Eltrombopag for Enhancing Platelet Engraftment in Pediatric Patients Undergoing Cord Blood Transplantation

NCT ID: NCT01940562

Last Updated: 2015-06-16

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-10-31
• Study Completion Date: 2016-06-30

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.

Conditions

Pediatric Patients Undergoing Allogeneic Cord Blood Transplantation.

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Eltrombopag

Pediatric patients undergoing allogeneic UCB transplantation will receive eltrombopag from day +1 until platelet count has exceeded 50,000/microliter for 14 consecutive days without platelet transfusion.

Starting doses are 100 mg/d for children >40 kg body weight (BW), 50 mg/d for children 20-40 kg BW, and 2 mg/kg/d for children <20 kg BW.

If unsupported platelet count has not reached the threshold of 20,000/microliter, doses will be escalated every 2 weeks up to maximal doses of 200 mg/d for children ≥ 40 kg BW, 150 mg/d for children 20-40 kg BW and 3.5 mg/kg for children <20 kg BW.

Group Type: EXPERIMENTAL

eltrombopag

Intervention Type: DRUG

Children ≥= 40 kg BW:

From day +1, start eltrombopag 100 mg/d. If primary end point not reached on day +14, then from day +15 - 150 mg/d. If primary end point not reached on day +28 then from day +29 - 200 mg/d (maximal dose).

Children 20-40 kg BW:

From day +1, start eltrombopag 50 mg/d. If primary end point not reached on day +14, then from day +15 - 75 mg/d. If primary end point not reached on day +28 then from day +29 - 100 mg/d.If primary end point not reached on day +42, then from day +43 and on - 150 mg/d (maximal dose).

Children < 20 kg BW:

From day +1, start eltrombopag - 2 mg/kg/d. If primary end point not reached on day +14, then from day +15 - 3 mg/kg/d. If primary end point not reached on day +28 then from day +29 - 3.5 mg/kg (maximal dose).

If present dose not tolerated, return to last tolerated dose. Eltrombopag will be discontinued after platelet count has exceeded 50,000/microliter for 14 consecutive days without administration of platelets.

Interventions

eltrombopag

Children ≥= 40 kg BW:

From day +1, start eltrombopag 100 mg/d. If primary end point not reached on day +14, then from day +15 - 150 mg/d. If primary end point not reached on day +28 then from day +29 - 200 mg/d (maximal dose).

Children 20-40 kg BW:

From day +1, start eltrombopag 50 mg/d. If primary end point not reached on day +14, then from day +15 - 75 mg/d. If primary end point not reached on day +28 then from day +29 - 100 mg/d.If primary end point not reached on day +42, then from day +43 and on - 150 mg/d (maximal dose).

Children < 20 kg BW:

From day +1, start eltrombopag - 2 mg/kg/d. If primary end point not reached on day +14, then from day +15 - 3 mg/kg/d. If primary end point not reached on day +28 then from day +29 - 3.5 mg/kg (maximal dose).

If present dose not tolerated, return to last tolerated dose. Eltrombopag will be discontinued after platelet count has exceeded 50,000/microliter for 14 consecutive days without administration of platelets.

Intervention Type: DRUG

Other Intervention Names

Revolade

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).

-

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

GlaxoSmithKline

INDUSTRY

Sponsor Role: collaborator

moshe yeshurun

OTHER

Sponsor Role: lead

Responsible Party

moshe yeshurun

Head, BMT unit, Rabin Medical Center

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Moshe Yeshurun, MD

Role: PRINCIPAL_INVESTIGATOR

Rabin Medical Center

Locations

Hadassah hospital

Jerusalem, , Israel

Site Status: RECRUITING

Schneider Children's Medical Center of Israel

Petah Tikva, , Israel

Site Status: RECRUITING

Countries

Israel

Central Contacts

Moshe Yeshurun, MD

Role: CONTACT

moshey@clalit.org.il

972-3-9378127

Ron Ram, MD

Role: CONTACT

RonRa@clalit.org.il

972-3-9378116

Facility Contacts

Michael Shapira, MD

Role: primary

972-50-7874433

Jerry Stein, MD

Role: primary

jstein@clalit.org.il

972-50-4057148

Other Identifiers

0195-13

Identifier Type: -

Identifier Source: org_study_id