Clinical trial NCT04943042: An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP
Official title: An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® (Avatrombopag) in Adult Patients With Immune Thrombocytopenia (ITP)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Immune Thrombocytopenia
ITP
Keywords: TPO RA (thrombopoietin receptor agonists )
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
First posted: 2021-06-29
Enrollment: 199
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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).

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.

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.

Eligibility criteria: Inclusion Criteria:

1. Patient is ≥18 years of age
2. Established and well documented ITP diagnosis
3. 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
4. Signed and dated informed consent provided by the patient before any study-related activities are undertaken
5. Willing and able to comply with protocol requirements

Exclusion Criteria:

1. Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
2. ITP secondary to Evan's syndrome, lupus and other autoimmune diseases
3. ITP secondary to other hematological disorders and hematological malignancies
4. ITP secondary to any other malignancies
5. ITP secondary to known drug toxicity
6. ITP secondary to any other disease considered relevant by the Investigator

Primary outcomes:
measure: Cumulative number of weeks with a platelet count ≥30×109/L during Doptelet treatment.
timeFrame: 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:
measure: Cumulative number of weeks with a platelet count ≥50×109/L during Doptelet treatment.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Number and proportion of patients with a platelet count ≥30×109/L, for at least 8 consecutive weeks.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Number and proportion of patients with a platelet count ≥50×109/L for at least 8 consecutive weeks.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Number and proportion of patients experiencing WHO bleeding grade ≥ 2.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Number and proportion of patients requiring rescue medication.
timeFrame: 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.
measure: Time from Doptelet treatment start to platelet count ≥30×109/L.
timeFrame: 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.
measure: Time from Doptelet treatment start to platelet count ≥50×109/L.
timeFrame: 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.
measure: Doptelet dose and dosing frequency per patient (assessed by prescription).
timeFrame: 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.
measure: Adherence to Doptelet treatment assessed via the 8-item Morisky Medication Adherence Scale (MMAS-8).
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Reason for ITP treatment discontinuation or change from one ITP treatment to another, prior to as well as during the study.
timeFrame: 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.
measure: Patient satisfaction with outcome of Doptelet treatment using the Treatment Satisfaction Questionnaire for Medication (TSQM-9).
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Physician satisfaction with outcome of Doptelet treatment using a 5 point scale.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Physician assessment of clinical change of Doptelet treatment using the Clinical Global Impression of Change (CGIC) scale.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Change from enrollment in the European Quality of Life - 5 Dimensions (EQ-5D-5L) scale.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Change from enrollment in the Immune Thrombocytopenic Purpura Patient Assessment Questionnaire (ITP-PAQ).
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Change from enrollment in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue) questionnaire.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Change from enrollment in Patient Global Impression of Change (PGIC) scale.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Change from enrollment in Work Productivity and Activity Impairment (WPAI) questionnaire.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Healthcare resource use including; Inpatient and /or outpatient visits since last routine visit.
timeFrame: 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.
measure: Use of concomitant ITP medications throughout the study.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Serious adverse events (SAEs)
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: Adverse events of special interest (AESIs) (e.g., thromboembolic events, significant bleeding (WHO bleeding scale grade ≥ 3)).
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
measure: AEs leading to Doptelet discontinuation.
timeFrame: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Other outcomes:
Locations:
city: Zagreb
country: Croatia
facility: Swedish Orphan Biovitrum Research Site 131
city: Brno
country: Czechia
facility: Swedish Orphan Biovitrum Research Site 122
city: Ostrava
country: Czechia
facility: Swedish Orphan Biovitrum Research Site 123
city: Praha
country: Czechia
facility: Swedish Orphan Biovitrum Research Site 121
city: Praha
country: Czechia
facility: Swedish Orphan Biovitrum Research Site 124
city: Praha
country: Czechia
facility: Swedish Orphan Biovitrum Research Site 125
city: Aschaffenburg
country: Germany
facility: Swedish Orphan Biovitrum Research Site 108
city: Augsburg
country: Germany
facility: Swedish Orphan Biovitrum Research Site 114
city: Augsburg
country: Germany
facility: Swedish Orphan Biovitrum Research Site 116
city: Bad Homburg
country: Germany
facility: Swedish Orphan Biovitrum Research Site 103
city: Berlin
country: Germany
facility: Swedish Orphan Biovitrum Research Site 106
city: Dresden
country: Germany
facility: Swedish Orphan Biovitrum Research Site 113
city: Frankfurt
country: Germany
facility: Swedish Orphan Biovitrum Research Site 102
city: Frankfurt
country: Germany
facility: Swedish Orphan Biovitrum Research Site 105
city: Hannover
country: Germany
facility: Swedish Orphan Biovitrum Research Site 104
city: Kaiserslautern
country: Germany
facility: Swedish Orphan Biovitrum Research Site 112
city: Kassel
country: Germany
facility: Swedish Orphan Biovitrum Research Site 101
city: Leipzig
country: Germany
facility: Swedish Orphan Biovitrum Research Site 115
city: Schorndorf
country: Germany
facility: Swedish Orphan Biovitrum Research Site 109
city: Stolberg
country: Germany
facility: Swedish Orphan Biovitrum Research Site 111
city: Bologna
country: Italy
facility: Swedish Orphan Biovitrum Research Site 509
city: Catania
country: Italy
facility: Swedish Orphan Biovitrum Research Site 504
city: Firenze
country: Italy
facility: Swedish Orphan Biovitrum Research Site 502
city: Meldola
country: Italy
facility: Swedish Orphan Biovitrum Research Site 506
city: Milano
country: Italy
facility: Swedish Orphan Biovitrum Research Site 510
city: Milan
country: Italy
facility: Swedish Orphan Biovitrum Research Site 511
city: Roma
country: Italy
facility: Swedish Orphan Biovitrum Research Site 503
city: Rome
country: Italy
facility: Swedish Orphan Biovitrum Research Site 505
city: Apeldoorn
country: Netherlands
facility: Swedish Orphan Biovitrum Research Site 202
city: Arnhem
country: Netherlands
facility: Swedish Orphan Biovitrum Research Site 204
city: Den Haag
country: Netherlands
facility: Swedish Orphan Biovitrum Research Site 203
city: Bergen
country: Norway
facility: Swedish Orphan Biovitrum Research Site 302
city: Oslo
country: Norway
facility: Swedish Orphan Biovitrum Research Site 301
city: Barcelona
country: Spain
facility: Swedish Orphan Biovitrum Research Site 611
city: Burgos
country: Spain
facility: Swedish Orphan Biovitrum Research Site 609
city: Granada
country: Spain
facility: Swedish Orphan Biovitrum Research Site 610
city: Madrid
country: Spain
facility: Swedish Orphan Biovitrum Research Site 601
city: Madrid
country: Spain
facility: Swedish Orphan Biovitrum Research Site 605
city: Murcia
country: Spain
facility: Swedish Orphan Biovitrum Research Site 607
city: Oviedo
country: Spain
facility: Swedish Orphan Biovitrum Research Site 606
city: Palma De Mallorca
country: Spain
facility: Swedish Orphan Biovitrum Research Site 608
city: Salamanca
country: Spain
facility: Swedish Orphan Biovitrum Research Site 602
city: Sevilla
country: Spain
facility: Swedish Orphan Biovitrum Research Site 604
city: Vigo
country: Spain
facility: Swedish Orphan Biovitrum Research Site 603
city: Basel
country: Switzerland
facility: Swedish Orphan Biovitrum Research Site 802
city: Lausanne
country: Switzerland
facility: Swedish Orphan Biovitrum Research Site 801
city: Edinburgh
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 709
state: Scotland
city: Glasgow
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 708
state: Scotland
city: Bristol
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 701
city: Leicester
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 704
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 707
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 710
city: Newcastle
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 705
city: Nottingham
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 702
city: Oxford
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site 703
Study officials:
affiliation: Swedish Orphan Biovitrum AB
name: Nina Skuban, MD
role: STUDY_DIRECTOR
affiliation: Royal London Hospital, London, UK
name: Vickie McDonald, MD
role: STUDY_DIRECTOR
References: