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Study of Global Coagulation Tests in Patients With Paroxysmal Nocturnal Haemoglobinuria

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: NCT01020188
Recruitment Status : Completed
First Posted : November 25, 2009
Last Update Posted : September 5, 2019
Information provided by:
King's College Hospital NHS Trust

Brief Summary:
Paroxysmal nocturnal haemaoglobinuria (PNH) is a rare disease which results in breakdown of the red blood cells and bone marrow failure. It is associated with an increased risk of blood clots. Until recently, treatment has been with blood transfusions and in patients with a blood clot, blood thinners. A new treatment called eculizumab is now standard for patients who require regular blood transfusions. It works in the majority of patients by preventing the breakdown of red blood cells. This can eliminate the need for blood transfusion and reduce the risk of blood clots. It is not well understood why patients with PNH are at high risk of blood clots. The investigators plan to use specialised blood tests to assess the stickiness of the blood before starting eculizumab treatment and monthly after starting treatment. The investigators will compare these tests with standard tests of clotting.

Condition or disease
Paroxysmal Nocturnal Haemoglobinuria

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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Pilot Study Investigating the Role of Thrombin Generation and Rotational Thromboelastometry in Assessing the Prothrombotic Phenotype of Paroxysmal Nocturnal Haemaglobinuria
Study Start Date : August 2009
Actual Primary Completion Date : August 2012
Actual Study Completion Date : August 2012

Primary Outcome Measures :
  1. To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH [ Time Frame: After sample is obtained ]

Secondary Outcome Measures :
  1. To establish the role of thromboelastometry in evaluating the prothrombotic phenotype of PNH [ Time Frame: After sample is obtained ]
  2. To establish effects of eculizumab treatment on thrombin generation and thromboelastometry [ Time Frame: 3-6 months ]

Biospecimen Retention:   Samples Without DNA

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
Subjects with paroxysmal nocturnal haemoglobinuria

Inclusion Criteria:

  • Presence of a PNH clone

Exclusion Criteria:

  • Longterm anticoagulation for previous venous thrombosis

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): NCT01020188

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United Kingdom
King's College Hospital NHS Trust
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
King's College Hospital NHS Trust
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Principal Investigator: Raj K Patel, MD King's College Hospital NHS Trust

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Responsible Party: Dr Raj Patel, King's College Hospital NHS Foundation Trust Identifier: NCT01020188     History of Changes
Other Study ID Numbers: KCH1125
First Posted: November 25, 2009    Key Record Dates
Last Update Posted: September 5, 2019
Last Verified: September 2019
Additional relevant MeSH terms:
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Hemoglobinuria, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases