An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP

NCT04943042 | Completed

• 1 other identifier: Sobi.Doptelet-001
• Study Type: observational
• Target: 199
• Locations: 9 countries
• Sites: 55

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.

Conditions

Immune Thrombocytopenia; ITP

Keywords

TPO RA (thrombopoietin receptor agonists )

Outcome Measures

Primary Outcomes (1)

• 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.

  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.

  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.

  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.

  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.

  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.

  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.

Study Arms (1)

Full-analysis set (FAS)

The FAS includes all enrolled patients. The FAS will be used for all analyses.

Drug: Avatrombopag

Interventions

Avatrombopag DRUG

According to prescription

Also known as: Doptelet®

Full-analysis set (FAS)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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).

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (55)

Swedish Orphan Biovitrum Research Site 131

Zagreb, Croatia

Location

Swedish Orphan Biovitrum Research Site 122

Brno, Czechia

Location

Swedish Orphan Biovitrum Research Site 123

Ostrava, Czechia

Location

Swedish Orphan Biovitrum Research Site 121

Prague, Czechia

Location

Swedish Orphan Biovitrum Research Site 124

Prague, Czechia

Location

Swedish Orphan Biovitrum Research Site 125

Prague, Czechia

Location

Swedish Orphan Biovitrum Research Site 108

Aschaffenburg, Germany

Location

Swedish Orphan Biovitrum Research Site 114

Augsburg, Germany

Location

Swedish Orphan Biovitrum Research Site 116

Augsburg, Germany

Location

Swedish Orphan Biovitrum Research Site 103

Bad Homburg, Germany

Location

Swedish Orphan Biovitrum Research Site 106

Berlin, Germany

Location

Swedish Orphan Biovitrum Research Site 113

Dresden, Germany

Location

Swedish Orphan Biovitrum Research Site 102

Frankfurt, Germany

Location

Swedish Orphan Biovitrum Research Site 105

Frankfurt, Germany

Location

Swedish Orphan Biovitrum Research Site 104

Hanover, Germany

Location

Swedish Orphan Biovitrum Research Site 112

Kaiserslautern, Germany

Location

Swedish Orphan Biovitrum Research Site 101

Kassel, Germany

Location

Swedish Orphan Biovitrum Research Site 115

Leipzig, Germany

Location

Swedish Orphan Biovitrum Research Site 109

Schorndorf, Germany

Location

Swedish Orphan Biovitrum Research Site 111

Stolberg, Germany

Location

Swedish Orphan Biovitrum Research Site 509

Bologna, Italy

Location

Swedish Orphan Biovitrum Research Site 504

Catania, Italy

Location

Swedish Orphan Biovitrum Research Site 502

Florence, Italy

Location

Swedish Orphan Biovitrum Research Site 506

Meldola, Italy

Location

Swedish Orphan Biovitrum Research Site 511

Milan, Italy

Location

Swedish Orphan Biovitrum Research Site 510

Milan, Italy

Location

Swedish Orphan Biovitrum Research Site 503

Roma, Italy

Location

Swedish Orphan Biovitrum Research Site 505

Rome, Italy

Location

Swedish Orphan Biovitrum Research Site 202

Apeldoorn, 7334 DZ, Netherlands

Location

Swedish Orphan Biovitrum Research Site 204

Arnhem, Netherlands

Location

Swedish Orphan Biovitrum Research Site 203

The Hague, Netherlands

Location

Swedish Orphan Biovitrum Research Site 302

Bergen, Norway

Location

Swedish Orphan Biovitrum Research Site 301

Oslo, Norway

Location

Swedish Orphan Biovitrum Research Site 611

Barcelona, Spain

Location

Swedish Orphan Biovitrum Research Site 609

Burgos, Spain

Location

Swedish Orphan Biovitrum Research Site 610

Granada, Spain

Location

Swedish Orphan Biovitrum Research Site 601

Madrid, Spain

Location

Swedish Orphan Biovitrum Research Site 605

Madrid, Spain

Location

Swedish Orphan Biovitrum Research Site 607

Murcia, Spain

Location

Swedish Orphan Biovitrum Research Site 606

Oviedo, Spain

Location

Swedish Orphan Biovitrum Research Site 608

Palma de Mallorca, Spain

Location

Swedish Orphan Biovitrum Research Site 602

Salamanca, Spain

Location

Swedish Orphan Biovitrum Research Site 604

Seville, Spain

Location

Swedish Orphan Biovitrum Research Site 603

Vigo, Spain

Location

Swedish Orphan Biovitrum Research Site 802

Basel, Switzerland

Location

Swedish Orphan Biovitrum Research Site 801

Lausanne, Switzerland

Location

Swedish Orphan Biovitrum Research Site 709

Edinburgh, Scotland, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 708

Glasgow, Scotland, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 701

Bristol, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 704

Leicester, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 707

London, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 710

London, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 705

Newcastle, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 702

Nottingham, United Kingdom

Location

Swedish Orphan Biovitrum Research Site 703

Oxford, United Kingdom

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Nina Skuban, MD

  Swedish Orphan Biovitrum AB

  STUDY DIRECTOR

• Vickie McDonald, MD

  Royal London Hospital, London, UK

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2021
• First Posted: June 29, 2021
• Study Start: February 22, 2022
• Primary Completion: June 10, 2025
• Study Completion: June 10, 2025
• Last Updated: July 14, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share
• Time Frame: Evaluated on a case by case basis
• Access Criteria: A decision on data sharing will be based on the following: * The scientific merit of the proposal - i.e. the proposal should be scientifically sound, ethical, and have the potential to contribute to the advancement of public health. * The feasibility of the research proposal - i.e. the requesting research team must be scientifically qualified and have the resources to conduct the proposed project. * Maintenance of personal integrity - i.e. Sobi will not consider sharing individual data if there is a risk of re-identification of individuals despite a proper anonymisation. Moreover, the patients' informed consent will always be respected. Sobi reserves the right to reject the proposal if the anonymisation process will render unusable data. * Publication of results - the applicants should commit to submit their findings to a peer-reviewed scientific journal, alternatively to present the results at a congress (poster or similar), regardless of the research outcome.

Locations

GB (9); DE (14); CH (2); CZ (5); IT (8); NL (3); NO (2); ES (11); CR (1)