Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease

NCT02447627 | Completed

• 1 other identifier: 996SC001
• Study Type: observational
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to determine whether imaging techniques, such as magnetic resonance imaging (MRI), near infrared spectroscopy (NIRS), laser speckle contrast imaging (LSCI), and optical imaging (OI), can detect differences in blood flow and oxygen levels in different organ systems of participants with sickle cell disease (SCD). Differences in blood flow and oxygen levels detected by these techniques will be evaluated to determine their utility as biomarkers of clinical disease pathophysiology.

Conditions

Healthy; Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Blood flow in the brain of adults with severe SCD (4-10 vaso-occlusive crises [VOC]/ year) compared to healthy adults without SCD as assessed by MRI-ASL (arterial spin labeling)

  Up to day 18 post screening visit

Secondary Outcomes (9)

• Kidney blood flow rates as assessed by MRI-SWI (susceptibility-weighted imaging)

  Up to day 21 post screening visit

• Skeletal muscle blood flow rates as assessed by NIRS

  Up to day 21 post screening visit

• Skin blood flow rates as assessed by LSCI

  Up to day 21 post screening visit

• Retinal blood flow rates as assessed by OI

  Up to day 21 post screening visit

• Total oxygen levels in the brain as assessed by MRI-ASL

  Up to day 21 post screening visit

Study Arms (2)

Part A

Cohort 1- Participants with severe SCD (4-10 VOC/year) Cohort 2- Participants with milder SCD (\<4-10 VOC/year) Cohort 3- Healthy volunteers Part A and B can occur in parallel

Part B

Adults with SCD receiving chronic red blood cell exchange transfusion Part A and B can occur in parallel

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Participants with mild to severe VOC

You may qualify if:

• Have a diagnosis of SCD confirmed by hemoglobin analysis.
• Be in stable clinical condition, as determined by the Investigator.
• Subjects enrolled in Part B must also meet the following eligibility criterion at Screening:
• Receiving scheduled standard of care RBC exchange transfusion therapy, with ≥3 transfusions already received.
• Be deemed healthy, as determined by the Investigator, based on the physical examination, medical history, vital signs, 12-lead electrocardiogram (ECG), and clinical laboratory measurements.

You may not qualify if:

• Inability to lie still for ≥5 minutes, claustrophobia sufficient to interfere with generating reliable MRI scans, body weight exceeding 320.0 lbs., or girth exceeding the magnet bore.
• Presence of a metal device affected by MRI (e.g., any type of electronic, mechanical or magnetic implant, cardiac pacemaker, aneurysm clips, implanted cardiac defibrillator) or potential ferromagnetic foreign body (metal slivers, metal shavings, other metal objects) which would be a contraindication for MRI.
• Acute pain crisis requiring hospitalization, with a discharge ≤4 weeks prior to the first imaging visit, or when determined by the Investigator to not be at steady state.
• Recent (≤3 months) treatment with hydroxyurea therapy.

Sponsors & Collaborators

• Bioverativ Therapeutics Inc.: lead

Study Sites (1)

Research Site

Detroit, Michigan, 48201, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood samples will be collected for hematology, blood chemistry, and venous blood gas analysis. Blood samples will also be used for exploratory biomarker development specific to SCD.

MeSH Terms

Conditions

Anemia, Sickle Cell

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

Study Officials

• Medical Director

  Bioverativ Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2015
• First Posted: May 19, 2015
• Study Start: June 1, 2015
• Primary Completion: October 1, 2016
• Study Completion: October 1, 2016
• Last Updated: December 19, 2020

Record last verified: 2017-08

Locations

US (1)