Up-front Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia Patients Aged 65-75

Phase 2

Completed

• Conditions: Acute Myeloid Leukemia, Adult
• Interventions: Drug: Up-front allogeneic hematopoietic stem cell transplantation (HSCT)

• Registration Number: NCT03902665
• Lead Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Brief Summary

Patients with acute myeloid leukemia aged 65-75 have a very poor prognosis, irrespective of the treatment strategy, including demethylating agents or conventional chemotherapy. With these approaches, remission rates do not exceed 40%, and overall disease-free survival at 1 year is in the order of 15%. The hypothesis is that up-front allogeneic hematopoietic stem cell transplant will produce a complete remission rate of 60% on day +56-70, and disease-free survival at 1 year of 30%. This is a single arm phase II study of upfront allogeneic stem cell transplantation, for patients with acute myeloid leukemia aged 65-75: the primary endpoint is a complete remission rate on day +56-70. The secondary endpoint is a 1-year overall disease-free survival of 30%.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-03-15
• Study First Submitted: 2019-04-02
• Study First Submitted QC: 2019-04-02
• Study First Posted: 2019-04-04
• Primary Completion: 2024-03-31
• Study Completion: 2024-06-30
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-22
• Last Update Posted: 2024-07-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 19

Inclusion Criteria

• Patients aged 65-75
• Patients with de novo or secondary acute myeloid leukemia (AML) - intermediate or high risk according to European LeukemiaNet (ELN) recommendations 2017
• Untreated patients at diagnosis of acute myeloid leukemia - patients may have received treatment for high-risk myelodysplastic syndromes with hypomethylating agents (HMA). They should not have received a course of induction chemotherapy to be eligible for this study
• Haploidentical family stem cell donor or other suitable donors available
• Fit and unfit patients by geriatric scale assessment
• Signed informed consent.

Exclusion Criteria

• Acute Myeloid Leukemia good risk according to European LeukemiaNet 2017
• Positive serology for Human Immunodeficiency Virus.
• Serious organ dysfunction: left ventricular ejection fraction < 40%, forced expiratory volume in one second (FEV1), forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLCO) <50% of predicted, Liver Function Tests > 5 x the upper limit of normal, or creatinine clearance <30 ml/min .
• Life expectancy less than 30 days.
• Frail patients by geriatric scale assessment

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Allogeneic hematopoietic stem cell transplantation	Up-front allogeneic hematopoietic stem cell transplantation (HSCT)	Day -6, -5 Thiotepa 5 mg/kg/day . Day -4 to -3 Busulfan i. v 3,2 mg/kg/day and fludarabine i.v. 50 mg/m2 /day Day -2 fludarabine i.v. 50 mg/m2 Day -1 Rest Day 0 Begin cyclosporine; Infusion of T cell replete bone marrow transplant Day 1 Begin mycophenolate mofetil Day 3 and 5 Cyclophosphamide 50 mg/kg IV and Mesna Day 6 G-colony stimulating factor

Primary Outcome Measures

Name	Time	Method
Rate of complete remission equal or higher than 60%	From day 56 to day +70 post-transplant	As a primary outcome measure, the rate of complete remission in treated patients will be evaluated. Complete remission will be assessed from days +56 to days +70 after transplant. A complete remission rate equal to or higher than 60% is expected.

Trial Locations

Locations (1)

Fondazione Policlinico Universitario A. Gemelli IRCCS; 🇮🇹 Roma, RM, Italy