NCT01723657

Risk Adapted Treatment for Primary AML in Adults, Based on Genetics and Minimal Residual Disease (MRD) in Patients up to the Age of 70 Years.

Completed Phase 2 Last updated 6 November 2012

What this trial tests

Phase 2 trial testing Ara-C in Leukemia, Myelocytic, Acute in 862 participants. Completed in 1 March 2012.

Timeline

1 October 2003

Primary endpoint
1 March 2012

1 March 2012

Quick facts

Lead sponsor	Grupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	862
Start date	1 October 2003
Primary completion	1 March 2012
Estimated completion	1 March 2012
Sites	21 locations across Spain

Drugs / interventions tested

• Ara-C — full drug profile →
• Autologous peripheral blood stem cell transplantation.
• Allogeneic matched related or unrelated donor transplant.
• G-CSF (g-csf) — full drug profile →
• CD34+ selection.
• Mylotarg purging before autologous PBSC transplantation

Conditions studied

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

Sponsor

Grupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias — full company profile →

Who can join

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

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Complete remission rate (CRR)
  Time frame: 2 months.
  Analyze the efficacy and toxicity of IDICE-G (idarubicin, intermediate doses of ara-C and etoposide) and G-CSF to achieve complete remission.
• Disease free survival (DFS).
  Time frame: 4 years.
  Analyze the disease free suvival of patients in remission, with a therapeutic strategy adjusted to the prognostic factors. Mortality in remission after chemotherapy and/or peripheral blood stem cell tranplantation.

Sponsor's own description

The AML-03 regimen investigates the addition of G-CSF priming to both induction and consolidation chemotherapies administrated in the previous AML-99 trial (NCT01716793) refines risk-stratification based on biological characterization also the AML-03 trial incorporates novel approaches for hematopoietic stem cell transplantation: such as Mylotarg™ "in vivo purging" in autografts, extends unrelated volunteers donors for allotransplants in high-risk patients, and introduces reduced intensity conditioning in patients with elder age (more than 50 years old).

The aims of these modifications are to analyse eficacy and toxicity of this induction and consolidation therapy and to analyse the disease free survival in patients who achieved complete response following a risk adjusted therapy.

Publications & conference data

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

1. The lincRNA HOTAIRM1, located in the HOXA genomic region, is expressed in acute myeloid leukemia, impacts prognosis in patients in the intermediate-risk cytogenetic category, and is associated with a distinctive microRNA signature.
   Díaz-Beyá M, Brunet S, Nomdedéu J, Pratcorona M, et al · · 2015 · cited 82× · PMID 26436590 · DOI 10.18632/oncotarget.5148
2. MRD Tailored Therapy in AML: What We Have Learned So Far.
   Ngai LL, Kelder A, Janssen JJWM, Ossenkoppele GJ, et al · · 2020 · cited 37× · PMID 33575214 · DOI 10.3389/fonc.2020.603636
3. Prognostic impact of DNMT3A mutation in acute myeloid leukemia with mutated NPM1.
   Oñate G, Bataller A, Garrido A, Hoyos M, et al · · 2022 · cited 30× · PMID 34516636 · DOI 10.1182/bloodadvances.2020004136
4. Minimal Residual Disease in Acute Myeloid Leukemia: Still a Work in Progress?
   Mosna F, Capelli D, Gottardi M. · · 2017 · cited 25× · PMID 28587190 · DOI 10.3390/jcm6060057
5. A three‑lncRNA signature for prognosis prediction of acute myeloid leukemia in patients.
   Wang F, Tian X, Zhou J, Wang G, et al · · 2018 · cited 21× · PMID 29901168 · DOI 10.3892/mmr.2018.9139
6. The expression level of BAALC-associated microRNA miR-3151 is an independent prognostic factor in younger patients with cytogenetic intermediate-risk acute myeloid leukemia.
   Díaz-Beyá M, Brunet S, Nomdedéu J, Cordeiro A, et al · · 2015 · cited 18× · PMID 26430723 · DOI 10.1038/bcj.2015.76
7. Therapeutic Targeting of Acute Myeloid Leukemia by Gemtuzumab Ozogamicin.
   Gottardi M, Simonetti G, Sperotto A, Nappi D, et al · · 2021 · cited 16× · PMID 34572794 · DOI 10.3390/cancers13184566
8. Update on acute myeloid leukemia stem cells: New discoveries and therapeutic opportunities.
   Stahl M, Kim TK, Zeidan AM. · · 2016 · cited 15× · PMID 27822339 · DOI 10.4252/wjsc.v8.i10.316