Pediatric Patients Undergoing Allogeneic Cord Blood Transplantation. Clinical Trial
Official title:
Eltrombopag for Enhancing Platelet Engraftment in Pediatric Patients Undergoing Cord Blood Transplantation
Platelet recovery is significantly hampered following umbilical cord blood transplantation
(UCB). Thus, after UCB transplantations, patients are platelet transfusion-dependent for
prolong periods of time.
Eltrombopag is a thrombopoietin-receptor agonist that initiates thrombopoietin-receptor
signaling and thereby induces proliferation and maturation of megakaryocytes.
We will evaluate the safety and efficacy of eltrombopag for enhancing platelet engraftment
in pediatric patients undergoing cord blood transplantation.
| Status | Recruiting |
| Enrollment | 20 |
| Est. completion date | June 2016 |
| Est. primary completion date | June 2016 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 1 Year to 18 Years |
| Eligibility |
Inclusion Criteria: 1. Patient = 1 year and < 18 years old. 2. Patients receiving unmanipulated single or double UCB allogeneic grafts. 3. Malignant and non malignant indications for transplantation. 4. Myeloablative and reduced intensity conditioning regimens. 5. Patients must meet all other pre-transplantation criteria of the transplantation center including acceptable tests of heart, liver, kidney, and lung function (standard screening for transplantation per PI, and co-investigators). 6. Written informed must be obtained from the patient's guardian, and accompanying informed assent from the patient for children over 6 years old. 7. Able to comply with study protocol. Exclusion Criteria: 1. Indications for transplantation 1. Patients with primary myelofibrosis. 2. French-American-British classification (FAB)M7 acute myeloid leukemia. Acute leukemia secondary to a myeloproliferative neoplasm. 3. Patients with persistent acute leukemia (>5% bone marrow blasts) at the time of transplantation. 2. Patients with prior thromboembolic event. Patients with previous catheter related thrombosis will be eligible if more than 3 months elapsed. 3. Hypersensitivity to eltrombopag. 4. Liver enzymes abnormalities: Alanine aminotransferase (ALT) levels > 3 times the upper limit of normal (ULN) or serum bilirubin > 1.5 ULN (unless due to Gilbert's syndrome or hemolytic bilirubin). |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Israel | Hadassah hospital | Jerusalem | |
| Israel | Schneider Children's Medical Center of Israel | Petah Tikva |
| Lead Sponsor | Collaborator |
|---|---|
| moshe yeshurun | GlaxoSmithKline |
Israel,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Any grade 3/4 adverse event | Number of participants with any grade 3/4 adverse event as a measure of safety and tolerability. | 12 months | Yes |
| Other | Thromboembolic events | Number of participants with a thromboembolic event as a measure of safety. | 12 months | Yes |
| Primary | Platelet engraftment rate up to 50 days post transplantation, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter. | 50 days | No | |
| Secondary | Time to partial platelet engraftment, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter. | 180 days | No | |
| Secondary | Time to sustained complete platelet engraftment defined as the first of 7 consecutive days of an unsupported platelet count of at least 50,000/microliter. | 180 days | No | |
| Secondary | Complete platelet engraftment rates up to 50 days post transplantation defined as the first of 7 consecutive days of an unsupported platelet count of at least 50,000/microliter. | 50 days | No | |
| Secondary | Time to neutrophil engraftment defined as the first of 3 consecutive days to achieve an absolute neutrophil count = 500/microliter. | 60 days | No |