A randomized, multicenter, open-label, Phase 2 study with a safety run-in part to evaluate safety, pharmacodynamics and efficacy of azacitidine compared to no anticancer treatment in children and young adults with acute myeloid leukemia in molecular relapse after first complete remissio
- Conditions
- acute myeloid leukemiacancer of the blood10024324
- Registration Number
- NL-OMON44834
- Lead Sponsor
- Celgene Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 42
Patients* 3months and less than 18 years old, and/or when applicable parental/ legal representative(s), must understand and voluntarily sign an Informed Consent or Informed Assent Form according to local law and regulations. *At the time of signing the ICF/IAF, patients must have documented diagnosis of Acute Myeloid Leukemia and documentation of molecular remission confirmed at the start of last consolidation course or within 1 month after completion of consolidation treatment. *For the safety run-in: Detection of molecular relapse within the 7 days prior to signing consent/assent and confirmation of molecular relapse during the screening period.
*Concomitant treatment with any other anticancer therapy except those
specific in protocol and no maintenance therapy after end of
consolidation therapy and confirmed remission.
*Hematopoietic Stem Cell Transplantation within previous 3 months
*Pregnant or breastfeeding females
*Patients with a current disease that can interfere with protocol
procedures or study treatment
*Hypersensitivity to azacitidine
*Symptomatic CNS-involvement or isolated extramedullary disease at
initial diagnosis.
*FAB type M3 leukemia (acute promyelocytic leukemia)
*Therapy-related AML
* AML of Down syndrome or other congenital syndromes giving rise to
leukemia or
treatment complications.
*Symptomatic cardiac disorders (CTCAE Grade 3 or 4).
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Safety Run-in Part:<br /><br>- Identification of a safe and tolerable dose for the randomized part of the<br /><br>study<br /><br>- Assessment of treatment-related dose-limiting toxicities (DLTs)<br /><br>- Frequency and severity of treatment-related AEs<br /><br><br /><br>Randomized Part:<br /><br>- Progression-free rate at Day 84 (±4 days) post randomization.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Safety Run-Part:<br /><br>- Azacitidine plasma PK parameters<br /><br>- Changes in DNA methylation (assessments of BM samples using Nano-HELP assay)<br /><br>- Leukemia-free survival (LFS)<br /><br>- Minimal residual disease pre- HSCT, and 3 and 6 months post-HSCT<br /><br>- Overall survival<br /><br><br /><br>Randomized Part:<br /><br>- Changes in DNA methylation<br /><br>- Leukemia-free survival<br /><br>- Proportion treated with HSCT<br /><br>- MRD pre- HSCT, and 3 and 6 months post-HSCT<br /><br>- Overall survival<br /><br>- Molecular response<br /><br>- Treatment-related mortality/morbidity<br /><br>- Toxicity data after HSCT<br /><br>- Safety </p><br>