Primary Immune ThrombocytopeniaNCT04949009
Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP)
An observational study of Primary Immune Thrombocytopenia, run by Shandong University at 1 site in China.
Trial summary
The project was undertaking by Qilu Hospital of Shandong University and other well-known hospitals in China. Aims at evaluating effectiveness and safety of avatrombopag in the treatment of primary immune thrombocytopenia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Immune Thrombocytopenia
- What is being tested?
- Avatrombopag
- Type of treatment
- Pill or oral medicine
- How many people
- 400 participants
- Where
- 1 siteChina
- Study dates
- 1 Mar 2021to Mar 2022
- Phase
- Observational
- 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
Study population: 1. Aged 18+ years, male or female; 2. Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)"
Who may be able to join
- Aged 18+ years, male or female;
- Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)"
- At least 2 consecutive blood tests showed a decrease in platelet count; no obvious abnormality in the morphology of blood cells by peripheral blood smear microscopy.
- Spleen is generally not enlarged.
- Bone marrow examination: the morphological characteristics of bone marrow cells in ITP patients were increased or normal megakaryocytes with maturation disorders.
- Other secondary thrombocytopenia must be excluded: autoimmune diseases, thyroid disease, lymphoproliferative disorders, myelodysplastic syndrome (MDS), aplastic anemia (AA), various malignant hematologic diseases, tumor infiltration, Chronic liver disease, hypersplenism, common variant immunodeficiency disease (CVID), infection, vaccination causing secondary thrombocytopenia; Thrombocytopenia due to depletion; Drug induced thrombocytopenia; Alloimmune thrombocytopenia; Thrombocytopenia during pregnancy; Congenital thrombocytopenia and pseudo-thrombocytopenia;
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- ECOG general status score ≤ 2;
- Platelet count < 30×10^9/L；platelet count ≥< 30×10^9/L accompanied by active bleeding; If the platelet count is around 30×10^9/L and no active bleeding, a second examination must be performed to further confirm the platelet count.
- Voluntarily signed the informed consent.
- Any other circumstances that the investigator considers appropriate for the patient to participate in the study.
Who cannot join
- Patients with secondary thrombocytopenia.
- Currently receiving other TPO-RAs, rhIL-11 and rhTPO treatment, and subjects are unwilling to switch to avatrombopag treatment.
- Patients with severe insufficiency of heart, lung, liver and kidney.
- Pregnant or breast-feeding, or contraceptive measures cannot be taken during the trial.
- Subjects participated in clinical studies of other investigational drugs or devices within 30 days prior to screening.
- Having a history of psychotropic drug abuse and unable to quit or having mental disorders.
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- Having significant factors that affect the absorption of oral drugs, such as inability to swallow, chronic diarrhea and intestinal obstruction, etc.
- Subject is allergic to avatrombopag or any of its excipients;
- Any other circumstances that the investigator considers inappropriate for the patient to participate in the study.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Qilu Hospital, Shandong University
Jinan, Shandong
Ming Hou, Dr, +86-531-82169114 ext. 9879, houming@medmail.com.cn
About this study
Official title: Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP): a Single-arm, Multi-centre, Observational Study
The project was undertaking by Qilu Hospital of Shandong University and other well-known hospitals in China. Aims at evaluating effectiveness and safety of avatrombopag in the treatment of primary immune thrombocytopenia.
Read the detailed description
The investigators are undertakingSingle-arm, multi-Centre, Observational study of 400 Patients with primary immune thrombocytopenia (ITP) from Qilu Hospital of Shandong University and other well-known hospitals in China. ITP patients will be given one tablet (20 mg) of avatrombopag daily.If the platelet count is higher than 150×10^9/L, the dose should be reduced. Prolonged dosing intervals or a combination of reduced daily dose are preferred.If the drug was taken for ≥1 week and the platelet count was still less than 30×10^9/L, the dosage should be increased. The maximum dose is 40 mg daily. Aplatelet count,AE, laboratory parameters, ECG, vital signs and other symptoms will be evaluated before and after treatment. Adverse events will be recorded throughout the study.
- Conditions
- Primary Immune Thrombocytopenia
- Being tested
- Avatrombopag
- Keywords
- Avatrombopag,Primary Immune Thrombocytopenia
- 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
Also called Doptelet
Avatrombopag is an orally bioavailable, small molecule thrombopoietin receptor agonist that has been developed by Dova Pharmaceuticals for the treatment of thrombocytopenic disorders.
| Study group | Role | What they receive |
|---|---|---|
| Patients with primary immune thrombocytopenia (ITP) | Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)" Receives: Drug: Avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of participants achieving platelet response on day 28 of treatment
Percentage of participants achieving platelet response on day 28 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy.
Measured: on day 28 of treatment
Secondary outcomes (4)
Percentage of participants achieving platelet response on day 8 of treatment.
Percentage of participants achieving platelet response on day 8 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy.
Measured: on day 8 of treatment.
Percentage of participants achieving platelet response on day 14 of treatment.
Percentage of participants achieving platelet response on day 14 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy.
Measured: on day 14 of treatment.
Changes in participants' bleeding scores.
Thrombocytopenia bleeding scoring system is used to evaluate the changes in participants' bleeding scores.
Measured: through study completion, an average of 6 months
Evaluation of adverse effects related to avatrombopag.
Evaluation of adverse effects related to avatrombopag including side effect,toxic reaction,post effect,anaphylactic reaction.
Measured: through study completion, an average of 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2021
- Study start1 Mar 2021
- Primary completion1 Oct 2021 (estimated)
- Study completionMar 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Primary Immune 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shandong University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ming Hou, Shandong University
- Investigators
- Ming Hou, MD PhD, Principal investigator, Shandong University Qilu Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04949009
- Sponsor's protocol ID
- SKX-2007
- Status last verified
- Jun 2021
- First submitted
- 13 May 2021
- Last updated
- 2 Jul 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Dlugosz-Danecka M, Zdziarska J, Jurczak W. Avatrombopag for the treatment of immune thrombocytopenia. Expert Rev Clin Immunol. 2019 Apr;15(4):327-339. doi: 10.1080/1744666X.2019.1587294. Epub 2019 Mar 8.
PubMed 30799645Shirley M. Avatrombopag: First Global Approval. Drugs. 2018 Jul;78(11):1163-1168. doi: 10.1007/s40265-018-0949-8.
PubMed 29995177Cheloff AZ, Al-Samkari H. Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease. J Blood Med. 2019 Sep 5;10:313-321. doi: 10.2147/JBM.S191790. eCollection 2019.
PubMed 31565009Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. doi: 10.1182/blood-2013-07-514398. Epub 2014 May 6.
PubMed 24802775
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP): a Single-arm, Multi-centre, Observational Study
- ClinicalTrials.gov ID
- NCT04949009
- Acronym / sponsor ID
- SKX-2007
- Conditions
- Primary Immune Thrombocytopenia
- Keywords
- Avatrombopag,Primary Immune Thrombocytopenia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- Shandong UniversityUniversity, hospital or other
- Study start
- 1 Mar 2021 (actual)
- Primary completion
- 1 Oct 2021 (estimated)
- Study completion
- Mar 2022 (estimated)
- First posted
- 2 Jul 2021 (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.
The project was undertaking by Qilu Hospital of Shandong University and other well-known hospitals in China. Aims at evaluating effectiveness and safety of avatrombopag in the treatment of primary immune thrombocytopenia.
Detailed description
The investigators are undertakingSingle-arm, multi-Centre, Observational study of 400 Patients with primary immune thrombocytopenia (ITP) from Qilu Hospital of Shandong University and other well-known hospitals in China. ITP patients will be given one tablet (20 mg) of avatrombopag daily.If the platelet count is higher than 150×10^9/L, the dose should be reduced. Prolonged dosing intervals or a combination of reduced daily dose are preferred.If the drug was taken for ≥1 week and the platelet count was still less than 30×10^9/L, the dosage should be increased. The maximum dose is 40 mg daily. Aplatelet count,AE, laboratory parameters, ECG, vital signs and other symptoms will be evaluated before and after treatment. Adverse events will be recorded throughout the study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with primary immune thrombocytopenia (ITP) | – | Drug: AvatrombopagEver been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)" |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 1. Aged 18+ years, male or female; 2. Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)"
Key inclusion criteria (10 listed)
- Aged 18+ years, male or female;
- Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)"
- ECOG general status score ≤ 2;
- Platelet count < 30×10^9/L；platelet count ≥< 30×10^9/L accompanied by active bleeding; If the platelet count is around 30×10^9/L and no active bleeding, a second examination must be performed to further confirm the platelet count.
- Voluntarily signed the informed consent.
Key exclusion criteria (9 listed)
- Patients with secondary thrombocytopenia.
- Currently receiving other TPO-RAs, rhIL-11 and rhTPO treatment, and subjects are unwilling to switch to avatrombopag treatment.
- Patients with severe insufficiency of heart, lung, liver and kidney.
- Pregnant or breast-feeding, or contraceptive measures cannot be taken during the trial.
- Subjects participated in clinical studies of other investigational drugs or devices within 30 days prior to screening.
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: DopteletAvatrombopag is an orally bioavailable, small molecule thrombopoietin receptor agonist that has been developed by Dova Pharmaceuticals for the treatment of thrombocytopenic disorders. | Drug | Patients with primary immune thrombocytopenia (ITP) |
Primary outcome
Percentage of participants achieving platelet response on day 28 of treatment
Time frame: on day 28 of treatment
Percentage of participants achieving platelet response on day 28 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy.
4 secondary outcomes
Percentage of participants achieving platelet response on day 8 of treatment.
Time frame: on day 8 of treatment.
Percentage of participants achieving platelet response on day 14 of treatment.
Time frame: on day 14 of treatment.
Changes in participants' bleeding scores.
Time frame: through study completion, an average of 6 months
Evaluation of adverse effects related to avatrombopag.
Time frame: through study completion, an average of 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2021.
- Background: PubMed 30799645Dlugosz-Danecka M, Zdziarska J, Jurczak W. Avatrombopag for the treatment of immune thrombocytopenia. Expert Rev Clin Immunol. 2019 Apr;15(4):327-339. doi: 10.1080/1744666X.2019.1587294. Epub 2019 Mar 8.
- Background: PubMed 29995177Shirley M. Avatrombopag: First Global Approval. Drugs. 2018 Jul;78(11):1163-1168. doi: 10.1007/s40265-018-0949-8.
- Background: PubMed 31565009Cheloff AZ, Al-Samkari H. Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease. J Blood Med. 2019 Sep 5;10:313-321. doi: 10.2147/JBM.S191790. eCollection 2019.
- Background: PubMed 24802775Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. doi: 10.1182/blood-2013-07-514398. Epub 2014 May 6.
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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.
Ming Hou, MD PhD principal investigator
Shandong University Qilu Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immune Thrombocytopenia, run by Shandong University 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 10 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 2 Jul 2021. 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.
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 Ming Hou, MD PhD (86-531-82169114 ext. 9879, houming@medmail.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of participants achieving platelet response on day 28 of treatment (time frame: on day 28 of treatment). 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 NCT04949009, which also lists 4 linked publications.
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 2 Jul 2021.