Immune ThrombocytopeniaADOPTNCT04943042
An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP
An observational study of Immune Thrombocytopenia and ITP, run by Swedish Orphan Biovitrum at 55 sites in 9 countries.
Trial summary
This is a multi-center, observational, Phase 4 study in patients with Immune Thrombocytopenia (ITP) designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia, ITP
- What is being tested?
- Avatrombopag
- How many people
- 199 participants
- Where
- 55 sitesin 9 countries
- Study dates
- 22 Feb 2022to 10 Jun 2025
- 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: The Investigators will attempt to consecutively enroll all eligible patients who present for a routine clinical visit or during a routine visit where the patient is started on Doptelet treatment (new users of Doptelet).
Who may be able to join
- Patient is ≥18 years of age
- Established and well documented ITP diagnosis
- Patient is treated with, or at enrollment prescribed, Doptelet for ITP. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study
- Signed and dated informed consent provided by the patient before any study-related activities are undertaken
- Willing and able to comply with protocol requirements
Who cannot join
- Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
- ITP secondary to Evan's syndrome, lupus and other autoimmune diseases
- ITP secondary to other hematological disorders and hematological malignancies
- ITP secondary to any other malignancies
- ITP secondary to known drug toxicity
- ITP secondary to any other disease considered relevant by the Investigator
Trial locations and contacts
55 sites in 9 countries.
55 sites in 9 countries
Germany14 sites
Swedish Orphan Biovitrum Research Site 108
Aschaffenburg
Swedish Orphan Biovitrum Research Site 114
Augsburg
Swedish Orphan Biovitrum Research Site 116
Augsburg
Swedish Orphan Biovitrum Research Site 103
Bad Homburg
Swedish Orphan Biovitrum Research Site 106
Berlin
Swedish Orphan Biovitrum Research Site 113
Dresden
Swedish Orphan Biovitrum Research Site 102
Frankfurt
Swedish Orphan Biovitrum Research Site 105
Frankfurt
Swedish Orphan Biovitrum Research Site 104
Hanover
Swedish Orphan Biovitrum Research Site 112
Kaiserslautern
Swedish Orphan Biovitrum Research Site 101
Kassel
Swedish Orphan Biovitrum Research Site 115
Leipzig
Swedish Orphan Biovitrum Research Site 109
Schorndorf
Swedish Orphan Biovitrum Research Site 111
Stolberg
Spain11 sites
Swedish Orphan Biovitrum Research Site 611
Barcelona
Swedish Orphan Biovitrum Research Site 609
Burgos
Swedish Orphan Biovitrum Research Site 610
Granada
Swedish Orphan Biovitrum Research Site 601
Madrid
Swedish Orphan Biovitrum Research Site 605
Madrid
Swedish Orphan Biovitrum Research Site 607
Murcia
Swedish Orphan Biovitrum Research Site 606
Oviedo
Swedish Orphan Biovitrum Research Site 608
Palma de Mallorca
Swedish Orphan Biovitrum Research Site 602
Salamanca
Swedish Orphan Biovitrum Research Site 604
Seville
Swedish Orphan Biovitrum Research Site 603
Vigo
United Kingdom9 sites
Swedish Orphan Biovitrum Research Site 701
Bristol
Swedish Orphan Biovitrum Research Site 709
Edinburgh, Scotland
Swedish Orphan Biovitrum Research Site 708
Glasgow, Scotland
Swedish Orphan Biovitrum Research Site 704
Leicester
Swedish Orphan Biovitrum Research Site 707
London
Swedish Orphan Biovitrum Research Site 710
London
Swedish Orphan Biovitrum Research Site 705
Newcastle
Swedish Orphan Biovitrum Research Site 702
Nottingham
Swedish Orphan Biovitrum Research Site 703
Oxford
Italy8 sites
Swedish Orphan Biovitrum Research Site 509
Bologna
Swedish Orphan Biovitrum Research Site 504
Catania
Swedish Orphan Biovitrum Research Site 502
Florence
Swedish Orphan Biovitrum Research Site 506
Meldola
Swedish Orphan Biovitrum Research Site 511
Milan
Swedish Orphan Biovitrum Research Site 510
Milan
Swedish Orphan Biovitrum Research Site 503
Roma
Swedish Orphan Biovitrum Research Site 505
Rome
Czechia5 sites
Swedish Orphan Biovitrum Research Site 122
Brno
Swedish Orphan Biovitrum Research Site 123
Ostrava
Swedish Orphan Biovitrum Research Site 121
Prague
Swedish Orphan Biovitrum Research Site 124
Prague
Swedish Orphan Biovitrum Research Site 125
Prague
Netherlands3 sites
Swedish Orphan Biovitrum Research Site 202
Apeldoorn, 7334 DZ
Swedish Orphan Biovitrum Research Site 204
Arnhem
Swedish Orphan Biovitrum Research Site 203
The Hague
Norway2 sites
Swedish Orphan Biovitrum Research Site 302
Bergen
Swedish Orphan Biovitrum Research Site 301
Oslo
Switzerland2 sites
Swedish Orphan Biovitrum Research Site 802
Basel
Swedish Orphan Biovitrum Research Site 801
Lausanne
About this study
Official title: An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® (Avatrombopag) in Adult Patients With Immune Thrombocytopenia (ITP)
This is a multi-center, observational, Phase 4 study in patients with Immune Thrombocytopenia (ITP) designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice. Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.
Read the detailed description
This is a multi-center, observational, Phase 4 study in patients with ITP designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice.
Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.
The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective collecting data on usage, effectiveness, safety, patient- and clinician-reported outcomes and health economic parameters whereas the retrospective part will consist of collection of information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Data will be collected for up to 12 months prior to Doptelet treatment start.
Prospective data will be collected at routine clinical visits throughout the study. Patients will be followed for 12 (+6) months and will be enrolled until their first scheduled visit after 12 months of enrollment, or until early termination, whichever occurs first.
- Conditions
- Immune Thrombocytopenia, ITP
- Being tested
- Avatrombopag
- Keywords
- TPO RA (thrombopoietin receptor agonists )
- 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®
According to prescription
| Study group | Role | What they receive |
|---|---|---|
| Full-analysis set (FAS) | The FAS includes all enrolled patients. The FAS will be used for all analyses. Receives: Drug: Avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative number of weeks with a platelet count ≥30×109/L during Doptelet treatment.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results. Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP.
Platelet counts during rescue medication use and within 4 weeks after stopping a rescue medication or following splenectomy will not be counted in the cumulative number of weeks.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Secondary outcomes (23)
Cumulative number of weeks with a platelet count ≥50×109/L during Doptelet treatment.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results. Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP.
Platelet counts during rescue medication use and within 4 weeks after stopping a rescue medication or following splenectomy will not be counted in the cumulative number of weeks.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients with a platelet count ≥30×109/L, for at least 8 consecutive weeks.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results.Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients with a platelet count ≥50×109/L for at least 8 consecutive weeks.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results.Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients experiencing WHO bleeding grade ≥ 2.
All bleeding events will be assessed by the Investigator according to the WHO bleeding scale where the severity of the bleeding is graded from 0 to 4 (0=no bleeding; 1=petechial bleeding; 2=mild blood loss (clinically significant); 3=gross blood loss; requires transfusion (severe); 4=debilitating blood loss, retinal or cerebral associated with fatality).
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients requiring rescue medication.
Information will be collected via the patient's medical records.
Measured: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Time from Doptelet treatment start to platelet count ≥30×109/L.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results.Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP.
Measured: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Time from Doptelet treatment start to platelet count ≥50×109/L.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results.Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP.
Measured: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Doptelet dose and dosing frequency per patient (assessed by prescription).
Information will be collected via the patient's medical records.
Measured: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Adherence to Doptelet treatment assessed via the 8-item Morisky Medication Adherence Scale (MMAS-8).
MMAS-8 is an 8-item structured, self-reported medication adherence measure. The self-reported measure of medication taking was developed from a previously validated 4-item scale and supplemented with additional items addressing the circumstances surrounding adherence behavior. Each item measures a specific medication-taking behavior and not a determinant of adherence behavior. Response categories are yes/no for each item with a dichotomous response and a 5-point Likert response for the last item. Adherent patients are identified with the score of 8 on the scale, medium adherers with a score of 6 to <8, and low adherers with a score of <6.
The MMAS-8 Scale, content, name, and trademarks are protected by US copyright and trademark laws. Permission for use of the scale and its coding is required. A license agreement is available from Donald E. Morisky, ScD, ScM, MSPH; donald.morisky@moriskyscale.com.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Reason for ITP treatment discontinuation or change from one ITP treatment to another, prior to as well as during the study.
Information will be collected via the patient's medical records.
Measured: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Patient satisfaction with outcome of Doptelet treatment using the Treatment Satisfaction Questionnaire for Medication (TSQM-9).
TSQM-9 is a self-administered generic measure to assess patients' satisfaction with their medication. It consists of nine items distributed across three dimensions: Effectiveness (3 items), Convenience (3 items) and Global satisfaction scale (3 items). Each domain score ranges from 0 to 100, higher score indicating greater satisfaction.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Physician satisfaction with outcome of Doptelet treatment using a 5 point scale.
The treating physician will evaluate satisfaction with the Doptelet treatment by answering the question: "On a scale of 1-5 with 5 being highly satisfied and 1 being highly dissatisfied, how would you rate your satisfaction with the desired treatment outcome of the Doptelet treatment?"
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Physician assessment of clinical change of Doptelet treatment using the Clinical Global Impression of Change (CGIC) scale.
The treating physician will evaluate the clinical change of Doptelet treatment by grading the change on a 7-point scale; very much improved, much improved, minimally improved, no change, minimally worse, much worse, very much worse.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in the European Quality of Life - 5 Dimensions (EQ-5D-5L) scale.
The EQ-5D is a standardized generic instrument for use as a measure of health outcome. The EQ-5D consists of 2 parts - the EQ-5D descriptive profile which is mapped into a single index value for health status (utility value) and the EQ visual analogue scale (EQ VAS). The EQ-5D-5L descriptive system comprises the following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in the Immune Thrombocytopenic Purpura Patient Assessment Questionnaire (ITP-PAQ).
ITP-PAQ is a disease-specific self-administered tool which was developed and validated to assess health related quality of life (HRQoL) in adult patients with ITP using a 4-week recall period. It consists in 44 items distributed across 10 dimensions: Symptoms (6 items), Bother-Physical Health (3 items), Fatigue/Sleep (4 items), Activity (2 items), Fear (5 items), Psychological Health (5 items), Work (4 items), Social Activity (4 items), Women's Reproductive Health (6 items) and Overall QoL (5 items). Each scale is scored from 0 to 100, with higher scores representing better HRQoL.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue) questionnaire.
FACIT-Fatigue is a 13-item scale developed to assess specifically quality of life concerns related to fatigue in patients with chronic diseases. The scale was initially developed to assess cancer-related fatigue, however it has been since then used and psychometrically validated in other chronic diseases, including ITP. The instrument includes items such as tiredness, weakness, listlessness, lack of energy, and the impact of these feelings on daily functioning over the past seven days. Each item is scored on a 5-point Likert Scale ranging from "0-Not at all" to "4-Very much".
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in Patient Global Impression of Change (PGIC) scale.
PGIC will be used to evaluate patients' perception of changes in the severity of their ITP symptoms on the scale: very much improved, much improved, minimally improved, no change, minimally worse, much worse, very much worse.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in Work Productivity and Activity Impairment (WPAI) questionnaire.
WPAI for specific health problem, WPAI:SHP v2.0 questionnaire is a generic and standard instrument developed to measure the effect of specific health problems and symptom severity on work productivity and regular activities during the past seven days. It contains six items which ascertain employment status and quantify absenteeism due to health problems, presenteeism and overall health-related impairment in both paid work and regular activities over the previous 7 days. The WPAI provides quantitative data at the item level compatible with economic modelling.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Healthcare resource use including; Inpatient and /or outpatient visits since last routine visit.
The following data related to in-patient and/or out-patient visits since last routine visit will be collected:
- Length of hospitalization
- Reason for visit
- Surgical procedures
- ICU stay, CT scans
- Regular blood tests and haemato-chemistry blood tests
- Platelet transfusions
- Other.
Measured: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Use of concomitant ITP medications throughout the study.
Information will be collected via the patient's medical records.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Serious adverse events (SAEs)
Information will be collected via reports from the Investigators based on the patient's medical records.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Adverse events of special interest (AESIs) (e.g., thromboembolic events, significant bleeding (WHO bleeding scale grade ≥ 3)).
Information will be collected via reports from the Investigators based on the patient's medical records.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
AEs leading to Doptelet discontinuation.
Information will be collected via reports from the Investigators based on the patient's medical records.
Measured: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2021
- Study start22 Feb 2022
- Primary completion10 Jun 2025
- Study completion10 Jun 2025
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.
- 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
- Other
- Enrolment
- 199 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nina Skuban, MD, Study director, Swedish Orphan BiovitrumVickie McDonald, MD, Study director, Royal London Hospital, London, UK
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04943042
- Sponsor's protocol ID
- Sobi.Doptelet-001
- Acronym
- ADOPT
- Status last verified
- Jul 2025
- First submitted
- 21 Jun 2021
- Last updated
- 14 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: According to Sobi's data sharing policy Sobi may share anonymized clinical study data with qualified researchers. Sobi commits to sharing clinical study data on participant level and summary data for medicines and indications approved by the European Medicines Agency (EMA) and/or the Food and Drug Administration (FDA). Data access will be granted in response to qualified research requests. All requests are evaluated by a cross functional panel of experts within Sobi.
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® (Avatrombopag) in Adult Patients With Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT04943042
- Acronym / sponsor ID
- ADOPT / Sobi.Doptelet-001
- Conditions
- Immune Thrombocytopenia, ITP
- Keywords
- TPO RA (thrombopoietin receptor agonists )
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 199 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 22 Feb 2022 (actual)
- Primary completion
- 10 Jun 2025 (actual)
- Study completion
- 10 Jun 2025 (actual)
- First posted
- 29 Jun 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.
This is a multi-center, observational, Phase 4 study in patients with Immune Thrombocytopenia (ITP) designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice. Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.
Detailed description
This is a multi-center, observational, Phase 4 study in patients with ITP designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice.
Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.
The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective collecting data on usage, effectiveness, safety, patient- and clinician-reported outcomes and health economic parameters whereas the retrospective part will consist of collection of information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Data will be collected for up to 12 months prior to Doptelet treatment start.
Prospective data will be collected at routine clinical visits throughout the study. Patients will be followed for 12 (+6) months and will be enrolled until their first scheduled visit after 12 months of enrollment, or until early termination, whichever occurs first.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Full-analysis set (FAS) | – | Drug: AvatrombopagThe FAS includes all enrolled patients. The FAS will be used for all analyses. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The Investigators will attempt to consecutively enroll all eligible patients who present for a routine clinical visit or during a routine visit where the patient is started on Doptelet treatment (new users of Doptelet).
Key inclusion criteria (5 listed)
- Patient is ≥18 years of age
- Established and well documented ITP diagnosis
- Patient is treated with, or at enrollment prescribed, Doptelet for ITP. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study
- Signed and dated informed consent provided by the patient before any study-related activities are undertaken
- Willing and able to comply with protocol requirements
Key exclusion criteria (6 listed)
- Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
- ITP secondary to Evan's syndrome, lupus and other autoimmune diseases
- ITP secondary to other hematological disorders and hematological malignancies
- ITP secondary to any other malignancies
- ITP secondary to known drug toxicity
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: Doptelet®According to prescription | Drug | Full-analysis set (FAS) |
Primary outcome
Cumulative number of weeks with a platelet count ≥30×109/L during Doptelet treatment.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results. Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP. Platelet counts during rescue medication use and within 4 weeks after stopping a rescue medication or following splenectomy will not be counted in the cumulative number of weeks.
23 secondary outcomes
Cumulative number of weeks with a platelet count ≥50×109/L during Doptelet treatment.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients with a platelet count ≥30×109/L, for at least 8 consecutive weeks.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients with a platelet count ≥50×109/L for at least 8 consecutive weeks.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients experiencing WHO bleeding grade ≥ 2.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Number and proportion of patients requiring rescue medication.
Time frame: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Time from Doptelet treatment start to platelet count ≥30×109/L.
Time frame: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Time from Doptelet treatment start to platelet count ≥50×109/L.
Time frame: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Doptelet dose and dosing frequency per patient (assessed by prescription).
Time frame: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Adherence to Doptelet treatment assessed via the 8-item Morisky Medication Adherence Scale (MMAS-8).
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Reason for ITP treatment discontinuation or change from one ITP treatment to another, prior to as well as during the study.
Time frame: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Patient satisfaction with outcome of Doptelet treatment using the Treatment Satisfaction Questionnaire for Medication (TSQM-9).
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Physician satisfaction with outcome of Doptelet treatment using a 5 point scale.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Physician assessment of clinical change of Doptelet treatment using the Clinical Global Impression of Change (CGIC) scale.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in the European Quality of Life - 5 Dimensions (EQ-5D-5L) scale.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in the Immune Thrombocytopenic Purpura Patient Assessment Questionnaire (ITP-PAQ).
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue) questionnaire.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in Patient Global Impression of Change (PGIC) scale.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Change from enrollment in Work Productivity and Activity Impairment (WPAI) questionnaire.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Healthcare resource use including; Inpatient and /or outpatient visits since last routine visit.
Time frame: Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Use of concomitant ITP medications throughout the study.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Serious adverse events (SAEs)
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Adverse events of special interest (AESIs) (e.g., thromboembolic events, significant bleeding (WHO bleeding scale grade ≥ 3)).
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
AEs leading to Doptelet discontinuation.
Time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2025.
- Individual participant dataSharing planned: Yes. According to Sobi's data sharing policy Sobi may share anonymized clinical study data with qualified researchers. Sobi commits to sharing clinical study data on participant level and summary data for medicines and indications approved by the European Medicines Agency (EMA) and/or the Food and Drug Administration (FDA). Data access will be granted in response to qualified research requests. All requests are evaluated by a cross functional panel of experts within Sobi.
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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.
Nina Skuban, MD study director
Swedish Orphan Biovitrum
Vickie McDonald, MD study director
Royal London Hospital, London, UK
55 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Immune Thrombocytopenia and ITP, run by Swedish Orphan Biovitrum at 55 sites in 9 countries.
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 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 14 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 55 sites in Germany, Spain, United Kingdom, Italy, Czechia and 4 more countries. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Cumulative number of weeks with a platelet count ≥30×109/L during Doptelet treatment. (time frame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.). 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 14 Jul 2025.