ThrombocytopeniaPhase 1NCT00903929
Phase I Study of Eltrombopag for Promoting Thrombopoiesis After Total Body Irradiation
A Phase 1 study testing Eltrombopag in people with Thrombocytopenia and Acute Radiation Syndrome, run by Jane Liesveld.
Trial summary
Patients who undergo total body irradiation (TBI) for stem cell transplantation have prolonged periods of low counts of specific blood cells called platelets. These low platelets counts can cause bleeding and infection. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopenia, Acute Radiation Syndrome
- What is being tested?
- Eltrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- No sites listed yet
- Study dates
- Dec 2009to Dec 2015
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Age ≥ 18
- Able to give written informed consent for a clinical trial
- Scheduled to undergo an autologous or an allogeneic stem cell transplantation from a sibling, related donor, or unrelated donor using a conditioning regimen containing at least 400 cGy TBI
- Transplantation is being performed for one of the following medical conditions:
- Acute myelogenous leukemia
- Acute lymphoblastic leukemia
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- Chronic myelogenous leukemia in chronic, accelerated, or blastic phase
- Myelodysplastic syndrome
- Myeloproliferative diseases such as chronic myelomonocytic leukemia, agnogeneic myeloid metaplasia with myelofibrosis, polycythemia vera, or essential thrombocythemia
- Hodgkin's lymphoma
- Non-Hodgkin's lymphoma
- Multiple myeloma
- Chronic lymphocytic leukemia
- Other malignancies or marrow disease such as aplastic anemia where transplant would be appropriate with approval of principal investigator
- Patients with therapy-related AML or MDS may be included if their prior malignancy has been in remission for at least 12 months. If the remission is less than 12 months, approval of the principal investigator is required. Entry could be allowed if the malignancy is controlled and not expected to relapse e.g. localized prostate cancer treated with XRT.
- Patients must meet all other pre-transplantation criteria of the transplant center including acceptable tests of heart, liver, kidney, and lung function (Standard screening for HSCT per PI, and co-investigators).
- Karnofsky performance status must be ≥70%.
Who cannot join
- TBI dose less than 400 cGY
- Cord blood transplantation
- HIV infection
- Pregnancy or breastfeeding
- Creatinine or bilirubin or ALT or AST greater than two times the upper limit of normal, unless the abnormal bilirubin is due to Gilbert's syndrome
- Active infection requiring systemic antibiotic therapy with antibacterial, antifungal, or antiviral agents
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- Concomitant enrollment in another therapeutic clinical study except with PI approval
- Must not have previously received eltrombopag
- Patients with moderate or severe liver disease (ALT, AST, or bilirubin ≥ 2X the upper limit of normal, unless the abnormal bilirubin is due to Gilbert's syndrome) will be excluded
- Patients with high risk of thromboembolism based on genetic syndromes , or past thromboembolic disease in the past 6 months will be excluded from the study, with the exception of those with catheter related clots
About this study
Official title: Phase I Study of Eltrombopag for Promoting Thrombopoiesis in Patients Undergoing Stem Cell Transplantation After Total Body Irradiation
Patients who undergo total body irradiation (TBI) for stem cell transplantation have prolonged periods of low counts of specific blood cells called platelets. These low platelets counts can cause bleeding and infection. Thus far, no drug is available for use to speed the recovery of platelets, and therefore transfusions are often necessary.
The purpose of this study is to test the safety of a drug called eltrombopag in patients who have received TBI. The investigators want to find out what effects, good or bad, it has on people with low platelet counts due to treatment with TBI. The investigators will also be testing how well eltrombopag may work at different doses and determine if this drug speeds up the recovery of the platelets.
- Conditions
- Thrombocytopenia, Acute Radiation Syndrome
- Being tested
- Eltrombopag
- Medical subject headings
- Thrombocytopenia, Acute Radiation Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
dose escalation
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Not described Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD) of Eltrombopag
The MTD was defined as the highest dose if no dose limiting toxicity was observed, or the highest dose at which less than one-third of the patients experienced toxicities not expected in the standard stem cell transplantation setting.
Measured: 1.5 years
Secondary outcomes (2)
Median Time to Platelet Engraftment
Determined for all participants who completed at least 75% of the planned doses. Platelet engraftment was as defined by the Center for International Blood and Marrow Transplant Research as the first of 3 consecutive days of a platelet count 20,000/mL without platelet trans-fusions for 7 days and/or the first day of a platelet count 100,000/mL without platelet transfusions for 7 days.
Measured: 1.5 years
Median Number of Platelet Transfusions up to the Day of Engraftment
Measured: baseline to day of engraftment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2009 (estimated)
- Study startDec 2009
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ThrombocytopeniaStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jane Liesveld
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Jane Liesveld, University of Rochester
- Investigators
- Yuhchyau Chen, MD,PhD, Study director, University of RochesterJane Liesveld, MD, Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00903929
- Sponsor's protocol ID
- RSRB00028043
- Status last verified
- Apr 2016
- First submitted
- 15 May 2009
- Last updated
- 5 May 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Liesveld JL, Phillips GL 2nd, Becker M, Constine LS, Friedberg J, Andolina JR, Milner LA, DeBolt J, Smudzin T, Hyrien O, Erickson-Miller CL, Johnson BM, Dawson KL, Chen Y. A phase 1 trial of eltrombopag in patients undergoing stem cell transplantation after total body irradiation. Biol Blood Marrow Transplant. 2013 Dec;19(12):1745-52. doi: 10.1016/j.bbmt.2013.10.002. Epub 2013 Oct 8.
PubMed 24120380
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Eltrombopag for Promoting Thrombopoiesis in Patients Undergoing Stem Cell Transplantation After Total Body Irradiation
- ClinicalTrials.gov ID
- NCT00903929
- Acronym / sponsor ID
- RSRB00028043
- Conditions
- Thrombocytopenia, Acute Radiation Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Jane LiesveldUniversity, hospital or other
- Study start
- Dec 2009
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 19 May 2009 (estimated)
- Results
- Posted 5 May 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients who undergo total body irradiation (TBI) for stem cell transplantation have prolonged periods of low counts of specific blood cells called platelets. These low platelets counts can cause bleeding and infection. Thus far, no drug is available for use to speed the recovery of platelets, and therefore transfusions are often necessary.
The purpose of this study is to test the safety of a drug called eltrombopag in patients who have received TBI. The investigators want to find out what effects, good or bad, it has on people with low platelet counts due to treatment with TBI. The investigators will also be testing how well eltrombopag may work at different doses and determine if this drug speeds up the recovery of the platelets.
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: Eltrombopag |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Age ≥ 18
- Able to give written informed consent for a clinical trial
- Scheduled to undergo an autologous or an allogeneic stem cell transplantation from a sibling, related donor, or unrelated donor using a conditioning regimen containing at least 400 cGy TBI
- Transplantation is being performed for one of the following medical conditions:
- Patients with therapy-related AML or MDS may be included if their prior malignancy has been in remission for at least 12 months. If the remission is less than 12 months, approval of the principal investigator is required. Entry could be allowed if the malignancy is controlled and not expected to relapse e.g. localized prostate cancer treated with XRT.
Key exclusion criteria (10 listed)
- TBI dose less than 400 cGY
- Cord blood transplantation
- HIV infection
- Pregnancy or breastfeeding
- Creatinine or bilirubin or ALT or AST greater than two times the upper limit of normal, unless the abnormal bilirubin is due to Gilbert's syndrome
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 |
|---|---|---|
| Eltrombopagdose escalation | Drug | Eltrombopag |
Primary outcome
Maximum Tolerated Dose (MTD) of Eltrombopag
Time frame: 1.5 years
The MTD was defined as the highest dose if no dose limiting toxicity was observed, or the highest dose at which less than one-third of the patients experienced toxicities not expected in the standard stem cell transplantation setting.
2 secondary outcomes
Median Time to Platelet Engraftment
Time frame: 1.5 years
Median Number of Platelet Transfusions up to the Day of Engraftment
Time frame: baseline to day of engraftment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 24120380Liesveld JL, Phillips GL 2nd, Becker M, Constine LS, Friedberg J, Andolina JR, Milner LA, DeBolt J, Smudzin T, Hyrien O, Erickson-Miller CL, Johnson BM, Dawson KL, Chen Y. A phase 1 trial of eltrombopag in patients undergoing stem cell transplantation after total body irradiation. Biol Blood Marrow Transplant. 2013 Dec;19(12):1745-52. doi: 10.1016/j.bbmt.2013.10.002. Epub 2013 Oct 8.
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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.
Yuhchyau Chen, MD,PhD study director
University of Rochester
Jane Liesveld, MD principal investigator
University of Rochester
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Eltrombopag in people with Thrombocytopenia and Acute Radiation Syndrome, run by Jane Liesveld.
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 17 inclusion and 10 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 5 May 2016. Status can change between updates, so confirm with the study contact before making plans.
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?
Yes. Results are posted in the registry (first posted 5 May 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 (MTD) of Eltrombopag (time frame: 1.5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00903929, which also lists 1 linked publication.
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 5 May 2016.