Real-world outcomes for adults with AML treated with new drugs (BoxTrial)
Observational GIMEMA Study on the Outcome of Acute Myeloid Leukemia (AML) Patients Treated With New Drugs in Real-life
This project will follow adults with AML treated in routine care across Italy to see how new approved drugs and combinations perform over at least two years.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 397 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Gruppo Italiano Malattie EMatologiche dell'Adulto Academic / other |
| Drugs / interventions | gemtuzumab, gilteritinib |
| Locations | 2 sites (San Giovanni Rotondo and 1 other locations) |
| Trial ID | NCT06513273 on ClinicalTrials.gov |
What this trial studies
This multicenter observational project combines prospective and retrospective data collection at Italian GIMEMA centers to describe how newly approved AML drugs and combinations are used in routine practice. It enrolls adults (≥18 years) with AML according to ELN guidelines, excluding acute promyelocytic leukemia (M3), who start AIFA-authorized novel therapies through 31 December 2027. No extra procedures or visits beyond standard clinical care are required, and each patient will be followed for a minimum of 24 months. The protocol focuses on patients treated with agents such as gilteritinib for FLT3-mutant AML, IDH inhibitors, oral azacitidine maintenance, glasdegib, gemtuzumab ozogamicin, and other drugs approved during the study period.
Who should consider this trial
Good fit: Adults (≥18 years) with AML by ELN criteria (excluding M3) who begin AIFA-authorized novel monotherapy or combination therapy at a participating GIMEMA center and consent to data collection are eligible, provided they are not enrolled in interventional trials.
Not a fit: Patients enrolled in interventional clinical trials, pediatric patients, and those with acute promyelocytic leukemia (M3) are outside the intended population and are unlikely to benefit from this observational project's findings.
Why it matters
Potential benefit: If successful, the project could provide clearer real-world evidence on how new AML treatments perform outside clinical trials, helping clinicians and patients make more informed treatment choices.
How similar studies have performed: Randomized clinical trials have shown benefit for several targeted AML agents (for example FLT3 and IDH inhibitors, oral azacitidine, and gemtuzumab), but large multicenter real-world observational data confirming their routine-practice performance remain limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Aged 18 years or older 2. AML diagnosis according to the ELN guidelines, excluding M3 3. Signed Informed consent, if applicable 4. Treatment initiation with novel drugs in monotherapy or combination, in accordance with the AIFA authorizations, from the AIFA registration up to 31.12.2027 with particular attention to: * patients affected by FLT3-mutated AML treated with gilteritinib. * patients affected by IDH-mutated AML treated with IDH inhibitors. * patients affected by AML in maintenance therapy with oral azacytidine. * patients affected by AML treated with glasdegib. * patients affected by AML treated with gemtuzumab ozogamicin. * other novel drugs or combination for the treatment of AML approved during the study period. Exclusion Criteria: Patients included in interventional clinical trials.
Where this trial is running
San Giovanni Rotondo and 1 other locations
- Ematologia Ente Ecclesiastico Casa Sollievo Della Sofferenza — San Giovanni Rotondo, Italy (Recruiting)
- UOC Ematologia AOU di SASSARI — Sassari, Italy (Recruiting)
Study contacts
- Principal investigator: Anna Candoni — Haematology, University of Modena and Reggio Emilia
- Study coordinator: Paola Fazi
- Email: p.fazi@gimema.it
- Phone: 0670390528
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.