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

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.

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Key information

Conditions

Rheologic Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Bone Marrow Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes Paroxysmal Nocturnal Hemoglobinuria Sickle Cell Disease

Age range

19 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Imaging

Other

Contrast ultrasound perfusion imaging and complete echocardiography

Primary outcomes

Microvascular perfusion

Time frame: 1 hour
- 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

Sponsors and collaborators

Lead sponsor

Oregon Health and Science University

Other

Registry information

Important dates

Study start: 2011
Primary completion: 2014
Study completion: 2014
First posted: Feb 14, 2011
Registry last updated: Oct 28, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed