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

NCT01940562 | Unknown Phase 2

• 1 other identifier: 0195-13
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 2

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.

Keywords

cord blood transplantation; platelets; eltrombopag

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (4)

• 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

• 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

• 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

• Time to neutrophil engraftment defined as the first of 3 consecutive days to achieve an absolute neutrophil count ≥ 500/microliter.

  60 days

Other Outcomes (2)

• Any grade 3/4 adverse event

  12 months

• Thromboembolic events

  12 months

Study Arms (1)

Eltrombopag

EXPERIMENTAL

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.

Drug: eltrombopag

Interventions

eltrombopag 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.

Also known as: Revolade

Eltrombopag

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patient ≥ 1 year and \< 18 years old.
• Patients receiving unmanipulated single or double UCB allogeneic grafts.
• Malignant and non malignant indications for transplantation.
• Myeloablative and reduced intensity conditioning regimens.
• 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).
• Written informed must be obtained from the patient's guardian, and accompanying informed assent from the patient for children over 6 years old.
• Able to comply with study protocol.

You may not qualify if:

• Indications for transplantation
• Patients with primary myelofibrosis.
• French-American-British classification (FAB)M7 acute myeloid leukemia. Acute leukemia secondary to a myeloproliferative neoplasm.
• Patients with persistent acute leukemia (\>5% bone marrow blasts) at the time of transplantation.
• Patients with prior thromboembolic event. Patients with previous catheter related thrombosis will be eligible if more than 3 months elapsed.
• Hypersensitivity to eltrombopag.
• 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).

Sponsors & Collaborators

• moshe yeshurun: lead
• GlaxoSmithKline: collaborator

Study Sites (2)

Hadassah hospital

Jerusalem, Israel

RECRUITING

Schneider Children's Medical Center of Israel

Petah Tikva, 49202, Israel

RECRUITING

MeSH Terms

Interventions

eltrombopag

Study Officials

• Moshe Yeshurun, MD

  Rabin Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Moshe Yeshurun, MD

CONTACT

972-3-9378127; moshey@clalit.org.il

Ron Ram, MD

CONTACT

972-3-9378116; RonRa@clalit.org.il

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Head, BMT unit, Rabin Medical Center

Study Record Dates

• First Submitted: September 8, 2013
• First Posted: September 12, 2013
• Study Start: October 1, 2013
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: June 16, 2015

Record last verified: 2015-06

Locations

IL (2)