MRA With Feraheme in HHT

NCT02977637 | Completed Phase 1

• 1 other identifier: 15-001308
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

Magnetic resonance (MR) imaging is performed with contrast agents to highlight the blood vessels and allow interpretation and diagnosis of blood vessel abnormalities. HHT (Hereditary Hemorrhagic Telangiectasia) is a disease of blood vessels, and can suffer fatal bleeding if abnormal blood vessels are not detected and treated early. Patients with HHT also require many imaging studies through their lifetimes for surveillance of blood vessels. Many HHT patients also have co-existing iron deficiency anemia from bleeding in their nose and gastrointestinal tract, and receive daily iron therapy. Ferumoxytol is an alternative MR contrast agent, which is FDA (Food and Drug Administration) approved for the treatment of iron deficiency anemia. In addition, it is not associated with the risks to the kidneys of the other agents. The use of ferumoxytol for MR imaging may benefit the patients who do not currently receive imaging due to the contraindications of the conventional contrast agents. It avoids the use of ionizing radiation. Also, the conventional contrast agents are associated with risks. Iodinated contrast in CT is associated with significant risks of kidney damage. Another imaging technique, MR, uses gadolinium based contrast agents. Gadolinium, if used in patients with pre existing kidney dysfunction (defined as GFR \< 30ml/min) is associated with the development of another devastating disease called nephrogenic systemic fibrosis. As HHT patients will require repeated scans throughout their lifetimes, this study will provide them a safer alternative. Ten patients from the HHT clinic in whom the use of ferumoxytol as an MR agent is clinically indicated will be invited to participate in this study, which will determine if MR with ferumoxytol is able to detect and characterize vascular malformations in HHT.

Conditions

Hereditary Hemorrhagic Telangiectasia

Keywords

hht; hereditary; hemorrhagic; telangiectasia; feraheme

Outcome Measures

Primary Outcomes (1)

• Presence or absence of AVM

  Immediate

Secondary Outcomes (10)

• Size of AVM

  Immediate

• Location of AVM

  Immediate

• Overall image quality score

  Immediate

• Artifact score

  Immediate

• Vessel definition score

  Immediate

Study Arms (1)

Feraheme group

EXPERIMENTAL

All patients enrolled in the study will receive Feraheme MRI/MRA to detect vascular malformations. Ferumoxytol in its standard concentration (510 mg in 17 cc) will be administered IV at 0.15-0.21 mg/kg prior to MRI/MRA.

Procedure: Feraheme MRI/MRA

Interventions

Feraheme MRI/MRA PROCEDURE

Also known as: Ferumoxytol

Feraheme group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Definite diagnosis of HHT (clinically or genetically confirmed)
• Known or suspected AVMs in the brain, lung, and/or liver
• Use of ferumoxytol as an MR agent is clinically indicated

You may not qualify if:

• Age \<18
• Unable to have MRI scan
• Prior adverse reaction to ferumoxytol

Sponsors & Collaborators

• University of California, Los Angeles: lead

Study Sites (1)

UCLA

Los Angeles, California, 90095, United States

Location

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic; Telangiectasis

Interventions

Ferrosoferric Oxide

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Ferric Compounds; Iron Compounds; Inorganic Chemicals; Ferrous Compounds; Minerals

Study Officials

• Justin McWilliams, MD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 28, 2016
• First Posted: November 30, 2016
• Study Start: November 1, 2016
• Primary Completion: November 1, 2018
• Study Completion: November 1, 2018
• Last Updated: January 25, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)