Hematological MalignancyPhase 2NCT01757145
Eltrombopag for Enhancing Platelet Engraftment in Adult Patients Undergoing Cord Blood Transplantation
A Phase 2 study testing Eltrombopag in people with Hematological Malignancy and Bone Marrow Failure Syndrome, run by Rabin Medical Center at 2 sites in Israel.
Trial summary
The purpose of this study is to determine whether Eltrombopag may shorten time to platelet engraftment after allogeneic cord blood transplantation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematological Malignancy, Bone Marrow Failure Syndrome
- What is being tested?
- Eltrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesIsrael
- Study dates
- Jan 2013to Jun 2016
- Phase
- Phase 2
- 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
- Patient ≥ 18 year old.
- Patients receiving unmanipulated single or double umbilical cord blood 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).
- Able to give written informed consent for a clinical trial.
- Able to comply with study protocol.
Who cannot join
- Indications for transplantation
- Patients with primary myelofibrosis.
- M7 (French-American-British classification) 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.
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- Liver enzymes abnormalities:
- Alanine transaminase (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).
- Pregnancy: Women of child-bearing potential and men must agree to use contraception prior to study entry and for the duration of study participation. A woman of child-bearing potential is defined as a woman who has not been naturally post-menopausal for at least 12 consecutive months or with no previous surgical sterilization.
Trial locations and contacts
2 sites in 1 country.
Israel2 sites, 2 recruiting
- Recruiting
Hadassah hospital
Jerusalem
Michael Shapira, MD, 972-50-7874433
- Recruiting
Davidof Cancer Center, Beilinson hospital, Rabin medical center
Petah Tikva
Moshe Yeshurun, MD, 972-50-4065543, moshey@clalit.org.il
Moshe Yeshurun, MD
About this study
The purpose of this study is to determine whether Eltrombopag may shorten time to platelet engraftment after allogeneic cord blood transplantation.
Read the detailed description
Platelet recovery is significantly hampered following umbilical cord blood (UCB) transplantation. Median time to platelet engraftment (defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter) in a large retrospective study was more than 70 days and cumulative incidence of platelet recovery at 6 months was 50.5%. In the pediatric population with acute leukemia median time to platelet engraftment with UCB transplantation was 59 days and cumulative incidence of platelet recovery at 6 months was 43%-73%, depending on human leukocyte antigen (HLA) disparity and cell dose. Recently, in a cohort of adult patients given myeloablative conditioning followed by double UCB transplantation, the cumulative incidence of platelet recovery (≥ 50,000/microliter)at 100 days was 53%. In another cohort of patients given reduced intensity conditioning regimen followed by single or double UCB transplantation, the median time to platelet recovery (≥ 50,000/microliter) and cumulative incidence of platelet recovery at 6 months were 49 days and 65%, respectively. Thus, after UCB transplantations, patients are platelet transfusion-dependent for prolong periods of time, resulting in many drawbacks, such as exposure to blood transfusions hazards, higher incidence of platelet allo-reactivity and extended periods of bleeding diathesis and undesirable costly and long hospitalizations.
Eltrombopag is a thrombopoietin-receptor agonist that initiates thrombopoietin-receptor signaling and thereby inducing proliferation and maturation of megakaryocytes.Administration of eltrombopag increased platelet production in volunteers with normal platelet counts, patients with thrombocytopenia secondary to hepatitis C virus infection and in patients with chronic immune thrombocytopenic purpura.
We will evaluate the safety and efficacy of eltrombopag treatment given early after UCB transplantation. The study is an open non-comparative study. The primary outcome will be cumulative incidence of partial platelet engraftment (first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter)at day 50 post transplantation. Secondary objectives are safety, tolerability and other transplantation related outcomes.
- Being tested
- Eltrombopag
- Keywords
- Cord blood transplantation, Eltrombopag, Platelet engraftment
- Medical subject headings
- Hematologic Neoplasms, Bone Marrow Failure Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called Revolade
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. If primary end point not reached on day +42 then from day +43 and on - 300 mg/d 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. In case of decline of platelet count < 30,000/microliter within 15 days from eltrombopag discontinuation, it will be resumed for additional 4 weeks. After 4 weeks we will re-attempt to hold the drug (if the threshold of platelet count >50,000/microliter will be reached again).
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Eltrombopag will be given orally as a single daily dose. From day +1 after cord blood transplantation, 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. If primary end point not reached on day +42 then from day +43 and on - 300 mg/d (maximal dose). If 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.
In case of decline of platelet count < 30,000/microliter within 15 days from eltrombopag discontinuation, it will be resumed for additional 4 weeks.
After 4 weeks we will re-attempt to hold the drug. Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 50 days
Time to neutrophil engraftment defined as the first of 3 consecutive days to achieve an absolute neutrophil count ≥ 500/microliter.
Measured: 60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2012 (estimated)
- Study startJan 2013
- Primary completionJun 2016 (estimated)
- Study completionJun 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematological MalignancyStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rabin Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Moshe Yeshurun, MD, Principal investigator, Rabin Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01757145
- Sponsor's protocol ID
- 7114
- Status last verified
- Feb 2013
- First submitted
- 6 Dec 2012
- Last updated
- 19 Jun 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Eltrombopag for Enhancing Platelet Engraftment in Adult Patients Undergoing Cord Blood Transplantation
- ClinicalTrials.gov ID
- NCT01757145
- Acronym / sponsor ID
- 7114
- Keywords
- Cord blood transplantation, Eltrombopag, Platelet engraftment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Rabin Medical CenterUniversity, hospital or other
- Study start
- Jan 2013
- Primary completion
- Jun 2016 (estimated)
- Study completion
- Jun 2016 (estimated)
- First posted
- 28 Dec 2012 (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.
The purpose of this study is to determine whether Eltrombopag may shorten time to platelet engraftment after allogeneic cord blood transplantation.
Detailed description
Platelet recovery is significantly hampered following umbilical cord blood (UCB) transplantation. Median time to platelet engraftment (defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter) in a large retrospective study was more than 70 days and cumulative incidence of platelet recovery at 6 months was 50.5%. In the pediatric population with acute leukemia median time to platelet engraftment with UCB transplantation was 59 days and cumulative incidence of platelet recovery at 6 months was 43%-73%, depending on human leukocyte antigen (HLA) disparity and cell dose. Recently, in a cohort of adult patients given myeloablative conditioning followed by double UCB transplantation, the cumulative incidence of platelet recovery (≥ 50,000/microliter)at 100 days was 53%. In another cohort of patients given reduced intensity conditioning regimen followed by single or double UCB transplantation, the median time to platelet recovery (≥ 50,000/microliter) and cumulative incidence of platelet recovery at 6 months were 49 days and 65%, respectively. Thus, after UCB transplantations, patients are platelet transfusion-dependent for prolong periods of time, resulting in many drawbacks, such as exposure to blood transfusions hazards, higher incidence of platelet allo-reactivity and extended periods of bleeding diathesis and undesirable costly and long hospitalizations.
Eltrombopag is a thrombopoietin-receptor agonist that initiates thrombopoietin-receptor signaling and thereby inducing proliferation and maturation of megakaryocytes.Administration of eltrombopag increased platelet production in volunteers with normal platelet counts, patients with thrombocytopenia secondary to hepatitis C virus infection and in patients with chronic immune thrombocytopenic purpura.
We will evaluate the safety and efficacy of eltrombopag treatment given early after UCB transplantation. The study is an open non-comparative study. The primary outcome will be cumulative incidence of partial platelet engraftment (first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter)at day 50 post transplantation. Secondary objectives are safety, tolerability and other transplantation related outcomes.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Experimental | Drug: EltrombopagEltrombopag will be given orally as a single daily dose. From day +1 after cord blood transplantation, 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. If primary end point not reached on day +42 then from day +43 and on - 300 mg/d (maximal dose). If 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. In case of decline of platelet count < 30,000/microliter within 15 days from eltrombopag discontinuation, it will be resumed for additional 4 weeks. After 4 weeks we will re-attempt to hold the drug. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patient ≥ 18 year old.
- Patients receiving unmanipulated single or double umbilical cord blood 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).
Key exclusion criteria (9 listed)
- Indications for 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 transaminase (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).
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 |
|---|---|---|
| EltrombopagAlso: RevoladeFrom 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. If primary end point not reached on day +42 then from day +43 and on - 300 mg/d 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. In case of decline of platelet count < 30,000/microliter within 15 days from eltrombopag discontinuation, it will be resumed for additional 4 weeks. After 4 weeks we will re-attempt to hold the drug (if the threshold of platelet count >50,000/microliter will be reached again). | Drug | Eltrombopag |
Primary outcome
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.
Time frame: 50 days
4 secondary outcomes
Time to partial platelet engraftment, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter.
Time frame: 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.
Time frame: 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.
Time frame: 50 days
Time to neutrophil engraftment defined as the first of 3 consecutive days to achieve an absolute neutrophil count ≥ 500/microliter.
Time frame: 60 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jun 2015.
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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.
Moshe Yeshurun, MD principal investigator
Rabin Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eltrombopag in people with Hematological Malignancy and Bone Marrow Failure Syndrome, run by Rabin Medical Center at 2 sites in Israel.
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 7 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 19 Jun 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Israel. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Moshe Yeshurun, MD (972-50-4065543, moshey@clalit.org.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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. (time frame: 50 days). 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 19 Jun 2015.