Safety and Pregnancy Outcomes in Thrombocythemia Patients Exposed to XAGRID® (Anagrelide Hydrochloride) Compared to Other Treatments

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00567502
Recruitment Status : Completed
First Posted : December 5, 2007
Results First Posted : November 17, 2015
Last Update Posted : November 17, 2015
Information provided by (Responsible Party):

Brief Summary:
This is an observational safety study being conducted in Europe comparing patients taking Xagrid to patients taking other cytoreductive treatments. The plan is to enrol at least 750 subjects taking Xagrid with up to 3000 subjects taking other cytoreductive therapies. The study will collect follow up data for 5 years for each patient enrolled that will focus on collecting data related to pre-defined events (PDEs) and Suspected Serious Adverse Reactions (SSARs).

Condition or disease
Thrombocythemia, Essential

Study Type : Observational
Actual Enrollment : 3647 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Non-Interventional, Post Authorisation Safety Study, to Continuously Monitor Safety and Pregnancy Outcomes in a Cohort of At-Risk Essential Thrombocythaemia (ET) Subjects Exposed to Xagrid Compared to Other Conventional Cytoreductive Treatments
Study Start Date : May 2005
Actual Primary Completion Date : April 2014
Actual Study Completion Date : April 2014

XAGRID® (anagrelide hydrochloride)
Xagrid + Other cytoreductive
Other cytoreductive

Primary Outcome Measures :
  1. Percentage of Participants With At Least One Pre-Defined Event (PDE), Deaths, Pregnancies [ Time Frame: Up to 5 years ]
    Pre-defined events (PDEs) were evaluated whenever an event occurred and was defined by a panel of independent qualified physicians, blinded to cytoreductive therapy, validated all PDEs prior to analysis (Event Validation Panel). Non-PDE death only included deaths not recorded as outcome of another PDE.

  2. Number of Participants With Suspected Serious Adverse Reaction (SSAR) Events [ Time Frame: Up to 5 years ]
    SSAR: serious adverse event (SAE) that was considered related to cytoreductive therapy. SAE: any untoward medical occurrence that at any dose resulted in death, life-threatening (at the time of the event), in-patient hospitalization/prolongation of existing hospitalization (elective hospitalizations/procedures for pre-existing conditions that had not worsened were excluded), resulted in persistent or significant disability/incapacity or congenital abnormality/birth defect. Relatedness (suspected/not suspected) to XAGRID or other cytoreductive theraphy was determined by the investigator. As for SSARs, it was important to consider whether the events were related to XAGRID or other cytoreductive therapy. A participant was included in Xagrid or other treatment group based on treatment exposure, participants received Xagrid + Other was counted both in Xagrid and other treatment group.

Secondary Outcome Measures :
  1. Event Rate of Thrombohaemorrhagic Events [ Time Frame: Up to 5 years ]
    Event Rate of Thrombohaemorrhagic Events was calculated by dividing number of participants with events by total patient-year exposure. The reporting unit is per 100 participant-years of treatment exposure. Thrombohaemorrhagic Events is a composite endpoint of the PDEs myocardial infarction, angina, stroke, transient ischaemic attack, venous thromboembolic events, intermittent claudication/digital ischaemia, and major haemorrhagic events.

  2. Platelet Count [ Time Frame: Baseline, Month 6,12,18, 24, 30, 36, 42, 48, 54, 60 ]
  3. Duration of Exposure for Each Essential Thrombocythemia (ET) Therapy [ Time Frame: Up to 5 years ]
    Total duration for each participant = sum of [stop date - start date + 1] across all periods of time where the specific treatment was taken during the study, where start date = registration/consent date for treatments started before registration/consent date and/or stop date withdrawal/final date for treatments ongoing at the time of withdrawal/end of study. Where a participant has multiple records of the same therapy on the same day, the therapy is counted once for that day.

  4. Cumulative Dose for Each Essential Thrombocythemia (ET) Therapy [ Time Frame: Up to 5 years ]
    Since the study is observational nature, interpreting the table is difficult due to inconsistencies in reporting the units of the dose.

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients receiving cytoreductive therapy for the treatment of at-risk essential thrombocytopenia (ET).

Inclusion Criteria:

  • High risk ET patients
  • Subjects who can give written informed consent.
  • Subjects taking cytoreductive therapy

Exclusion Criteria:

  • Contraindications listed by the product being used.
  • Patient cannot be participating in another clinical trial.

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 identifier (NCT number): NCT00567502

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Sponsors and Collaborators
Principal Investigator: Gunnar Birgegard, MD University Hospital, Uppsala, Sweden

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Shire Identifier: NCT00567502     History of Changes
Other Study ID Numbers: SPD422-401
First Posted: December 5, 2007    Key Record Dates
Results First Posted: November 17, 2015
Last Update Posted: November 17, 2015
Last Verified: May 2014

Additional relevant MeSH terms:
Thrombocythemia, Essential
Blood Platelet Disorders
Hematologic Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Blood Coagulation Disorders
Hemorrhagic Disorders
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors