Immune ThrombocytopeniaPhase 2NCT06281327
Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia
A Phase 2 study testing Avatrombopag in people with Immune Thrombocytopenia and Treatment, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and… Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Immune Thrombocytopenia, Treatment
- What is being tested?
- Avatrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 1 Mar 2024to Dec 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 6-18 years old (including both ends), male and female;
- Diagnosed with primary immune thrombocytopenia (ITP);
- Patients who had previously received eltrombopag treatment and then converted to avatrombopag treatment because of ineffectiveness (platelet count < 30×10^9/L after eltrombopag treatment, or platelet count increased less than 2 times of the basic value, or bleeding) or large platelet fluctuation or due to patient preference, economic reasons and other reasons;
- Cardiac function of the New York Society of Cardiac Function ≤ 2;
- Understand the study procedure and voluntarily sign the informed consent.
Who cannot join
- Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
- Subjects with primary disease of important organs (liver, kidney, heart, etc.), or with immune system diseases;
- Subjects who are known to be allergic to avatrombopag or any of its excipients;
- Subjects who had used rituximab within the last 3 months;
- Subjects who underwent splenectomy within the last 3 months；
- Subjects with a history of abnormal platelet aggregation that may affect the reliability of platelet count measurements；
- Any medical history or condition that the investigator deems unsuitable for participation in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin
Lei Zhang, MD, +8602223909009, zhanglei1@ihcams.ac.cn
Ting Sun, MD, +8602223909009, sunting@ihcams.ac.cn
About this study
Official title: A Prospective, One-arm and Open Clinical Study to Assess Efficacy and Safety of Avatrombopag in the Treatment of Pediatric Primary Immune Thrombocytopenia
To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.
Read the detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.).
Eltrombopag is currently the only TPO receptor agonist with indications for pediatric immune thrombocytopenia. However, at present, the treatment response of pediatric ITP is not good, and a considerable number of patients need to switched to other TPO receptor agonist, such as avatrombopag, because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.
Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of avatrombopag in the treatment of pediatric immune thrombocytopenia in patients who who have been treated with eltrombopag before and switched to avatrobopag because of different reasons.
- Conditions
- Immune Thrombocytopenia, Treatment
- Being tested
- Avatrombopag
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvatrombopagDrug
After enrollment, all subjects receive Avatrombopag treatment. The initial dose of Avatrombopag administration was an oral 10 mg (<30kg) or 20mg (≥30kg) once daily in all participants. ps. For subjects weighing ≥30kg, the dose can be started from 40mg once daily if the platelet count is < 10×10^9/L or if there is severe bleeding or risk of bleeding.
Complete blood count including platelet count was done once a week. The dose of Avatrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. If the platelet count was less than 30×10^9/L for 2 weeks, avatrobopag was gradually increased. If the platelet count was greater than 150 x 10^9/L, avatrobopag was gradually reduced. If the platelet count >250×10^9/L, avatrobopag will be stoped until the platelet count <100×10^9/L.
| Study group | Role | What they receive |
|---|---|---|
| Treatment group: Avatrombopag | Experimental | Sixty subjects will be enrolled with the indicated treatment dose of avatrombopag Receives: Drug: Avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall efficacy response after AVA treatment within 12 weeks
Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding at the meanwhile within 12 weeks after initial administration in absence of rescue therapy.
Measured: 12 weeks
Secondary outcomes (12)
Treatment response-1
Number of participants achieving a platelet count ≥ 50×10^9/L at week 1,2,4,6,8,12 in absence of rescue therapy.
Measured: 12 weeks
Treatment response-2
Number of participants achieving a platelet count ≥ 100×10^9/L at week 1,2,4,6,8,12 in absence of rescue therapy.
Measured: 12 weeks
Time to Response
The time required from the start of treatment to the first time a subject's platelet count was greater than or equal to 30×10^9/L and at least a two-fold increase from the baseline platelet count without bleeding in absence of rescue therapy.
Measured: 12 weeks
Persistent response
Persistent response defined as proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding for 4 consecutive weeks or more within 12 weeks after initial administration in absence of rescue therapy.
Measured: 12 weeks
Emergency treatment
The proportion of subjects receiving emergency treatment
Measured: 12 weeks
Reduction of concomitant drug
Percentage of patients with reduced doses of concomitant drugs at baseline
Measured: 12 weeks
Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Measured: 12 weeks
Number of participants with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP.
The bleeding scale for pediatric patients with ITP is a measure of bleeding severity with the following grades: Grade 1 (minor) Minor bleeding, few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm in diameter), no mucosal bleeding；Grade 2 (mild) Mild bleeding, many petechiae (>100 total) and/or >5 large bruises (>3 cm in diameter), no mucosal bleeding；Grade 3 (moderate) Moderate bleeding, overt mucosal bleeding, troublesome lifestyle；Grade 4 (severe) Severe bleeding, mucosal bleeding leading to decrease in Hb>2 g/dL or suspected internal hemorrhage；
Measured: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-1
In all participants ,use ITP-PAQ (ITP Patient Assessment Questionnaire) to assess the HRQoL before and after treatment.
Measured: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-2
In all participants ,use FACIT-F（functional assessment of chronic illness therapy- fatigue）to assess the HRQoL before and after treatment.
Measured: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-3
In all participants ,use Kids' ITP tool KIT、to assess the HRQoL before and after treatment.
Measured: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-4
In all participants ,use Pediatric Quality of Life Inventory PedsQL to assess the HRQoL before and after treatment.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2024
- Study start1 Mar 2024
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Henan Cancer Hospital, Tianjin Medical University Second Hospital, Tianjin Children's Hospital, The Second Affiliated Hospital of Kunming Medical University
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06281327
- Sponsor's protocol ID
- IIT2023062
- Status last verified
- Feb 2025
- First submitted
- 20 Feb 2024
- Last updated
- 24 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, One-arm and Open Clinical Study to Assess Efficacy and Safety of Avatrombopag in the Treatment of Pediatric Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06281327
- Acronym / sponsor ID
- IIT2023062
- Conditions
- Immune Thrombocytopenia, Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Henan Cancer Hospital, Tianjin Medical University Second Hospital, Tianjin Children's Hospital, The Second Affiliated Hospital of Kunming Medical University
- Study start
- 1 Mar 2024 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 28 Feb 2024 (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.
To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.
Detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.).
Eltrombopag is currently the only TPO receptor agonist with indications for pediatric immune thrombocytopenia. However, at present, the treatment response of pediatric ITP is not good, and a considerable number of patients need to switched to other TPO receptor agonist, such as avatrombopag, because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.
Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of avatrombopag in the treatment of pediatric immune thrombocytopenia in patients who who have been treated with eltrombopag before and switched to avatrobopag because of different reasons.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment group: Avatrombopag | Experimental | Drug: AvatrombopagSixty subjects will be enrolled with the indicated treatment dose of avatrombopag |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Age 6-18 years old (including both ends), male and female;
- Diagnosed with primary immune thrombocytopenia (ITP);
- Patients who had previously received eltrombopag treatment and then converted to avatrombopag treatment because of ineffectiveness (platelet count < 30×10^9/L after eltrombopag treatment, or platelet count increased less than 2 times of the basic value, or bleeding) or large platelet fluctuation or due to patient preference, economic reasons and other reasons;
- Cardiac function of the New York Society of Cardiac Function ≤ 2;
- Understand the study procedure and voluntarily sign the informed consent.
Key exclusion criteria (7 listed)
- Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
- Subjects with primary disease of important organs (liver, kidney, heart, etc.), or with immune system diseases;
- Subjects who are known to be allergic to avatrombopag or any of its excipients;
- Subjects who had used rituximab within the last 3 months;
- Subjects who underwent splenectomy within the last 3 months；
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 |
|---|---|---|
| AvatrombopagAfter enrollment, all subjects receive Avatrombopag treatment. The initial dose of Avatrombopag administration was an oral 10 mg (<30kg) or 20mg (≥30kg) once daily in all participants. ps. For subjects weighing ≥30kg, the dose can be started from 40mg once daily if the platelet count is < 10×10^9/L or if there is severe bleeding or risk of bleeding. Complete blood count including platelet count was done once a week. The dose of Avatrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. If the platelet count was less than 30×10^9/L for 2 weeks, avatrobopag was gradually increased. If the platelet count was greater than 150 x 10^9/L, avatrobopag was gradually reduced. If the platelet count >250×10^9/L, avatrobopag will be stoped until the platelet count <100×10^9/L. | Drug | Treatment group: Avatrombopag |
Primary outcome
Overall efficacy response after AVA treatment within 12 weeks
Time frame: 12 weeks
Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding at the meanwhile within 12 weeks after initial administration in absence of rescue therapy.
12 secondary outcomes
Treatment response-1
Time frame: 12 weeks
Treatment response-2
Time frame: 12 weeks
Time to Response
Time frame: 12 weeks
Persistent response
Time frame: 12 weeks
Emergency treatment
Time frame: 12 weeks
Reduction of concomitant drug
Time frame: 12 weeks
Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Time frame: 12 weeks
Number of participants with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP.
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-1
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-2
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-3
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-4
Time frame: 12 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2025.
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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.
Lei Zhang, MD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Avatrombopag in people with Immune Thrombocytopenia and Treatment, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 5 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 24 Feb 2025. 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 China. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lei Zhang, MD (+86 13502118379, zhanglei1@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall efficacy response after AVA treatment within 12 weeks (time frame: 12 weeks). 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 24 Feb 2025.