A Pilot Study Investigating the Role of Thrombin Generation and Rotational Thromboelastometry in Assessing the Prothrombotic Phenotype of Paroxysmal Nocturnal Haemaglobinuria

Study of Global Coagulation Tests in Patients With Paroxysmal Nocturnal Haemoglobinuria

Sponsors

Lead sponsor: King's College Hospital NHS Trust

Source 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.

Overall Status Completed
Start Date August 2009
Completion Date August 2012
Primary Completion Date August 2012
Study Type Observational
Primary Outcome
Measure Time Frame
To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH After sample is obtained
Secondary Outcome
Measure Time Frame
To establish the role of thromboelastometry in evaluating the prothrombotic phenotype of PNH After sample is obtained
To establish effects of eculizumab treatment on thrombin generation and thromboelastometry 3-6 months
Enrollment 60
Condition
Eligibility

Sampling method: Non-Probability Sample

Criteria:

Inclusion Criteria:

- Presence of a PNH clone

Exclusion Criteria:

- Longterm anticoagulation for previous venous thrombosis

Gender: All

Minimum age: 18 Years

Maximum age: N/A

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Raj K Patel, MD Principal Investigator King's College Hospital NHS Trust
Location
facility
King's College Hospital NHS Trust
Location Countries

United Kingdom

Verification Date

September 2019

Responsible Party

Name title: Dr Raj Patel

Organization: King's College Hospital NHS Foundation Trust

Has Expanded Access No
Condition Browse
Study Design Info

Observational model: Case-Only

Time perspective: Prospective

Source: ClinicalTrials.gov