Paroxysmal Nocturnal HaemoglobinuriaNCT01020188
Study of Global Coagulation Tests in Patients With Paroxysmal Nocturnal Haemoglobinuria
An observational study of Paroxysmal Nocturnal Haemoglobinuria, run by King's College Hospital NHS Trust at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Haemoglobinuria
- How many people
- 60 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Aug 2009to Aug 2012
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Subjects with paroxysmal nocturnal haemoglobinuria
Who may be able to join
- Presence of a PNH clone
Who cannot join
- Longterm anticoagulation for previous venous thrombosis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study Investigating the Role of Thrombin Generation and Rotational Thromboelastometry in Assessing the Prothrombotic Phenotype of Paroxysmal Nocturnal Haemaglobinuria
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.
- Conditions
- Paroxysmal Nocturnal Haemoglobinuria
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What is being measured
The primary outcome is the main question the study is designed to answer.
To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH
Measured: After sample is obtained
Secondary outcomes (2)
To establish the role of thromboelastometry in evaluating the prothrombotic phenotype of PNH
Measured: After sample is obtained
To establish effects of eculizumab treatment on thrombin generation and thromboelastometry
Measured: 3-6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2009 (estimated)
- Study startAug 2009
- Primary completionAug 2012
- Study completionAug 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HaemoglobinuriaStudies of the same condition, open and under way.
- More trials run by King's College Hospital NHS TrustThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- King's College Hospital NHS Trust
- Sponsor type
- University, hospital or other
- Investigators
- Raj K Patel, MD, Principal investigator, King's College Hospital NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01020188
- Sponsor's protocol ID
- KCH1125
- Status last verified
- Sep 2019
- First submitted
- 20 Nov 2009
- Last updated
- 6 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study Investigating the Role of Thrombin Generation and Rotational Thromboelastometry in Assessing the Prothrombotic Phenotype of Paroxysmal Nocturnal Haemaglobinuria
- ClinicalTrials.gov ID
- NCT01020188
- Acronym / sponsor ID
- KCH1125
- Conditions
- Paroxysmal Nocturnal Haemoglobinuria
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- King's College Hospital NHS TrustUniversity, hospital or other
- Study start
- Aug 2009
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 25 Nov 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Subjects with paroxysmal nocturnal haemoglobinuria
Key inclusion criteria (1 listed)
- Presence of a PNH clone
Key exclusion criteria (1 listed)
- Longterm anticoagulation for previous venous thrombosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH
Time frame: After sample is obtained
2 secondary outcomes
To establish the role of thromboelastometry in evaluating the prothrombotic phenotype of PNH
Time frame: After sample is obtained
To establish effects of eculizumab treatment on thrombin generation and thromboelastometry
Time frame: 3-6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Sep 2019.
Related trials for Paroxysmal Nocturnal Haemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Raj K Patel, MD principal investigator
King's College Hospital NHS Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Haemoglobinuria, run by King's College Hospital NHS Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH (time frame: After sample is obtained). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Sep 2019.