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

Primary Purpose

Acute Myeloid Leukemia, Adult

Status

Unknown status

Phase

Phase 2

Locations

Italy

Study Type

Interventional

Intervention

Up-front allogeneic hematopoietic stem cell transplantation (HSCT)

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia, Adult

Eligibility Criteria

65 Years - 75 Years (Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Fondazione Policlinico Universitario A. Gemelli IRCCS

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Allogeneic hematopoietic stem cell transplantation

Arm Description

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

Outcomes

Primary Outcome Measures

Rate of complete remission equal or higher than 60%

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT03902665

First Posted

April 2, 2019

Last Updated

April 8, 2019

Sponsor

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Collaborators

Associazione Italiana per la Ricerca sul Cancro

1. Study Identification

Unique Protocol Identification Number

NCT03902665

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2019

Overall Recruitment Status

Unknown status

Study Start Date

March 15, 2019 (Actual)

Primary Completion Date

March 15, 2021 (Anticipated)

Study Completion Date

March 30, 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Collaborators

Associazione Italiana per la Ricerca sul Cancro

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

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%.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Acute Myeloid Leukemia, Adult

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

3 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Allogeneic hematopoietic stem cell transplantation

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Up-front allogeneic hematopoietic stem cell transplantation (HSCT)

Other Intervention Name(s)

HSCT

Intervention Description

Patients classified as fit/unfit are included in the HSCT program. There are two early approaches allowed. A) Patients will be left untreated until HSCT B) Patients will receive 1 short course of chemotherapy before HSCT (Ara-C and anthracycline). Selection of strategy A or B, will be patient based on disease characteristics and dynamics or presence of high tumor load. Conditioning for haplo-HSCT should be started as soon as possible, within day 45 after initial diagnosis. This is to avoid delayed transplantation. Two dosing levels of the Thiotepa-Busulfan-Fludarabine (TBF) based protocol are allowed based on the clinical condition of the patient: fit patients below 70 will receive the TBF with 2 days of Busulfan, whereas patients with poorer clinical condition or above the age of 70 will receive a dose-reduced TBF, in which Busulfan may be reduced to 1 day only.

Primary Outcome Measure Information:

Title

Rate of complete remission equal or higher than 60%

Description

Time Frame

From day 56 to day +70 post-transplant

10. Eligibility

Sex

All

Minimum Age & Unit of Time

65 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Andrea Bacigalupo, Prof.

Organizational Affiliation

Fondazione Policlinico Universitario A. Gemelli, IRCCS

Official's Role

Principal Investigator

Facility Information:

Facility Name

Fondazione Policlinico Universitario A. Gemelli IRCCS

City

Roma

State/Province

ZIP/Postal Code

00168

Country

Italy

12. IPD Sharing Statement

Learn more about this trial

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

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