NCT01020188

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Aug 2009

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2009

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

November 20, 2009

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 25, 2009

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2012

Completed
Last Updated

September 6, 2019

Status Verified

September 1, 2019

Enrollment Period

3 years

First QC Date

November 20, 2009

Last Update Submit

September 4, 2019

Conditions

Paroxysmal Nocturnal Haemoglobinuria

Outcome Measures

Primary Outcomes (1)

  • To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH

    After sample is obtained

Secondary Outcomes (2)

  • 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

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Subjects with paroxysmal nocturnal haemoglobinuria

You may qualify if:

  • Presence of a PNH clone

You may not qualify if:

  • Longterm anticoagulation for previous venous thrombosis

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

King's College Hospital NHS Trust

London, SE5 9RS, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Officials

  • Raj K Patel, MD

    King's College Hospital NHS Trust

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER

Study Record Dates

First Submitted

November 20, 2009

First Posted

November 25, 2009

Study Start

August 1, 2009

Primary Completion

August 1, 2012

Study Completion

August 1, 2012

Last Updated

September 6, 2019

Record last verified: 2019-09

Locations

GB(1)