Rheologic DiseaseNCT01294891
Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
An observational study of Rheologic Disease and Sickle Cell Disease, run by Oregon Health and Science University at 1 site in United States.
Trial summary
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. Read more about this study
- Who is it for?
- Ages 19 years and olderHealthy volunteers can take part
- Conditions the study is for
- Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Imaging
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2011to Oct 2014
- 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 19 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Patients with SCD or PNH who are seen in the OHSU Hematology clinic and are asked to participate
Who may be able to join
- Established diagnosis of PNH or SCD Age > 18 years old
Who cannot join
- Pregnant or lactating women Presence of significant right to left shunting Allergy to ultrasound contrast agent Reactive airways disease Significant peripheral or coronary artery disease
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health & Science University
Portland, Oregon, 97239
About this study
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops.
- Being tested
- Imaging
- Keywords
- Rheology, Sickle cell, Paroxysmal nocturnal hemoglobinuria, contrast ultrasound
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ImagingOther
Contrast ultrasound perfusion imaging and complete echocardiography
| Study group | Role | What they receive |
|---|---|---|
| Control | Age matched healthy subjects Receives: Other: Imaging | |
| Sickle Cell Patients | Patients with established SCD Receives: Other: Imaging | |
| Paroxysmal Nocturnal Hemoglobinuria patients | Patients with PNH Receives: Other: Imaging |
What is being measured
The primary outcome is the main question the study is designed to answer.
Microvascular perfusion
- Myocardial microvascular blood flow (MBF) and capillary blood velocity (CBV) on study day.
- Renal MBF on study day
- Skeletal muscle MBF and CBV on study day
- Myocardial MBF and CBV during hyperemia
- Skeletal muscle CBV during hyperemia
Measured: 1 hour
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2011 (estimated)
- Study startFeb 2011
- Primary completionOct 2014
- Study completionOct 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rheologic DiseaseStudies of the same condition, open and under way.
- More trials run by Oregon Health and Science UniversityThe 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-control
- Time perspective
- Prospective
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jonathan R. Lindner, MD, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01294891
- Sponsor's protocol ID
- IRB00006948
- Status last verified
- Oct 2014
- First submitted
- 11 Feb 2011
- Last updated
- 28 Oct 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01294891
- Acronym / sponsor ID
- IRB00006948
- Keywords
- Rheology, Sickle cell, Paroxysmal nocturnal hemoglobinuria, contrast ultrasound
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Study start
- Feb 2011
- Primary completion
- Oct 2014 (actual)
- Study completion
- Oct 2014 (actual)
- First posted
- 14 Feb 2011 (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.
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Control | – | Other: ImagingAge matched healthy subjects |
| Sickle Cell Patients | – | Other: ImagingPatients with established SCD |
| Paroxysmal Nocturnal Hemoglobinuria patients | – | Other: ImagingPatients with PNH |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with SCD or PNH who are seen in the OHSU Hematology clinic and are asked to participate
Key inclusion criteria (1 listed)
- Established diagnosis of PNH or SCD Age > 18 years old
Key exclusion criteria (1 listed)
- Pregnant or lactating women Presence of significant right to left shunting Allergy to ultrasound contrast agent Reactive airways disease Significant peripheral or coronary artery disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ImagingContrast ultrasound perfusion imaging and complete echocardiography | Other | Control, Paroxysmal Nocturnal Hemoglobinuria patients, Sickle Cell Patients |
Primary outcome
Microvascular perfusion
Time frame: 1 hour
1. Myocardial microvascular blood flow (MBF) and capillary blood velocity (CBV) on study day. 2. Renal MBF on study day 3. Skeletal muscle MBF and CBV on study day 4. Myocardial MBF and CBV during hyperemia 5. Skeletal muscle CBV during hyperemia
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 28 Oct 2014.
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.
The record lists no contact. Use the official record to reach the sponsor.
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 Rheologic Disease and Sickle Cell Disease, run by Oregon Health and Science University at 1 site in United States.
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 28 Oct 2014. 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 States. 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?
Microvascular perfusion (time frame: 1 hour). 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 28 Oct 2014.