Paroxysmal Nocturnal Haemoglobinuria Clinical Trial
Official title:
A Pilot Study Investigating the Role of Thrombin Generation and Rotational Thromboelastometry in Assessing the Prothrombotic Phenotype of Paroxysmal Nocturnal Haemaglobinuria
| NCT number | NCT01020188 |
| Other study ID # | KCH1125 |
| Secondary ID | |
| Status | Completed |
| Phase | |
| First received | |
| Last updated | |
| Start date | August 2009 |
| Est. completion date | August 2012 |
| Verified date | September 2019 |
| Source | King's College Hospital NHS Trust |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational |
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.
| Status | Completed |
| Enrollment | 60 |
| Est. completion date | August 2012 |
| Est. primary completion date | August 2012 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: - Presence of a PNH clone Exclusion Criteria: - Longterm anticoagulation for previous venous thrombosis |
| Country | Name | City | State |
|---|---|---|---|
| United Kingdom | King's College Hospital NHS Trust | London |
| Lead Sponsor | Collaborator |
|---|---|
| King's College Hospital NHS Trust |
United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH | After sample is obtained | ||
| Secondary | To establish the role of thromboelastometry in evaluating the prothrombotic phenotype of PNH | After sample is obtained | ||
| Secondary | To establish effects of eculizumab treatment on thrombin generation and thromboelastometry | 3-6 months |