to Provide Preliminary Data for Further Research.
Timeframe: 24 months
5
The Pro- vs. Anti-cancer Cellular Immune Response of Targeted Anthracycline Treatment in Patients With Relapsed/Refractory Acute Leukemia