Platelet DisorderPhase 2NCT04312789
Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant
A Phase 2 study testing Avatrombopag in people with Platelet Disorder, run by Ayman Saad.
Trial summary
This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Platelet Disorder
- What is being tested?
- Avatrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Dec 2022to 31 Dec 2022
- 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
- Failure to engraft platelets by day 30 (D30) after hematopoietic cell transplantation (HCT) defined as: Platelet count less than 20,000/uL and patient is still dependent on platelet transfusion support
- Patient must be able to start treatment with avatrombopag within 30-60 days following transplant
- Able to provide written informed consent from patient or legal representative
Who cannot join
- Serious uncontrolled infections
- Steroid refractory graft versus host disease (GVHD)
- Patients with thrombotic microangiopathy
- Pregnant or lactating women
- Creatinine clearance < 30 ml/min
- Active thromboembolism requiring anticoagulation
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- Unable to understand the investigational nature of the study or provide informed consent
- Evidence of disease relapse by flow cytometry of chimerisms
- Concomitant use of other thrombopoietin receptor agonists (TPO-RA) medication during the treatment phase of the study or two weeks prior to enrollment
About this study
Official title: A Phase II Trial of Avatrombopag for the Treatment of Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplant
This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine the safety and efficacy of avatrombopag for the treatment of thrombocytopenia after allogenic hematopoietic stem cell transplantation.
SECONDARY OBJECTIVE:
- To identify predictors of response to avatrombopag.
OUTLINE:
Patients receive avatrombopag orally (PO) once daily (QD) for up to 1 year in the absence of disease progression or unacceptable toxicity. Avatrombopag will be titrated weekly until platelet count of greater than or equal to 60,000/uL is achieved and persists for 7 consecutive days, and the patient remains free from platelet transfusion.
After completion of study treatment, patients are followed up weekly for 4 weeks and then monthly up to 1 year.
- Conditions
- Platelet Disorder
- Being tested
- Avatrombopag
- Medical subject headings
- Blood Platelet Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvatrombopagDrug
Also called AKR-501, AS 1670542, Doptelet, E5501
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (avatrombopag) | Experimental | Patients receive avatrombopag PO QD for up to 1 year in the absence of disease progression or unacceptable toxicity. Avatrombopag will be titrated weekly until platelet count of greater than or equal to 60,000/uL is achieved and persists for 7 consecutive days, and the patient remains free from platelet transfusion. Receives: Drug: Avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events of avatrombopag treatment
Toxicities will be evaluated using the Common Terminology Criteria for Adverse Events (CTCAE) version (v).5 standard toxicity grading. Frequency and other descriptive statistics will be used to present the toxicity pattern.
Measured: Up to 30 days after the last dose
Failure rate of platelet recovery
The proportion will be provided with 95% exact binomial confidence interval.
Measured: At day 90
Secondary outcomes (8)
Independence from platelet transfusion
Measured: Up to 1 year
Duration of platelet response
Will be presented in a descriptive manner.
Measured: Up to 1 year
Platelet count >= 50,000/uL for 7 consecutive days without transfusion support
Measured: Up to 1 year
Duration of exposure to avatrombopag
Will be presented in a descriptive manner.
Measured: Up to 1 year
Incidence of adverse events associated with avatrombopag treatment
Measured: Up to 30 days after last dose
Transplant-related mortality
Measured: At day 100 and 1 year post-hematopoietic stem cell transplant (HCT)
Progression-free survival (PFS) of underlying malignant hematologic disorder
The method of Kaplan-Meier will be used to estimate PFS.
Measured: From the time of HCT to progression and death, assessed up to 1 year
Overall survival (OS)
The method of Kaplan-Meier will be used to estimate OS.
Measured: From the time of HCT to death from any cause, assessed 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.
- First posted18 Mar 2020
- Study startDec 2022 (estimated)
- Primary completion31 Dec 2022 (estimated)
- Study completion31 Dec 2022 (estimated)
Why it stopped: Drug availability
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Platelet DisorderStudies 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ayman Saad
- Sponsor type
- University, hospital or other
- Collaborators
- Sobi, Inc.
- Responsible party
- Sponsor-investigator, Ayman Saad, Ohio State University Comprehensive Cancer Center
- Investigators
- Ayman Saad, MB/BCH, Principal investigator, Ohio State University Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04312789
- Sponsor's protocol ID
- OSU-19328
- Other IDs
- NCI-2020-01035
- Status last verified
- Nov 2021
- First submitted
- 16 Mar 2020
- Last updated
- 9 Dec 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Avatrombopag for the Treatment of Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplant
- ClinicalTrials.gov ID
- NCT04312789
- Acronym / sponsor ID
- OSU-19328
- Conditions
- Platelet Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Drug availability
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Ayman SaadUniversity, hospital or other
- Collaborators
- Sobi, Inc.
- Study start
- Dec 2022 (estimated)
- Primary completion
- 31 Dec 2022 (estimated)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 18 Mar 2020 (actual)
- 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 phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
Detailed description
PRIMARY OBJECTIVE:
- To determine the safety and efficacy of avatrombopag for the treatment of thrombocytopenia after allogenic hematopoietic stem cell transplantation.
SECONDARY OBJECTIVE:
- To identify predictors of response to avatrombopag.
OUTLINE:
Patients receive avatrombopag orally (PO) once daily (QD) for up to 1 year in the absence of disease progression or unacceptable toxicity. Avatrombopag will be titrated weekly until platelet count of greater than or equal to 60,000/uL is achieved and persists for 7 consecutive days, and the patient remains free from platelet transfusion.
After completion of study treatment, patients are followed up weekly for 4 weeks and then monthly up to 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (avatrombopag) | Experimental | Drug: AvatrombopagPatients receive avatrombopag PO QD for up to 1 year in the absence of disease progression or unacceptable toxicity. Avatrombopag will be titrated weekly until platelet count of greater than or equal to 60,000/uL is achieved and persists for 7 consecutive days, and the patient remains free from platelet transfusion. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Failure to engraft platelets by day 30 (D30) after hematopoietic cell transplantation (HCT) defined as: Platelet count less than 20,000/uL and patient is still dependent on platelet transfusion support
- Patient must be able to start treatment with avatrombopag within 30-60 days following transplant
- Able to provide written informed consent from patient or legal representative
Key exclusion criteria (9 listed)
- Serious uncontrolled infections
- Steroid refractory graft versus host disease (GVHD)
- Patients with thrombotic microangiopathy
- Pregnant or lactating women
- Creatinine clearance < 30 ml/min
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 |
|---|---|---|
| AvatrombopagAlso: AKR-501, AS 1670542, Doptelet, E5501Given PO | Drug | Treatment (avatrombopag) |
Primary outcome
Incidence of adverse events of avatrombopag treatment
Time frame: Up to 30 days after the last dose
Toxicities will be evaluated using the Common Terminology Criteria for Adverse Events (CTCAE) version (v).5 standard toxicity grading. Frequency and other descriptive statistics will be used to present the toxicity pattern.
Failure rate of platelet recovery
Time frame: At day 90
The proportion will be provided with 95% exact binomial confidence interval.
8 secondary outcomes
Independence from platelet transfusion
Time frame: Up to 1 year
Duration of platelet response
Time frame: Up to 1 year
Platelet count >= 50,000/uL for 7 consecutive days without transfusion support
Time frame: Up to 1 year
Duration of exposure to avatrombopag
Time frame: Up to 1 year
Incidence of adverse events associated with avatrombopag treatment
Time frame: Up to 30 days after last dose
Transplant-related mortality
Time frame: At day 100 and 1 year post-hematopoietic stem cell transplant (HCT)
Progression-free survival (PFS) of underlying malignant hematologic disorder
Time frame: From the time of HCT to progression and death, assessed up to 1 year
Overall survival (OS)
Time frame: From the time of HCT to death from any cause, assessed up to 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2021.
- The JameslineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Ayman Saad, MB/BCH principal investigator
Ohio State University Comprehensive Cancer Center
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Avatrombopag in people with Platelet Disorder, run by Ayman Saad.
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 3 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: withdrawn, as of the sponsor's last update on 9 Dec 2021. 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 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Incidence of adverse events of avatrombopag treatment (time frame: Up to 30 days after the last dose). The study lists 2 primary outcomes in all. 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 9 Dec 2021.