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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01294891
• Other study ID #: IRB00006948
• Status: Completed
• Phase: N/A
• First received: February 11, 2011
• Last updated: October 27, 2014
• Start date: February 2011
• Est. completion date: October 2014

Study information

• Verified date: October 2014
• Source: Oregon Health and Science University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical 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. 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: October 2014
• Est. primary completion date: October 2014
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 19 Years and older

Eligibility

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

Study Design

Observational Model: Case Control, Time Perspective: Prospective

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria
• Rheologic Disease
• Sickle Cell Disease

Intervention

Other:
• Imaging
Contrast ultrasound perfusion imaging and complete echocardiography

Locations

Country	Name	City	State
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Oregon Health and Science University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	1 hour	No