Bone Marrow Transplantation RecipientPhase 2NCT01927731

Eltrombopag Olamine in Increasing Platelet Counts in Patients Undergoing Transplant

A Phase 2 study testing Eltrombopag Olamine and Laboratory Biomarker Analysis in people with Bone Marrow Transplantation Recipient and Cord Blood Transplant Recipient, run by M.D. Anderson Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This phase II trial studies how well eltrombopag olamine works in increasing platelet counts in patients undergoing transplant. Eltrombopag olamine may help platelet counts and the immune system recover from blood or bone marrow transplant. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Bone Marrow Transplantation Recipient, Cord Blood Transplant Recipient, Hematopoietic Cell Transplantation Recipient
What is being tested?: Eltrombopag Olamine and Laboratory Biomarker Analysis
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 48 participants
Where: 1 siteUnited States
Study dates: 14 Oct 2013to 6 Jun 2023
Phase: Phase 2
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

Patients undergoing a cord blood or haploidentical transplantation on any protocol or standard of care treatment plan.
Age >/= 18.
Females of child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization or breast-feeding must be willing to use an effective contraceptive measure until 30 days after the last dose of eltrombopag. Males who have had sexual contact with female of child-bearing potential must be willing to use contraceptive techniques until 30 days after the last dose of eltrombopag.
Patient or patient's legal representative(s) is/are able to provide written informed consent to participate.

Who cannot join

ALT and AST >/= 2.5 ULN.
Serum direct bilirubin >/= 1 mg/dl (except Gilbert's syndrome or hemolysis).
Patients of east Asian ancestry (Chinese, Japanese, or Korean Origin).
Calculated creatinine clearance < 30ml./min. Creatinine clearance will be calculated using the MDRD method.
Arterial or venous thrombosis in the last year except for line-related venous thrombosis more than 3 months ago.
Positive beta HCG within 7 days prior to consent in female of child-bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization or breast-feeding.

Trial locations and contacts

1 site in 1 country.

United States1 site

M D Anderson Cancer Center
Houston, Texas, 77030

Other trials in United States

About this study

Official title: Safety and Efficacy of Eltrombopag in Patients Undergoing Cord Blood or Haploidentical Bone Marrow Transplantation

Read the detailed description

PRIMARY OBJECTIVES:

To estimate the rate of platelet engraftment by day 60 in patients undergoing cord blood transplant (CBT) or haploidentical donor stem cell transplantation treated with eltrombopag (eltrombopag olamine).

SECONDARY OBJECTIVES:

To assess safety of eltrombopag in this population. II. To assess neutrophil engraftment with eltrombopag in this population. III. To characterize immune reconstitution. IV. To assess overall survival (OS). V. To assess progression free survival (PFS). VI. To assess incidence of acute graft-versus-host disease (GVHD).

OUTLINE: Patients are assigned to 1 of 2 arms.

ARM I (CORD BLOOD TRANSPLANT): Patients receive eltrombopag olamine orally (PO) once daily (QD) for 60 days beginning on day -1.

ARM II (HAPLOIDENTICAL DONOR STEM CELL TRANSPLANT): Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.

After completion of study treatment, patients are followed up at 1, 2, 3, 6, and 12 months.

Conditions: Bone Marrow Transplantation Recipient, Cord Blood Transplant Recipient, Hematopoietic Cell Transplantation Recipient
Being tested: Eltrombopag Olamine, Laboratory Biomarker Analysis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Eltrombopag OlamineDrug
Also called Promacta, SB-497115-GR
Given PO
Laboratory Biomarker AnalysisOther
Correlative studies

Study groups in NCT01927731
Study group	Role	What they receive
Arm I (cord blood transplant patients)	Experimental	Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1. Receives: Drug: Eltrombopag Olamine; Other: Laboratory Biomarker Analysis
Arm II (haploidentical donor stem cell transplant patients)	Experimental	Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5. Receives: Drug: Eltrombopag Olamine; Other: Laboratory Biomarker Analysis

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul
Number of participants that achieved platelet engraftment greater than 50K/ul 60 days post transplant.
Measured: Up to day 60

Secondary outcomes (2)

Overall Survival
Number of participants that survived at one year post transplant.
Measured: Up to 1 year
Progression Free Survival
Number of participants without disease progression at 1 year post transplant.
Measured: Up to 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

14 Oct 20136 Jun 2023

First posted23 Aug 2013 (estimated)
Study start14 Oct 2013
Primary completion6 Jun 2023
Study completion6 Jun 2023

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.
Other trials for Bone Marrow Transplantation RecipientStudies of the same condition, open and under way.
More trials run by M.D. Anderson Cancer CenterThe 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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 48 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01927731
Sponsor's protocol ID: 2012-0920
Other IDs: NCI-2013-02348, 2012-0920
Status last verified: Mar 2024
First submitted: 20 Aug 2013
Last updated: 23 Apr 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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CMV-MVA Triplex Vaccination of Stem Cell Donors in Preventing CMV Viremia in Participants With Allogeneic Transplant
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Active, not recruitingPhase 2
Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant
Acute Biphenotypic Leukemia, Acute Leukemia
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Cell Therapy for Immunomodulation in Kidney Transplantation
Kidney Transplantation
RecruitingObservational study
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All Bone Marrow Transplantation Recipient trials

Study at a glance

From the registry record, last updated by the sponsor on 23 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Safety and Efficacy of Eltrombopag in Patients Undergoing Cord Blood or Haploidentical Bone Marrow Transplantation
ClinicalTrials.gov ID: NCT01927731
Acronym / sponsor ID: 2012-0920
Conditions: Bone Marrow Transplantation Recipient, Cord Blood Transplant Recipient, Hematopoietic Cell Transplantation Recipient
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 48 participants (actual)
Lead sponsor: M.D. Anderson Cancer CenterUniversity, hospital or other
Collaborators: National Cancer Institute (NCI)
Study start: 14 Oct 2013 (actual)
Primary completion: 6 Jun 2023 (actual)
Study completion: 6 Jun 2023 (actual)
First posted: 23 Aug 2013 (estimated)
Results: Posted 23 Apr 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

PRIMARY OBJECTIVES:

To estimate the rate of platelet engraftment by day 60 in patients undergoing cord blood transplant (CBT) or haploidentical donor stem cell transplantation treated with eltrombopag (eltrombopag olamine).

SECONDARY OBJECTIVES:

To assess safety of eltrombopag in this population. II. To assess neutrophil engraftment with eltrombopag in this population. III. To characterize immune reconstitution. IV. To assess overall survival (OS). V. To assess progression free survival (PFS). VI. To assess incidence of acute graft-versus-host disease (GVHD).

OUTLINE: Patients are assigned to 1 of 2 arms.

ARM I (CORD BLOOD TRANSPLANT): Patients receive eltrombopag olamine orally (PO) once daily (QD) for 60 days beginning on day -1.

ARM II (HAPLOIDENTICAL DONOR STEM CELL TRANSPLANT): Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.

After completion of study treatment, patients are followed up at 1, 2, 3, 6, and 12 months.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Arm I (cord blood transplant patients)	Experimental	Drug: Eltrombopag Olamine, Other: Laboratory Biomarker AnalysisPatients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Arm II (haploidentical donor stem cell transplant patients)	Experimental	Drug: Eltrombopag Olamine, Other: Laboratory Biomarker AnalysisPatients receive eltrombopag olamine PO QD for 60 days beginning on day 5.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Patients undergoing a cord blood or haploidentical transplantation on any protocol or standard of care treatment plan.
Age >/= 18.
Females of child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization or breast-feeding must be willing to use an effective contraceptive measure until 30 days after the last dose of eltrombopag. Males who have had sexual contact with female of child-bearing potential must be willing to use contraceptive techniques until 30 days after the last dose of eltrombopag.
Patient or patient's legal representative(s) is/are able to provide written informed consent to participate.

Key exclusion criteria (6 listed)

ALT and AST >/= 2.5 ULN.
Serum direct bilirubin >/= 1 mg/dl (except Gilbert's syndrome or hemolysis).
Patients of east Asian ancestry (Chinese, Japanese, or Korean Origin).
Calculated creatinine clearance < 30ml./min. Creatinine clearance will be calculated using the MDRD method.
Arterial or venous thrombosis in the last year except for line-related venous thrombosis more than 3 months ago.

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
Eltrombopag OlamineAlso: Promacta, SB-497115-GRGiven PO	Drug	Arm I (cord blood transplant patients), Arm II (haploidentical donor stem cell transplant patients)
Laboratory Biomarker AnalysisCorrelative studies	Other	Arm I (cord blood transplant patients), Arm II (haploidentical donor stem cell transplant patients)

Primary outcome

Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul
Time frame: Up to day 60
Number of participants that achieved platelet engraftment greater than 50K/ul 60 days post transplant.

2 secondary outcomes

Overall Survival
Time frame: Up to 1 year
Progression Free Survival
Time frame: Up to 1 year

Results and safety

Results posted to the registry on 23 Apr 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Arm I (Cord Blood Transplant Patients)	6	3
Arm II (Haploidentical Donor Stem Cell Transplant Patients)	42	32

Primary outcome: Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul

Time frame: Up to day 60. Count of participants. Unit: Participants

Measure	Arm I (Cord Blood Transplant Patients) n = 6	Arm II (Haploidentical Donor Stem Cell Transplant Patients) n = 42
Value	3	30

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 1 year. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Arm I (Cord Blood Transplant Patients)	2 of 6 (33.3%)	2 of 6 (33.3%)	0 of 6
Arm II (Haploidentical Donor Stem Cell Transplant Patients)	2 of 42 (4.8%)	8 of 42 (19%)	3 of 42

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
MD Anderson Cancer Center WebsiteLink provided by the sponsor.

Related trials for Bone Marrow Transplantation Recipient

Recruiting
Mosaic Trial for Stem Cell Transplant Recipients
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Active, not recruitingPhase 1
CMV-MVA Triplex Vaccination of Stem Cell Donors in Preventing CMV Viremia in Participants With Allogeneic Transplant
Accelerated Phase CML, BCR-ABL1 Positive, Acute Lymphoblastic Leukemia in Remission
Active, not recruitingPhase 2
Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant
Acute Biphenotypic Leukemia, Acute Leukemia
Active, not recruitingPhase 1/2
Cell Therapy for Immunomodulation in Kidney Transplantation
Kidney Transplantation
RecruitingObservational study
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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.

Uday Popat principal investigator

M.D. Anderson Cancer Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 4 inclusion and 6 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 23 Apr 2024. 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 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?

Yes. Results are posted in the registry (first posted 23 Apr 2024). 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?

Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul (time frame: Up to day 60). 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 NCT01927731.

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 23 Apr 2024.