An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP (ADOPT)
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| ClinicalTrials.gov Identifier: NCT04943042 |
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Recruitment Status :
Recruiting
First Posted : June 29, 2021
Last Update Posted : June 2, 2023
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Sponsor:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Swedish Orphan Biovitrum
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Brief 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. Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.
| Condition or disease | Intervention/treatment |
|---|---|
| Immune Thrombocytopenia ITP | Drug: Avatrombopag |
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 Type : | Observational |
| Estimated Enrollment : | 150 participants |
| Observational Model: | Cohort |
| Time Perspective: | Other |
| Official Title: | An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® (Avatrombopag) in Adult Patients With Immune Thrombocytopenia (ITP) |
| Actual Study Start Date : | February 22, 2022 |
| Estimated Primary Completion Date : | May 2025 |
| Estimated Study Completion Date : | May 2025 |
Resource links provided by the National Library of Medicine
| Group/Cohort | Intervention/treatment |
|---|---|
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Full-analysis set (FAS)
The FAS includes all enrolled patients. The FAS will be used for all analyses.
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Drug: Avatrombopag
According to prescription
Other Name: Doptelet® |
Primary Outcome Measures :
- 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.
Secondary Outcome Measures :
- 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. ]
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.
- 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. ]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.
- 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. ]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.
- 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. ]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).
- 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. ]Information will be collected via the patient's medical records.
- 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. ]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.
- 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. ]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.
- 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. ]Information will be collected via the patient's medical records.
- 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. ]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.
- 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. ]Information will be collected via the patient's medical records.
- 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. ]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.
- 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. ]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?"
- 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. ]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.
- 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. ]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.
- 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. ]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.
- 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. ]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".
- 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. ]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.
- 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. ]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.
- 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. ]
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.
- 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. ]Information will be collected via the patient's medical records.
- 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. ]Information will be collected via reports from the Investigators based on the patient's medical records.
- 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. ]Information will be collected via reports from the Investigators based on the patient's medical records.
- 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. ]Information will be collected via reports from the Investigators based on the patient's medical records.
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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).
Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04943042
Contacts
| Contact: Linda Harrill Clinical Study Manager | +1 844-506-3682 | Linda.Harrill@sobi.com | |
| Contact: Rebecca Salgueiro Clinical Study Manager | Rebecca.Salgueiro@sobi.com |
Locations
Show 60 study locations
Sponsors and Collaborators
Swedish Orphan Biovitrum
Investigators
| Study Director: | Stefan Lethagen, MD, PhD | Swedish Orphan Biovitrum AB | |
| Principal Investigator: | Vickie McDonald, MD | Royal London Hospital, London, UK |
| Responsible Party: | Swedish Orphan Biovitrum |
| ClinicalTrials.gov Identifier: | NCT04943042 |
| Other Study ID Numbers: |
Sobi.Doptelet-001 |
| First Posted: | June 29, 2021 Key Record Dates |
| Last Update Posted: | June 2, 2023 |
| Last Verified: | June 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | 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. |
| Time Frame: | Evaluated on a case by case basis |
| Access Criteria: | A decision on data sharing will be based on the following:
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| URL: | https://www.sobi.com/en/policies |
Keywords provided by Swedish Orphan Biovitrum:
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TPO RA (thrombopoietin receptor agonists ) |
Additional relevant MeSH terms:
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Thrombocytopenia Immune System Diseases Purpura, Thrombocytopenic, Idiopathic Blood Platelet Disorders Hematologic Diseases Purpura, Thrombocytopenic Purpura |
Blood Coagulation Disorders Thrombotic Microangiopathies Hemorrhagic Disorders Autoimmune Diseases Hemorrhage Pathologic Processes Skin Manifestations |