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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Jonathan R. Lindner, MD, Oregon Health and Science University
ClinicalTrials.gov Identifier:
NCT01294891
First received: February 11, 2011
Last updated: October 27, 2014
Last verified: October 2014
  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 Oregon Health and Science University:

Primary Outcome Measures:
  • Microvascular perfusion [ Time Frame: 1 hour ] [ Designated as safety issue: No ]
    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

Ages Eligible for Study:   19 Years and older
Genders Eligible for Study:   Both
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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01294891

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

No publications provided

Responsible Party: Jonathan R. Lindner, MD, Professor, Oregon Health and Science University
ClinicalTrials.gov Identifier: NCT01294891     History of Changes
Other Study ID Numbers: IRB00006948
Study First Received: February 11, 2011
Last Updated: October 27, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Oregon Health and Science University:
Rheology
Sickle cell
Paroxysmal nocturnal hemoglobinuria
contrast ultrasound

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

ClinicalTrials.gov processed this record on November 23, 2014