Phase 2
N=1
Low Dose Daunorubicin in Pediatric Relapsed/Refractory Acute Leukemia
Relapsed Pediatric ALL · Relapsed Pediatric AML · Refractory Acute Myeloid Leukemia · Refractory Acute Lymphoblastic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT04562792 ↗Enrolled (actual)
1
Serious AEs
0.0%
Results posted
May 2023
Primary outcome: Primary: Incidence of Low Dose Daunorubicin Feasbility as Assessed by Absolute Blast Count — 0; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Daunorubicin (Drug)
- Age
- Pediatric, Adult · 1+ yrs
- Sex
- All
- Sponsor
- Children's Mercy Hospital Kansas City
- Primary completion
- Jun 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Low Dose Daunorubicin Feasbility as Assessed by Absolute Blast Count |
0; 1 | — |
| PRIMARY Incidence of Low Dose Daunorubicin Feasbility as Assessed by Extramedullary Leukemia Progression |
0; 1 | — |
| PRIMARY Incidence of Low Dose Daunorubicin Feasbility as Assessed by Patient Symptoms |
0; 1 | — |
| PRIMARY 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. |
— | — |
| PRIMARY The Pro- vs. Anti-cancer Cellular Immune Response of Targeted Anthracycline Treatment in Patients With Relapsed/Refractory Acute Leukemia |
— | — |
| SECONDARY Pharmacokinetic Parameters of Low Dose Daunorubicin in Children With Relapsed/Refractory AML and ALL as Assessed by Maximum Concentration. |
18.7 | — |
| SECONDARY Pharmacokinetic Parameters of Low Dose Daunorubicin in Children With Relapsed/Refractory AML and ALL as Assessed by Time at Maximum Concentration. |
0.33333 | — |
| SECONDARY Pharmacokinetic Parameters of Low Dose Daunorubicin in Children With Relapsed/Refractory AML and ALL as Assessed by Area Under the Curve. |
20.889865 | — |
| SECONDARY Pharmacokinetic Parameters of Low Dose Daunorubicin in Children With Relapsed/Refractory AML and ALL as Assessed by Elimination Half-life |
1.8588 | — |
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.
Eligibility Criteria
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 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 doses of anthracyclines will not be an exclusion regardless of the total cumulative dose previously received.
Data sourced from ClinicalTrials.gov (NCT04562792). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.