Clinical trial NCT02046291: Safety of Romiplostim (Nplate®) Following UCBT
Official title: Safety of Romiplostim (Nplate®) in Patients Who Have Failed to Achieve Platelet Engraftment Following Umbilical Cord Blood Transplant
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hematologic Malignancies
Keywords: umbilical cord transplant
acute leukemia
UCBT
Hodgkin's Lymphoma
Lymphoid Leukemia
Multiple Myeloma
Myeloid and Monocytic Leukemia
Non-Hodgkin's Lymphoma
Hematopoietic malignancies
Lead sponsor: Masonic Cancer Center, University of Minnesota
Sponsor class: OTHER
First posted: 2014-01-27
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: This is a single institution, phase I dose escalation study of weekly romiplostim post umbilical cord blood transplantation in patients who fail to achieve platelet engraftment by day +30. Engraftment is defined as a platelet count ≥ 20 x 109/L on 3 consecutive measurements without transfusion for 7 days. Romiplostim is administered at the assigned dose as 6 weekly injections beginning by day +42 post transplant. Up to 4 dose levels (4, 6, 8, and 10 mcg/kg/dose) will be evaluated with the maximum tolerated dose (MTD) of romiplostim determined by using the Continual Reassessment Method (CRM). The goal of this CRM will be to identify 1 of the 4 dose levels which corresponds to the desired maximum toxicity rate of 20% or less.


Eligibility criteria: Inclusion Criteria:

* Any patient who has undergone a single or double umbilical cord blood transplant (UCBT) except those with primary myelofibrosis.

  * Those with acute leukemia must be in remission at the time of transplant
* Must have achieved neutrophil engraftment (defined as an ANC \>500 for three consecutive days) and be off daily G-CSF prior to starting romiplostim. Intermittent G-CSF is allowed.
* Failure to achieve platelet engraftment (defined as platelet count ≥20x10\^9/L on 3 consecutive measurements without transfusion for 7 days) by day +28 post UCBT
* Between day +28 and day +42 status post myeloablative or nonmyeloablative UCBT (single or double cord blood transplant)
* Age ≥ 18 years
* Adequate organ function within 7 days of enrollment defined as:

  * Creatinine: ≤ 2.0 mg/dL
  * Hepatic: SGOT and SGPT \< 5 x upper limit of institutional normal (ULN)
* Women of child bearing potential agree to use effective contraception during therapy and for 4 months after completion of therapy
* Voluntary written consent

Exclusion Criteria:

* Known pregnancy - Pregnancy Category C: there are no adequate and well-controlled studies of romiplostim in pregnancy
* Recurrence of AML or myelodysplastic syndrome on bone marrow evaluation done within 21 days of enrollment
* Presence of clinically significant bone marrow fibrosis on the bone marrow examination immediately prior to UCBT
* Patients requiring more than one platelet transfusion per day
* History of an allergy to romiplostim

Primary outcomes:
measure: Maximum tolerated dose of romiplostim
timeFrame: Day +28 blood transplant (UCBT)
Secondary outcomes:
measure: Platelet recovery
timeFrame: Day +28
measure: Thrombocytopenia
timeFrame: Day +28
measure: Bone marrow fibrosis
timeFrame: Day 100 post transplant
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Cancer Center
state: Minnesota
Study officials:
affiliation: University of Minnesota
name: Margaret MacMillan, MD, MSc, FRCPC
role: PRINCIPAL_INVESTIGATOR
References: