NCT05599360

Vyxeos for Induction of Low- or Intermediate-risk.

Active, enrolled Phase 2 Last updated 2 December 2024

What this trial tests

Phase 2 trial testing Vyxeos in Leukemia, Myeloid, Acute in 20 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

7 August 2022

Primary endpoint
7 September 2028

7 September 2028

Quick facts

Lead sponsor	Shaare Zedek Medical Center
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	20
Start date	7 August 2022
Primary completion	7 September 2028
Estimated completion	7 September 2028
Sites	1 location across Israel

Drugs / interventions tested

• Vyxeos — full drug profile →
• Mylotarg (gemtuzumab-ozogamicin) — full drug profile →

Conditions studied

• Leukemia, Myeloid, Acute — all drugs for Leukemia, Myeloid, Acute →

Sponsor

Shaare Zedek Medical Center

Who can join

Adults 18 to 70, any sex, with Leukemia, Myeloid, Acute. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Vyxeos Vyxeos is a liposomal-encapsulated combination of cytarabine and daunorubicin, at a molar ratio of 5:1. Delivery of the 5:1 molar ratio seems to prevent antagonistic drug-drug interactions and the liposomal encapsulation increases the plasma half-life of cytarabine and daunorubicin and leads to drug accumulation within the bone marrow (BM).

Despite previous results that highlighted the advantage of Vyxeos for sAML, it is intuitively likely that this powerful drug is also suitable for non-sAML. The mechanism of action is relevant for every AML. Following the FDA approval of the drug for sAML we would like to evaluate its efficacy for low or intermediate risk fms-like tyrosine kinase 3 (FLT3)-negative de novo AML patients. This consideration is particularly relevant by the inclusion of young AML patients in the study.

Gemtuzumab ozogamicin (GO) Gemtuzumab ozogamicin (Mylotarg) - an anti-cluster of differentiation 33 (CD33) monoclonal antibody linked to calicheamicin, was approved for the treatment of newly diagnosed AML patients, when given as a combination with the '7+3' regimen.

One of the goals of the current study is to examine the feasibility and efficacy of the combination of Mylotarg plus Vyxeos.

Minimal/ measurable residual disease (MRD) Minimal or measurable residual disease (MRD) denotes the presence of leukemia cells down to levels of 1:10-4 to 1:10-6, compared with 1:20 in morphology-based assessments. MRD can be evaluated using a variety of multiparameter flow cytometry (MFC) and molecular methods. There are no data regarding the achievement or impact of MRD using Vyxeos as induction therapy. The current trial will address this issue.

Purpose of this Trial The current study is designed to examine the response rate of the Vyxeos as induction therapy for newly diagnosed low/intermediate risk AML patients in the 'real world' setting. Patients will receive the same induction therapy that they were to receive had they not entered this study (cytarabine /daunorubicin ± Mylotarg) but the combination of cytarabine /daunorubicin will be given in the unique formulation of Vyxeos. In addition to classic CR+CRi evaluation, MFC MRD evaluation, using an centralized, internationally recognized laboratory, will be done at the end of induction. In addition, this pilot study will also provide clinical safety information about the combination of Vyxeos with Mylotarg.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Advancements in antibody-drug conjugates as cancer therapeutics.
   Fong JY, Phuna Z, Chong DY, Heryanto CM, et al · · 2025 · cited 11× · PMID 40814440 · DOI 10.1016/j.jncc.2025.01.007
2. CPX-351 +/- gemtuzumab ozogamicin as induction therapy for adult patients with newly diagnosed, favourable-intermediate risk, FLT3-ITD negative, AML: A pilot study.
   Ganzel C, Frisch A, Wolach O, Moshe Y, et al · · 2025 · cited 2× · PMID 39721590 · DOI 10.1111/bjh.19967