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Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01294891
First Posted: February 14, 2011
Last Update Posted: October 28, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Jonathan R. Lindner, MD, Oregon Health and Science University
  Purpose
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.

Condition Intervention
Rheologic Disease Sickle Cell Disease Paroxysmal Nocturnal Hemoglobinuria Other: Imaging

Study Type: Observational
Study Design: Observational Model: Case Control
Time Perspective: Prospective
Official Title: Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by Jonathan R. Lindner, MD, Oregon Health and Science University:

Primary Outcome Measures:
  • 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


Enrollment: 6
Study Start Date: February 2011
Study Completion Date: October 2014
Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Control
Age matched healthy subjects
Other: Imaging
Contrast ultrasound perfusion imaging and complete echocardiography
Sickle Cell Patients
Patients with established SCD
Other: Imaging
Contrast ultrasound perfusion imaging and complete echocardiography
Paroxysmal Nocturnal Hemoglobinuria patients
Patients with PNH
Other: Imaging
Contrast ultrasound perfusion imaging and complete echocardiography

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   19 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Patients with SCD or PNH who are seen in the OHSU Hematology clinic and are asked to participate
Criteria

Inclusion Criteria:

  • Established diagnosis of PNH or SCD Age > 18 years old

Exclusion Criteria:

  • 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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01294891


Locations
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
Oregon Health and Science University
  More Information

Responsible Party: Jonathan R. Lindner, MD, Professor, Oregon Health and Science University
ClinicalTrials.gov Identifier: NCT01294891     History of Changes
Other Study ID Numbers: IRB00006948
First Submitted: February 11, 2011
First Posted: February 14, 2011
Last Update Posted: October 28, 2014
Last Verified: October 2014

Keywords provided by Jonathan R. Lindner, MD, Oregon Health and Science University:
Rheology
Sickle cell
Paroxysmal nocturnal hemoglobinuria
contrast ultrasound

Additional relevant MeSH terms:
Anemia, Sickle Cell
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Myelodysplastic Syndromes
Bone Marrow Diseases