Efficacy of Prophylactic Oral Eltrombopag for Promoting Platelet and Neutrophil Engraftment After Haploidentical Hematopoietic Stem Cell Transplantation in Patients with acute lymphoblastic leukemia: A Phase II Non Randomized-controlled Trial
Studying the effect of prophylactic oral Eltrombopag administration after haploidentical Hematopoietic-Stem-Cell-Transplantation on platelet and neutrophil engraftment
Design
Phase II clinical trial with a historical control group, non-randomized, and open-label
Settings and conduct
This study will be conducted as a phase II, non-randomized, open-label clinical trial with a historical control group at the Hematology, Oncology, and Cell Therapy Research Center. Patients with acute leukemias who undergo haploidentical bone marrow transplantation at this center and meet the eligibility criteria will be enrolled. The outcomes of interest will be compared with those of the historical control group.
Participants/Inclusion and exclusion criteria
Inclusion: Definitive diagnosis of acute lymphocytic leukemia, medical indication of haploidentical hematopoietic stem cell transplantation, the patient must be in complete remission before the transplant.
Exclusion:
patients with abnormal liver function tests such as alanine aminotransferase greater equal than 2.5 times the upper limit of normal or bilirubin greater than1 mg/dL will not enter the study.
Intervention groups
Intervention:
Patients in this group will receive prophylactic oral Eltrombopag, purchased from Nano Darou Company, a thrombopoietin receptor agonist to promote engrafment, at a daily dose of 150–300 mg, starting from the fifth day after transplantation and continuing daily until engraftment for a minimum of 10 days.
Historical Control Group:
Patients in this group will receive standard conditioning regimen drugs without any engraftment-promoting medication.
Research Institute for Hematology, Oncology and Stem Cell Transplantation,Tehran University of Medic
Country
Iran (Islamic Republic of)
Phone
+98 21 8490 3691
Email address
ctu@sina.tums.ac.ir
Recruitment status
recruiting
Funding source
Expected recruitment start date
2025-10-23, 1404/08/01
Expected recruitment end date
2027-10-23, 1406/08/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Efficacy of Prophylactic Oral Eltrombopag for Promoting Platelet and Neutrophil Engraftment After Haploidentical Hematopoietic Stem Cell Transplantation in Patients with acute lymphoblastic leukemia: A Phase II Non Randomized-controlled Trial
Public title
Does Eltrombopag Improve Blood Cell Recovery in Leukemia Patients After Transplant?
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Definitive diagnosis of acute lymphocytic leukemia
Medical indication of Haploidentical Hematopoietic Stem Cell Transplantation
The patient must be in Complete Remission before the transplant
Exclusion criteria:
Patients with abnormal liver function tests such as alanine aminotransferase greater equal than 2.5 times the upper limit of normal or bilirubin greater than1 mg/dL will not enter the study.
Age
No age limit
Gender
Both
Phase
2
Groups that have been masked
No information
Sample size
Target sample size:
30
Randomization (investigator's opinion)
Not randomized
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Research Ethics Committees of Research Institute for Oncology, Hematology and Cell Therapy - Tehran
Street address
Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, North Kargar Street, Jalal Al-Ahmad Intersection, Tehran
City
Tehran
Province
Tehran
Postal code
1411713135
Approval date
2025-06-02, 1404/03/12
Ethics committee reference number
IR.TUMS.HORCSCT.REC.1404.017
Health conditions studied
1
Description of health condition studied
Acute lymphoblastic leukemia [ALL]
ICD-10 code
C91.0
ICD-10 code description
Acute lymphoblastic leukemia [ALL]
Primary outcomes
1
Description
Platelet engraftment (when the patient is independent of platelet transfusion for at least 7 days with a platelet count >20 × 10⁹/L)
Timepoint
Daily measurements, from the start of the intervention and at least until 10 days after the intervention.
Method of measurement
According to the complete blood count (CBC) laboratory test performed using an automated cell counter.
2
Description
Neutrophil engraftment (when the absolute neutrophil count consistently exceeds 0.5 × (10)9/L for 3 days in a row without a growth factor support.)
Timepoint
Daily measurements, from the start of the intervention and at least until 10 days after the intervention.
Method of measurement
According to the complete blood count (CBC) laboratory test performed using an automated cell counter.
3
Description
The occurrence of Graft versus Host disease according to the MAGIC criteria
Timepoint
Daily assessments , from 5 days before the start of the intervention (from the day of transplantation) and until 3 months after the intervention.
Method of measurement
In accordance with the Mount Sinai Acute GVHD International Consortium (MAGIC) criteria
4
Description
Overall survival
Timepoint
Patients will be followed from 5 days before the start of the intervention daily during hospitalization and then monthly after discharge and until 2 years after the intervention
Method of measurement
Survival data will be collected using a checklist that includes survival status and time of assessment by trained personnel during hospitalization in the ward and during post-discharge follow-ups.
5
Description
Relapse
Timepoint
Patients will be followed from 5 days before the start of the intervention daily during hospitalization and then monthly after discharge and until 2 years after the intervention
Method of measurement
Relapse data will be collected using a checklist that includes relapse status and time of assessment by trained personnel during hospitalization in the ward and during post-discharge follow-ups according to patients clinical records.
6
Description
Progression Free survival
Timepoint
Patients will be followed from 5 days before the start of the intervention daily during hospitalization and then monthly after discharge and until 2 years after the intervention
Method of measurement
Survival and relapse data will be collected using a checklist that includes survival and relapse status and time of assessment by trained personnel during hospitalization in the ward and during post-discharge follow-ups according to patients clinical records.
7
Description
Cytomegalovirus (CMV) reactivation
Timepoint
Weekly assessments , from 5 days before the start of the intervention (from the day of transplantation) and until 3 months after the intervention.
Method of measurement
Polymerase chain reaction (PCR) lab test
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: Patient with acute leukemia and recipient of haploidentical transplant will receive oral Eltrombopag, which is a thrombopoietin receptor agonist and will be used to promote post- allogenic-hematopoietic-stem-cell-transplantation engraftment, at a daily dose of 150–300 mg starting from fifth day after transplantation and continuing daily until engraftment. Each patient will receive Eltrombopag for a minimum of 10 days, regardless of whether engraftment occurs before day 10 of Eltrombopag therapy. Patients must receive at least 10 consecutive days of Eltrombopag in order to be included in the final analysis. As a safety rule, if the patient’s platelet count reaches or exceeds 500,000 per μL after 10 days, at any point during treatment, Eltrombopag must be discontinued. The eltrombopag used in this study will be purchased from Nano Darou Pharmaceutical Company.
Category
Treatment - Drugs
2
Description
Historical control patients will include patients with acute leukemia who received a haploidentical transplant and did not receive any medication to promote engraftment.
Category
N/A
Recruitment centers
1
Recruitment center
Name of recruitment center
Cell Therapy and Hematopoietic Stem Cell Transplantation Research Center
Full name of responsible person
Tahereh Rostami
Street address
Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital,North Kargar Street, Jalal Al-Ahmad Intersection, Tehran
City
Tehran
Province
Tehran
Postal code
1411713135
Phone
+98 21 8490 3691
Email
tah.rostami94@gmail.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Ramin Kordi
Street address
Office of the Vice-Chancellor for Research and Technology, Central Administration Complex, Keshavarz Boulevard, Corner of Ghods Street, Tehran
City
Tehran
Province
Tehran
Postal code
1417653837
Phone
+98 21 8163 3698
Email
vcr@tums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Tehran University of Medical Sciences
Proportion provided by this source
100
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic
Person responsible for general inquiries
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Tahereh Rostami
Position
Assistant Professor
Latest degree
Subspecialist
Other areas of specialty/work
Hematology
Street address
Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, North Kargar Street, Jalal Al-Ahmad Intersection, Tehran
City
Tehran
Province
Tehran
Postal code
1411713135
Phone
+98 21 8490 2635
Email
tah.rostami94@gmail.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Tahereh Rostami
Position
Assistant Professor
Latest degree
Subspecialist
Other areas of specialty/work
Hematology
Street address
Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, North Kargar Street, Jalal Al-Ahmad Intersection, Tehran
City
Tehran
Province
Tehran
Postal code
1411713135
Phone
+98 21 8490 2635
Email
tah.rostami94@gmail.com
Person responsible for updating data
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Ghazal Razani
Position
Research Assistant
Latest degree
Medical doctor
Other areas of specialty/work
General Practitioner
Street address
Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, North Kargar Street, Jalal Al-Ahmad Intersection, Tehran
City
Tehran
Province
Tehran
Postal code
1411713135
Phone
+98 21 8490 2635
Email
mohseni.vahideh@yahoo.com
Sharing plan
Deidentified Individual Participant Data Set (IPD)
No - There is not a plan to make this available
Justification/reason for indecision/not sharing IPD