Clinical trial NCT01940562: Eltrombopag for Enhancing Platelet Engraftment in Pediatric Patients Undergoing Cord Blood Transplantation
Official title: Eltrombopag for Enhancing Platelet Engraftment in Pediatric Patients Undergoing Cord Blood Transplantation
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Pediatric Patients Undergoing Allogeneic Cord Blood Transplantation.
Keywords: cord blood transplantation
platelets
eltrombopag
Lead sponsor: Rabin Medical Center
Sponsor class: OTHER
Collaborators: name: GlaxoSmithKline
First posted: 2013-09-12
Enrollment: 20
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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).

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Primary outcomes:
measure: 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.
timeFrame: 50 days
Secondary outcomes:
measure: Time to partial platelet engraftment, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter.
timeFrame: 180 days
measure: 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.
timeFrame: 180 days
measure: 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.
timeFrame: 50 days
measure: Time to neutrophil engraftment defined as the first of 3 consecutive days to achieve an absolute neutrophil count ≥ 500/microliter.
timeFrame: 60 days
Other outcomes:
measure: Any grade 3/4 adverse event
timeFrame: 12 months
measure: Thromboembolic events
timeFrame: 12 months
Locations:
city: Jerusalem
country: Israel
facility: Hadassah hospital
city: Petah Tikva
country: Israel
facility: Schneider Children's Medical Center of Israel
Study officials:
affiliation: Rabin Medical Center
name: Moshe Yeshurun, MD
role: PRINCIPAL_INVESTIGATOR
References: