Hematologic MalignanciesPhase 1NCT02046291
Safety of Romiplostim (Nplate®) Following UCBT
A Phase 1 study testing Romiplostim in people with Hematologic Malignancies, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- Romiplostim
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Apr 2015to 1 Jun 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 11Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
About this study
Official title: Safety of Romiplostim (Nplate®) in Patients Who Have Failed to Achieve Platelet Engraftment Following Umbilical Cord Blood Transplant
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.
- Conditions
- Hematologic Malignancies
- Being tested
- Romiplostim
- Keywords
- umbilical cord transplant, acute leukemia, UCBT, Hodgkin's Lymphoma, Lymphoid Leukemia, Multiple Myeloma, Myeloid and Monocytic Leukemia, Non-Hodgkin's Lymphoma, Hematopoietic malignancies
- Medical subject headings
- Hematologic Neoplasms, Hodgkin Disease, Leukemia, Lymphoid, Multiple Myeloma, Leukemia, Monocytic, Acute, Lymphoma, Non-Hodgkin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Also called Nplate
Romiplostim at the assigned dose will be administered subcutaneously (SQ) once a week for 6 weeks. The dose will be assigned at the time of subject registration. This is a dose escalation to determine the maximum tolerated dose. The doses include 4 mcg/kg/dose, 6 mcg/kg/dose, 8 mcg/kg/dose, and 10 mcg/kg/dose.
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim treatment | Experimental | Romiplostim at the assigned dose will be administered subcutaneously (SQ) once a week for 6 weeks (i.e. 6 doses) unless platelet count exceeds 100 x 10^9/L and at least 4 doses of therapy were given.After the initial dose, subsequent doses will be administered within a window of +/- 3 days. Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose of romiplostim
Measured by number of patients who have failed to achieve platelet engraftment by day +28 after a myeloablative or nonmyeloablative umbilical cord blood transplant (UCBT)
Measured: Day +28 blood transplant (UCBT)
Secondary outcomes (3)
Platelet recovery
Average speed of platelet recovery in patients who have failed to achieve platelet engraftment.
Measured: Day +28
Thrombocytopenia
Incidence of clinically significant bleeding episodes and number of platelet transfusions.
Measured: Day +28
Bone marrow fibrosis
Incidence of bone marrow fibrosis.
Measured: Day 100 post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2014 (estimated)
- Study start10 Apr 2015
- Primary completion12 Dec 2020
- Study completion1 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic MalignanciesStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Margaret MacMillan, MD, MSc, FRCPC, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02046291
- Sponsor's protocol ID
- 2012LS089
- Other IDs
- MT2012-17R
- Status last verified
- Jun 2021
- First submitted
- 23 Jan 2014
- Last updated
- 8 Jun 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety of Romiplostim (Nplate®) in Patients Who Have Failed to Achieve Platelet Engraftment Following Umbilical Cord Blood Transplant
- ClinicalTrials.gov ID
- NCT02046291
- Acronym / sponsor ID
- 2012LS089
- 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
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 10 Apr 2015 (actual)
- Primary completion
- 12 Dec 2020 (actual)
- Study completion
- 1 Jun 2021 (actual)
- First posted
- 27 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim treatment | Experimental | Drug: RomiplostimRomiplostim at the assigned dose will be administered subcutaneously (SQ) once a week for 6 weeks (i.e. 6 doses) unless platelet count exceeds 100 x 10^9/L and at least 4 doses of therapy were given.After the initial dose, subsequent doses will be administered within a window of +/- 3 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Any patient who has undergone a single or double umbilical cord blood transplant (UCBT) except those with primary myelofibrosis.
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RomiplostimAlso: NplateRomiplostim at the assigned dose will be administered subcutaneously (SQ) once a week for 6 weeks. The dose will be assigned at the time of subject registration. This is a dose escalation to determine the maximum tolerated dose. The doses include 4 mcg/kg/dose, 6 mcg/kg/dose, 8 mcg/kg/dose, and 10 mcg/kg/dose. | Drug | Romiplostim treatment |
Primary outcome
Maximum tolerated dose of romiplostim
Time frame: Day +28 blood transplant (UCBT)
Measured by number of patients who have failed to achieve platelet engraftment by day +28 after a myeloablative or nonmyeloablative umbilical cord blood transplant (UCBT)
3 secondary outcomes
Platelet recovery
Time frame: Day +28
Thrombocytopenia
Time frame: Day +28
Bone marrow fibrosis
Time frame: Day 100 post transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Margaret MacMillan, MD, MSc, FRCPC principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Romiplostim in people with Hematologic Malignancies, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose of romiplostim (time frame: Day +28 blood transplant (UCBT)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2021.