International multicenter, open-label, phase 2 study to treat molecular relapse of pediatric acute myeloid leukemia with azacitidine

Phase 2

Recruiting

• Conditions: Acute myeloid leukemia; 10024324; AML

• Registration Number: NL-OMON52770
• Lead Sponsor: German Pediatric Oncology Group

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 6

Inclusion Criteria

1. Aged 3 months to <21 years with documented diagnosis of AML according to WHO
classification with at least one quantitative genetic maker, e.g. one of the
following
aberrations:
• t(8;21); RUNX1-RUNX1T1
• inv(16); CBFb-MYH11
• t(9;11); MLL-AF9
• t(10;11); MLL-AF10
• NPM1
• FLT3-ITD
• WT1; etc.
2. First complete remission (MRD in PB less than 5 x 10-4) confirmed at the
start of last
consolidation course or within 1 month after completion of consolidation
treatment
3. Detection of a confirmed molecular relapse of an AML
4. Understand and voluntarily provide permission (subjects and when applicable,
parental/legal representative(s)) to the ICF prior to conducting any study
related
assessments/procedures
5. Able to adhere to the study visit schedule and other protocol requirements
6. Lansky performance score at least equal to 50; or Karnofsky performance
status at
least equal to 50, whichever is applicable
7. Negative serum pregnancy tests for females of child bearing potential within
10 days
prior to treatment

Exclusion Criteria

1. Concomitant treatment with any other anticancer therapy except those
specified in
protocol
2. HSCT within previous 3 months
3. Treated by any investigational agent in a clinical study within previous 4
weeks
4. Pregnancy or lactating
5. FAB type M3 leukemia (acute promyelocytic leukemia)
6. Therapy-related AML
7. AML of Down syndrome or other congenital syndromes giving rise to leukemia or
treatment complications
8. Symptomatic cardiac disorders (CTCAE 4.0 Grade 3 or 4)
9. Evidence of invasive fungal infection or other severe systemic infection
requiring
treatment doses of systemic/parenteral therapy including known active viral
infection
with human immunodeficiency virus (HIV) or Hepatitis Type B and C
10. Any other organ dysfunction (CTCAE 4.0 Grade 3 or 4) that will interfere
with the
administration of the therapy according to this protocol
11. Ongoing severe toxicities (CTCAE 4.0 Grade 3 or 4) of prior
chemotherapy/stem cell
transplantation
12. Hypersensitivity to the active substance or other excipients contained in
the
investigational medical product listed in the summary of product
characteristics (SmPC)
or Investigators Brochure (IB).
13. Abnormal liver function:
a. serum bilirubin > 3 x ULN or
b. ALT or AST > 5 times ULN
14. Symptomatic CNS-involvement or isolated extramedullary disease at initial
diagnosis
15. Female and male subjects with child bearing potential who avoid using
highly effective
anticonceptive measure(ment)s

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary endpoint based on molecular response will be assessed at the end of<br /><br>the azacitidine treatment.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>• Toxicities<br /><br>• Event-free-survival<br /><br>• Disease free survival<br /><br>• Overall-survival<br /><br>• Quality of life</p><br>