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

NCT01294891 | Completed DMC

• 1 other identifier: IRB00006948
• Study Type: observational
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Rheologic Disease; Sickle Cell Disease; Paroxysmal Nocturnal Hemoglobinuria

Keywords

Rheology; Sickle cell; Paroxysmal nocturnal hemoglobinuria; contrast ultrasound

Outcome Measures

Primary Outcomes (1)

• Microvascular perfusion

  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

  1 hour

Study Arms (3)

Control

Age matched healthy subjects

Other: Imaging

Sickle Cell Patients

Patients with established SCD

Other: Imaging

Paroxysmal Nocturnal Hemoglobinuria patients

Patients with PNH

Other: Imaging

Interventions

Imaging OTHER

Contrast ultrasound perfusion imaging and complete echocardiography

Control; Paroxysmal Nocturnal Hemoglobinuria patients; Sickle Cell Patients

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Oregon Health and Science University: lead

Study Sites (1)

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobinuria, Paroxysmal

Interventions

X-Rays

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Myelodysplastic Syndromes; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Electromagnetic Radiation; Electromagnetic Phenomena; Magnetic Phenomena; Physical Phenomena; Radiation; Radiation, Ionizing

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 11, 2011
• First Posted: February 14, 2011
• Study Start: February 1, 2011
• Primary Completion: October 1, 2014
• Study Completion: October 1, 2014
• Last Updated: October 28, 2014

Record last verified: 2014-10

Locations

US (1)