Master Framework For Relapse or Refractory Acute Myeloid Leukemia

Recruiting

• Conditions: Acute Myeloid Leukemia, in Relapse; Acute Myeloid Leukemia Refractory
• Interventions: Other: Registration into the STREAM platform

• Registration Number: NCT06459024
• Lead Sponsor: Istituto Romagnolo per lo Studio dei Tumori Dino Amadori IRST S.r.l. IRCCS

Brief Summary

This is an observational (non-interventional), prospective, cohort study that will collects data from patients diagnosed with relapsed or refractory acute myeloid leukemia afferent to the participanting clinical sites

Detailed Description

Treatment outcome for acute myeloid leukemia (AML) has improved over the last few decades. With intensive multidrug induction regimens, a complete remission is expected. Despite such advances, therapy resistance or relapse remains the main causes of death in adult patients and in children. Relapsed or refractory (R/R) AML is a hard-to-treat disease, with a 5-year overall survival (OS) estimate below 20% in adults and 30%-35% in children, and no standard of care exists. HSCT (allogeneic hematopoietic stem cell transplantation) remains the only curative strategy for R/R AML patients, including pediatric and elderly ones. Some new personalized therapies have recently been integrated into the treatment options of R/R AML. However, there is a need to collect consistent data on the efficacy of these new strategies in real-world settings. Lack of data from clinical trials can be a barrier in most countries, limiting patient accessibility to some very effective drugs. The STREAM study is part of the Horizon Europe Mission Cancer 2022 IMPACT-AML project, that proposes to create an inclusive master framework for relapsed or refractory acute myeloid leukemia. In STREAM we will collect data and monitor outcomes of patients with R/R AML across Europe. In close collaboration with the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet, GA101157011), STREAM is developed under ERN-EuroBloodNet central registry, the European Rare Blood Disorders Platform (ENROL), incorporating EU standards for interoperability and the GPDR-compliant SPIDER pseudonymization tool offered by the EU-RD Platform in the context of rare disease registries will be implemented, to allow cross-hospital and cross-country participation. The STREAM study will allow to collect high-quality data from patients at any stage of the disease, for any cancer subtype, in any age group, including pediatric and elderly population, unfit patients, patients with rare mutations, patients with high-impact end-organ damage, patients from rural regions, post-transplant relapse. Data collected in STREAM will foster the production of novel knowledge on rare populations in a pragmatic setting and will form the basis for building international trials to be conducted worldwide.

Study Timeline

• Study First Submitted: 2024-06-10
• Study First Submitted QC: 2024-06-10
• Study First Posted: 2024-06-14
• Study Start: 2024-06-28
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-28
• Last Update Posted: 2025-04-29
• Primary Completion: 2032-07
• Study Completion: 2032-07

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 4000

Inclusion Criteria

• Patients with AML diagnosis according to WHO2022 or ICC2022
• Treatment failure (i.e. relapse, refractory or progression, including MRD) according to ELN2022 criteria
• Participant or his/her legal representative is willing and able to give informed consent for participation in the study

Exclusion Criteria

• Patients included in clinical trials may be enrolled except where otherwise specified in the experimental protocol.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with relapsed or refractory acute myeloid leukemia	Registration into the STREAM platform	All patients with relapsed or refractory acute myeloid leukemia

Primary Outcome Measures

Name	Time	Method
The primary endpoint is to determine the overall survival of R/R AML patients	8 years	Overall survival is defined as the time from R/R diagnosis to death due to any cause.

Secondary Outcome Measures

Name	Time	Method
Remission rate	8 years	Response is defined according to ELN 2022 criteria. Rates of CR, CRi, CRh and overall response (CR+CRi+CRh) as best response will be considered
Relapse free survival	8 years	Relapse-free survival is defined for patients achieving a response to rescue treatment as the time from response onset to relapse or death due to any cause, whichever occurs first.
Association between baseline clinical, molecular and biological parameters and clinical outcomes	8 years	Rates of CR, CRi, CRh and overall response (CR+CRi+CRh) in specific sub-populations bearing common molecular features
Describe the methods for diagnosis and the treatment options offered to the patients affected by R/R AML	8 years	type and frequency of diagnostic procedures used and treatments prescribed
Event-free survival	8 years	Event-free survival is defined as the time from R/R diagnosis to rescue treatment failure (according to ELN 2022 definitions), relapse or death due to any cause, whichever occurs first.
To characterize potential populations that could be target for future studies/clinical trials	8 years	descriptive statistics (mean, median, frequency, range) of subpopulations characteristics
Transplant rate	8 years	Proportion of patients undergoing transplantation

Trial Locations

Locations (30)

University Hospital Greifswald; 🇩🇪 Greifswald, Germany

University Hospital Halle; 🇩🇪 Halle, Germany

University Hospital of Rostock; 🇩🇪 Rostock, Germany

Policlinico Sant'Orsola-Malpighi; 🇮🇹 Bologna, BO, Italy

Ospedale "A. Perrino"; 🇮🇹 Brindisi, BR, Italy

ASST Cremona; 🇮🇹 Cremona, CR, Italy

ASST Spedali Civili; 🇮🇹 Brescia, BS, Italy

Azienda Ospedaliera S. Croce e Carle; 🇮🇹 Cuneo, CN, Italy

Ospedale Valduce; 🇮🇹 Como, CO, Italy

IRCCS Ospedale Policlinico San Martino; 🇮🇹 Genova, GE, Italy

ASST Grande Ospedale Metropolitano Niguarda; 🇮🇹 Milano, MI, Italy

Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello; 🇮🇹 Palermo, PA, Italy

Ospedale Santa Maria delle Croci; 🇮🇹 Ravenna, RA, Italy

Fondazione Policlinico Tor Vergata; 🇮🇹 Roma, RM, Italy

AOU Universitaria Arcispedale Sant'Anna; 🇮🇹 Ferrara, FE, Italy

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico; 🇮🇹 Milano, MI, Italy

Presidio Ospedaliero "Santo Spirito"; 🇮🇹 Pescara, PE, Italy

Ospedale Santa Maria della Misericordia; 🇮🇹 Perugia, PG, Italy

Policlinico Umberto I; 🇮🇹 Roma, RM, Italy

Policlinico Universitario Fondazione Agostino Gemelli; 🇮🇹 Roma, RM, Italy

Ospedale Sant'Andrea; 🇮🇹 Roma, RM, Italy

Ospedale infermi di Rimini; 🇮🇹 Rimini, RN, Italy

AOU San Giovanni di Dio e Ruggi d'Aragona; 🇮🇹 Salerno, SA, Italy

AOU Città della Salute e della Scienza di Torino; 🇮🇹 Torino, TO, Italy

AO Ordine Mauriziano; 🇮🇹 Torino, TO, Italy

IOV Istituto Oncologico Veneto; 🇮🇹 Castelfranco Veneto, TV, Italy

ASST Valle Olona; 🇮🇹 Busto Arsizio, VA, Italy

IRST Istituto Romagnolo per lo Studio dei Tumori Dino Amadori; 🇮🇹 Meldola, Italy

AOU Maggiore della Carità; 🇮🇹 Novara, Italy

University Hospital La Fe; 🇪🇸 Valencia, Spain